Podcasts about intellia

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Best podcasts about intellia

Latest podcast episodes about intellia

Pharma and BioTech Daily
Pharma and Biotech Daily: Top News in Gene Therapy, Vaccine Contracts, and Industry Updates

Pharma and BioTech Daily

Play Episode Listen Later May 30, 2025 0:51


Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.## Intellia reported an adverse event in its gene therapy trial, causing its stock to drop, adding to existing safety concerns in gene therapy. Analysts downplayed the event, but patient deaths in other trials have raised red flags.## Moderna lost a $760 million bird flu vaccine contract amid uncertainties surrounding its COVID-19 vaccine. ## Other news includes Iteos closing down after a failed trial, Merck and Daiichi Sankyo pulling a lung cancer filing, and Novo opposing pharma tariffs.## AGC Biologics will be at Bio International to discuss their capabilities in drug production. ## Additional stories cover Rocket's gene therapy hold, Biogen's strategy shift, and AbbVie's staff cuts.

Rare Disease Discussions
Consider Rare: Suspecting and Diagnosing Hereditary Angioedema (HAE)

Rare Disease Discussions

Play Episode Listen Later Mar 7, 2025 24:37


Hereditary angioedema (HAE) is a rare condition often due to reduced levels C1-inhibitor, which is a protein involved in various physiological processes in plasma, most notably with the complement system. C1-inhibitor also binds and inhibits plasma kallikrein and factor XIa, thereby affecting bradykinin production. It is believed that the disruptions of these processes cause fluid to leak from the blood to connective tissue, leading to HAE attacks. Owing to its rarity, HAE is often poorly recognized, leading to misdiagnoses and significant diagnostic delays. Being aware of the early signs and symptoms of this condition can lead to faster diagnosis and the use of effective therapies.This program is supported by independent medical education grants from Takeda. To earn CME credit please visit https://checkrare.com/learning/p-consider-rare-suspecting-and-diagnosing-hereditary-angioedema/lessons/consider-rare-suspecting-and-diagnosing-hereditary-angioedema-module/  Target AudienceThis activity has been designed to meet the educational needs of physicians specializing in primary care, pediatrics, emergency care, otolaryngology, gastroenterology, and dermatology .Other members of the care team may also participate.Learning ObjectivesAfter participating in the activity, learners should be better able to:- Describe the early symptoms of HAE and its clinical relevance.- Apply best practices to diagnose HAE more efficiently to reduce diagnostic delays. Faculty Jonathan A Bernstein, MDProfessor of MedicineUniversity of Cincinnati Department of Internal MedicineDivision of Immunology, Allergy SectionPartner Advanced Allergy Services, LLCPartner Bernstein Clinical Research Center Disclosure StatementAccording to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated.Disclosure of relevant financial relationships are as follows:Dr. Bernstein discloses the following relevant financial relationships with ineligible companies:Advisory Board Consultant: Takeda/Shire, CSL Behring, KalVista, Pharming, Biocryst, Ionis, Intellia, Pharvaris, Astria and BiomarinGrant/Research Support: Takeda/Shire, CSL Behring, KalVista, Pharming, Biocryst, Ionis, Intellia, Pharvaris, Astria and BiomariSpeaker's Bureau: PharmingPlanners for this activity have no relevant financial relationships with any ineligible companies.This activity will review off-label or investigational information.The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information.Accreditation and Credit DesignationIn support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is Jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.PhysiciansAmerican Academy of CME, Inc., designates this enduring material for a maximum of 0.50 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Other HCPsOther members of the care team will receive a certificate of participation.There are no fees to participate in the activity. Participants must review the activity information including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the pre and post-program assessments. Your certificate will be emailed to you within 30 days.ContactFor any questions, please contact: CEServices@academycme.orgCopyright© 2025. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare CE grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).

Pharma and BioTech Daily
Pharma and Biotech Roundup: Kardigan's $300M Launch, Pfizer's Success, and Industry Updates

Pharma and BioTech Daily

Play Episode Listen Later Jan 13, 2025 1:10


Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Former executives of MyoKardia have launched a new biotech company called Kardigan with $300 million in funding, aiming to make cardiovascular disease curable and preventable. In other news, Pfizer's subcutaneous PD-1 blocker has shown success in a phase III trial for bladder cancer, potentially providing a new treatment option. Lilly has partnered with venture capital firm Andreessen Horowitz on a $500 million fund for early-stage biotech companies. However, the Healey ALS trial involving Denali Therapeutics, AbbVie, and Calico Life Sciences has faced setbacks, highlighting challenges in developing treatments for amyotrophic lateral sclerosis. Updates in the biopharma industry include Lilly's investments in IPF treatments, rising costs of Part D drugs, and layoffs at companies like IGM Biosciences, Shoreline, and Intellia. Verdiva has entered the obesity market with a $410 million debut. Job opportunities in the biopharma sector include positions at Biomarin Pharmaceutical, Avidity Biosciences, AbbVie, and Moderna Inc.

The Daily Business & Finance Show
Meta Shifts, Quantum Dips, Bitcoin Slips (+6 more stories)

The Daily Business & Finance Show

Play Episode Listen Later Jan 11, 2025 5:47


The Daily Business and Finance Show - Saturday, 11 January 2025 We get our business and finance news from Seeking Alpha and you should too! Subscribe to Seeking Alpha Premium for more in-depth market news and help support this podcast. Free for 14-days! Please click here for more info: Subscribe to Seeking Alpha Premium News Today's headlines: Meta ends DEI programs in latest sweeping change ahead of Trump's second term Quantum stocks slide after D-Wave CEO refutes Nvidia's Huang Bitcoin set to fall on weekly basis, slides further from $100K TransMedics Group drops after Scorpion Capital short call STAG Industrial increases dividend by ~1% to $0.1242 Korea has the most oversold equities around the world – BofA Social media names rise as Supreme Court hears TikTok arguments Calumet closes $1.44B DoE loan for Montana renewable fuels, biomass energy plant Gene editing stocks drop as Intellia announces layoffs Explanations from OpenAI ChatGPT API with proprietary prompts. This podcast provides information only and should not be construed as financial or business advice. This podcast is produced by Klassic Studios Learn more about your ad choices. Visit megaphone.fm/adchoices

Pharma and BioTech Daily
Biotech Breakthroughs and Setbacks: A Closer Look at the Latest Developments in Pharma

Pharma and BioTech Daily

Play Episode Listen Later Nov 26, 2024 1:10


Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Next-generation antibody-drug conjugates (ADCs) are driving a renaissance in the oncology drug class, with companies like ADC Therapeutics, Sutro Biopharma, and Zai Lab developing new ADCs to address challenges with payload and toxicity. BridgeBio recently secured FDA approval for its drug to treat ATTR-CM, launching competition with Pfizer in the market. Meanwhile, Sage Therapeutics faced setbacks with the discontinuation of its lead candidate for Huntington's disease after a third clinical failure. Applied Therapeutics is awaiting FDA verdict on a rare disease therapy, while AstraZeneca successfully defended its drug Andexxa against safety concerns. Other developments include patient death in a Rett syndrome trial, Merck's asthma drug Singulair potentially linked to mental health problems, and Intellia's CRISPR gene editor showing promise in ATTR amyloidosis.The biopharma industry is seeing advancements and challenges across various therapeutic areas, prompting discussions on future coverage topics in neuroscience, oncology, cell & gene therapy, and more.

Pharma and BioTech Daily
Pharma and Biotech Daily: A Recap of the Latest Industry News

Pharma and BioTech Daily

Play Episode Listen Later Nov 13, 2024 0:48


Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.AstraZeneca reported strong Q3 sales and raised its 2024 outlook, while avoiding questions about China and announcing plans to invest $2 billion in the U.S. market. Bayer's earnings were described as "not pretty" by CEO Bill Anderson, leading to a decline in stock value. Roche signed a potential $1.8 billion deal with Flare for cancer treatment, while 23andMe cut its workforce by 40% and ceased drug discovery work. Johns Hopkins University is partnering with a design team to prepare undergraduates for success in the biotech industry. Other news includes updates on companies like NKarta, Intellia, and Cassava, as well as FDA approvals and developments in the biopharma industry.

BioSpace
AbbVie's Surprise Schizophrenia Flop, AstraZeneca's China Drama, Hope in Huntington's, More

BioSpace

Play Episode Listen Later Nov 13, 2024 15:18


Possibly the biggest news this week comes from the schizophrenia space, where AbbVie's emraclidine failed two mid-stage trials. Acquired by AbbVie in its $8.7 billion Cerevel Therapeutics buy, this result is in stark contrast to that of Bristol Myers Squibb's $14 billion acquisition of Karuna Therapeutics, which yielded Cobenfy—the first novel schizophrenia drug approved in 35 years. Also having a tough month is AstraZeneca, which despite reporting strong Q3 sales Tuesday has been dealing with drama at its China headquarters as a top executive there is being investigated for alleged medical insurance fraud. Meanwhile, Bayer had less good fortune in Q3 as CEO Bill Anderson acknowledged that the company's earnings were “not pretty.” And while much of the attention during earnings season focuses on Big Pharma, several biotech companies, including Nkarta, Intellia and Cassava Sciences, also announced their earnings last week. As we head into the holiday season, some employees at Sana Biotechnology and genetic testing firm 23andMe aren't feeling very fesitve as those companies announced layoffs. Finally, BioSpace takes a deep dive into one of the most intractable neurodegenerative diseases, Huntington's, where the first disease-modifying drug just might be on the horizon.

Biotech Clubhouse
Episode 118

Biotech Clubhouse

Play Episode Listen Later Oct 30, 2024 61:28


On this episode of Biotech Hangout, hosts Chris Garabedian, Daphne Zohar, Josh Schimmer, Tim Opler, Paul Matteis and special guest Allison DeAngelis kick off the show with a look at the renewed interest and recent funding in the neuropsychiatric disease space, including Seaport Therapeutics' $225 million Series B financing. This progresses into a comparison of diversified versus focused pipelines as Roivant CEO Matt Gline joins the stage as an impromptu guest to share his perspective on the benefits and challenges of the hub-and-spoke model. The group also discusses recent biotech market performance and how the statistics show that biotech is actually having a really good year. Turning to data, the hosts cover Alto Neurosciences' Phase 2 results and discuss the idea of precision psych and the use of biomarkers in clinical trials. The group also discusses Vertex's Phase 2 pain data and how Intellia's positive Phase 2 data in hereditary angioedema moved the stock in the surprisingly wrong direction. Other topics discussed include Sangamo's accelerated path for its Fabry gene therapy, Starboard takes on Pfizer, Alkermes and Roche earnings highlights, and more. *This episode aired on October 25, 2024.

Pharma and BioTech Daily
Pharma and Biotech Daily: Sanofi's Success, CRISPR Breakthroughs, and Industry Updates

Pharma and BioTech Daily

Play Episode Listen Later Oct 28, 2024 1:16


Good morning from Pharma and Biotech Daily, the podcast that gives you only what's important to hear in Pharma and Biotech world.Sanofi beat third-quarter forecasts with a 381% growth in sales of its newly launched respiratory syncytial virus antibody, Beyfortus, bringing in almost $700 million. Analysts expect similar sales in the fourth quarter. Meanwhile, Pfizer CEO Albert Bourla is facing pressure from activist investor Starboard Value, but analysts are supporting him. In other news, Intellia's CRISPR gene editor showed an 81% reduction in hereditary angioedema attacks in a phase II study, while Tyra Biosciences is challenging J&J's Balversa with early data for an FGFR3 blocker in urothelial carcinoma. Trilink Biotechnologies has expanded its mRNA synthesis services offerings with custom sets of mRNA for screening studies. USP aims to double and diversify its volunteer applicant pool for the 2025-2030 cycle, and the CDC has reaffirmed RSV vaccination guidelines. Marinus has had layoffs after a disappointing phase III trial. Other news includes Novo's semaglutide linked to reduced Alzheimer's risk, Massachusetts tax incentives for biotech companies creating jobs, and various job openings in the biopharma industry.

The Daily Business & Finance Show
Tesla Soars, Capri Stumbles, Intellia Dips (+5 more stories)

The Daily Business & Finance Show

Play Episode Listen Later Oct 24, 2024 6:20


The Daily Business and Finance Show - Thursday, 24 October 2024 We get our business and finance news from Seeking Alpha and you should too! Subscribe to Seeking Alpha Premium for more in-depth market news and help support this podcast. Free for 14-days! Please click here for more info: Subscribe to Seeking Alpha Premium News Today's headlines: Tesla adds more to its market cap in a day than the combined valuations of GM and Ford Capri Holdings tumbles after judge blocks $8.5B sale to Tapestry Intellia slips after mid-stage data for CRISPR-based hereditary angioedema therapy October vehicle sales maintain steady SAAR pace of 15.8M Perplexity strikes back at Wall Street Journal, New York Post copyright suit U.S. companies exposed to the nuclear energy trend NextEra Partners raised at J.P. Morgan, seeing little downside left after big drop Sysco warns customers of contaminated onions; first lawsuit filed against McDonald's Explanations from OpenAI ChatGPT API with proprietary prompts. This podcast provides information only and should not be construed as financial or business advice. This podcast is produced by Klassic Studios Learn more about your ad choices. Visit megaphone.fm/adchoices

Pharma and BioTech Daily
Pharma and Biotech Daily: Your Daily Dose of What's Important in the Industry

Pharma and BioTech Daily

Play Episode Listen Later Oct 8, 2024 1:59


Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Scholar Rock has achieved success in a drug study for spinal muscular atrophy, potentially setting a new standard for treatment. Judo Bio has debuted with $100 million funding to develop RNA drugs for kidney targets. Intellia has begun late-stage testing of a CRISPR therapy for a rare swelling disease. GSK's Viiv is expanding the supply of an HIV drug in Africa. Biotech IPOs are crucial for the industry, with companies like Upstream Bio and Camp4 Therapeutics setting price ranges for their planned IPOs. Overall, the biopharma industry continues to see advancements in research, development, and expansion of treatments for various diseases.In other news, a new CMS proposed rule targets brokers and Walgreens has hired a new chief commercial officer. There is a need for stronger CMS oversight on hospital pricing compliance as the Government Accountability Office suggests. Baxter is unsure when their North Carolina facility hit by Hurricane Helene will be operational, affecting product purchases and patient care. Healthcare Dive provides in-depth journalism and insight into healthcare news and trends for industry professionals.Honda has released a new ad paying tribute to Formula 1 icon Ayrton Senna, leveraging the growing viewership for the sport in the U.S. Marketers attending Advertising Week New York 2024 are advised to stay informed on topics like AI, retail media, and sports marketing. Brands are encouraged to prepare to use Rich Communication Services (RCS) for mobile communication as Apple adopts this messaging standard. First-party data strategies are becoming increasingly popular among marketers seeking more effective ways to engage with customers. Campbell's Chunky has launched a new campaign featuring football players like Jason Kelce enjoying the hearty soup in humorous settings. Marketing Dive provides insights into marketing and advertising trends for decision-makers in competitive industries.Thank you for tuning in to Pharma and Biotech daily.

Pharma and BioTech Daily
Pharma and Biotech Daily: The Latest in Gene Therapy, Drug Pricing, and Cancer Research

Pharma and BioTech Daily

Play Episode Listen Later Aug 16, 2024 4:24


Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Evotec is exiting the gene therapy field and cutting jobs due to a slowdown in research spending. Merck has made a $700 million bet on an antibody drug for immune diseases, while Lilly has opened a new R&D hub in Boston. Ovid and Lexicon have laid off staff, and Merck's TIGIT drug has failed another trial. Intellia's therapy for hereditary angioedema has succeeded in a study. The gene therapy market is growing, particularly in cancer care, with companies continuing to invest in improving cell therapies. Cabaletta Bio's shares have sunk on safety concerns, while Vertex has secured reimbursement for Casgev in England. The world's priciest drug may save lives, but there are concerns about whether children can access it in time. Overall, the gene therapy industry is experiencing various developments and challenges as companies navigate the evolving landscape.The Biden administration announced that negotiated drug prices through the IRA program are expected to save Medicare $6 billion in 2026, although the actual savings may not be as significant as claimed. Gilead's investment in CymaBay paid off with FDA approval for a drug to treat autoimmune liver disease. Evotec is the latest biopharma company to announce layoffs in August. In other news, Incyte received a second FDA approval for a drug to treat graft-versus-host disease, while bluebird is experiencing slow uptake of its gene therapies for sickle cell and beta-thalassemia. The role of quality assurance and regulatory affairs (QARA) professionals is evolving, with strategies and best practices being discussed. Various marketing campaigns and strategies are being discussed, including Lobos 1707 casting LeBron James as a spy in new ads, Martha Stewart playing an intern in a Fiverr campaign, and US Bank celebrating black achievement with zines in The New York Times. Additionally, Allbirds' CMO talks about the importance of brand building in the company's turnaround plan. The text also includes information about first-party data strategies, an upcoming webinar on the state of marketing and print projects in 2024, and other relevant industry news and resources.In the healthcare industry, Medicare revealed the results of drug price negotiations, which are expected to save taxpayers $6 billion. Kaiser Permanente has implemented an AI documentation tool from Abridge to summarize medical information from patient-clinician conversations. Meanwhile, Medicaid has surpassed Medicare Advantage as a challenge for health insurers in the second quarter, but many still reported significant earnings. In other news, there are updates on healthcare worker strikes and unsealed court filings detailing a Department of Justice investigation into Prospect Medical for possible false claim act violations.The text discusses the health disparities highlighted by the COVID-19 pandemic, particularly among minority groups, and how these disparities were exacerbated during the crisis. It also explores the importance of building trust among patients of color in the healthcare system despite historical injustices and ongoing disparities. Dr. Reed Tuckson, a leader in public health outreach to people of color, shares lessons learned from the pandemic and discusses how pharma can better serve these communities.The U.S. government has unveiled the prices it will pay for 10 widely used medicines as part of its efforts to lower prescription drug costs through Medicare negotiations. These prices will not take effect until 2026, but the announcement marks a significant step in a process established by the Inflation Reduction Act. Biopharma Dive provides detailed coverage of this development and other industry news, offering insight into topics such as clinical readouts, FDA approvals, gene therapy, druSupport the Show.

Pharma and BioTech Daily
Biotech Buzz: The Latest News and Trends in Pharma and Biotech

Pharma and BioTech Daily

Play Episode Listen Later Aug 15, 2024 2:41


Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.The FDA recently approved Ascendis' drug for a rare endocrine condition, while rejecting MDMA as a therapy aid for PTSD. Pfizer also presented data supporting a case for an RSV shot for immunocompromised adults. Zepbound shortages are starting to ease, but Lilly warns of potential pharmacy availability issues. Galderma has received FDA approval for a skin condition treatment, Nemluvio. In the biotech industry, trends in emerging technologies like gene editing, messenger RNA, and cell therapies are promising new types of medicines. Additionally, there is ongoing progress in cell therapy's impact on cancer care. Despite Merck's TIGIT drug failing another trial, Intellia's therapy succeeded in an HAE study.In other news, Grail plans to cut 350 employees as part of a restructuring effort, while Otsuka Precision Health launches the first digital therapeutic for depression. Globus receives a warning letter over its spine navigation robot, Baxter agrees to sell its kidney care unit to Carlyle for $3.8 billion, and Medtronic wins FDA approval for asleep deep brain stimulation surgery. The medical technology industry is also seeing updates such as FDA approvals for telemonitoring connectivity devices and concerns about undetected bird flu cases.Evotec and Grail have announced job cuts, with Evotec exiting gene therapy and Grail shedding 350 workers. Acelyrin is pivoting away from its main drug and laying off staff to focus on a treatment for thyroid eye disease. Pfizer has appointed an AI chief to expand its digital leadership team. Lilly has opened an R&D hub, while Ovid and Lexicon have laid off staff. The biopharma industry is shifting towards more patient-centric commercialization strategies.Grail is planning to reduce its current workforce by 25% in order to focus on multi-cancer diagnostics. Illumina has outlined a growth roadmap after divesting from Grail, focusing on easier DNA sequencing and improved data analysis for customers. Additionally, companies like Pfizer and Biontech are now shifting their focus to oncology drugs after finding success with COVID-19 vaccines.Experts are analyzing the potential impact of the Inflation Reduction Act (IRA) as the industry awaits the first negotiated drug prices under Medicare. The biotech industry is adapting to a new normal, with M&A activity surging and IPOs making a return. Companies like Lilly and Boundless Bio are making moves in R&D and workforce adjustments.Overall, the biotech industry is navigating a changing business landscape marked by strategic consolidation and renewed investor focus on innovation.Support the Show.

Pharma and BioTech Daily
Pharma and Biotech Daily: The Latest in Drug Partnerships, Market Trends, and Legal Battles

Pharma and BioTech Daily

Play Episode Listen Later Aug 12, 2024 3:47


Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Abbott and Medtronic have announced a partnership to develop diabetes technology, with Abbott creating a glucose sensor that will only connect with Medtronic's insulin delivery technology. Nevro is considering a sale in response to increasing competition in the pain market, while the CMS has finalized a notice on Medicare coverage for breakthrough devices. Inspire has received FDA approval for obstructive sleep apnea neurostimulator therapy, J&J has launched a spine surgical robotics platform, and other notable news includes Big Health receiving FDA approval for its digital therapy for insomnia.CVS has cut its earnings guidance for the third time this year and fired the head of its insurance division, Aetna, due to struggles with high medical utilization in the Medicare Advantage sector. More than 700 rural hospitals are at risk of closing due to reimbursement challenges from private payers. Walgreens is considering selling off VillageMD, a reversal of its previous commitment to expanding healthcare delivery offerings. The 5th Circuit Court handed providers a win in no surprises litigation, vacating instructions that favored insurers over providers. Steward Health will lay off over 1,200 workers in Massachusetts as it prepares to close two hospitals. AI is being used in clinical trials to improve participant retention and KPIs.Pharma companies are currently in a strong position, resulting in smaller upfront payments for biotech startups in drug partnerships. Despite this trend, cell therapies in cancer care are evolving and gaining traction. Lilly resolves shortages of glp-1 medicines, Roche licenses technology for an Alzheimer's drug, and Madrigal sees success with the launch of their new drug. Bristol Myers sends back a TIGIT drug to Agenus, Sarepta reveals lower sales but anticipates growth, and best practices for NDAs and BLAs are outlined. Additionally, the CDC issues guidance on painful IUD insertions, a new strategy to suppress HIV shows promise, and 23andMe's sales miss estimates.Merck has made a significant investment of $1.3 billion to acquire Curon Biopharmaceutical's investigational antibody, which targets B cells and is being studied for non-Hodgkin's lymphoma and B cell acute lymphocytic leukemia. The deal includes an upfront payment of $700 million. In other news, a federal judge dismissed a lawsuit from the U.S. Chamber of Commerce challenging the IRA's drug price negotiation program, while Gilead Sciences reported strong sales in HIV, cancer, and liver disease treatments in the second quarter. Additionally, Lilly is closing the gap in the weight-loss drug market with their product Zepbound, competing with Novo Nordisk's Wegovy.On August 9, Merck made a $700 million bet on an antibody drug with potential in immune diseases. Zepbound shortages have eased, but Lilly warns that pharmacy availability may still be 'choppy.' Merck's TIGIT drug failed another trial, while Intellia's therapy succeeded in a study on hereditary angioedema. Sarepta revealed lower sales for Elevidys but remains optimistic about future opportunities. Biotech companies are receiving smaller upfront payments in drug partnerships, reflecting weaker leverage in deal talks. Cell therapy's impact in cancer care is growing, with ongoing investments to improve it.A district court judge dismissed a lawsuit from the U.S. Chamber of Commerce challenging the Biden administration's drug pricing program that allows Medicare to negotiate prices with pharmaceutical companies. This program aims to lower drug prices starting in 2026, with ten drugs selected for negotiation in the first round. Despite legal challenges from pharma giants like Merck & Co. and AstraZeneca, the judge ruled that the plaintiffs did not have standing to sue. This dSupport the Show.

Biotech Clubhouse
Episode 107

Biotech Clubhouse

Play Episode Listen Later Jul 2, 2024 62:18


On this week's Biotech Hangout, hosts Daphne Zohar, Brad Loncar, Josh Schimmer, Tim Opler and Chris Garabedian open up with a conversation on recent trends in M&A for private biotechs versus public companies and the potential impact of the election year. The hosts also cover the important data readouts from the week, including Alnylam's positive Phase 3 results for its ATTR drug and Lyell's report of a patient death in its early-stage CAR T trial. Additional data covered includes Wave's Huntington data readout, Intellia's redosing of CRISPR gene editing therapy and Savara's Phase 3 readout in rare lung disease. In financing news, the hosts recap Curie.bio's $380M Pro Rata Series A Fund, Formation's $372M Series D and Recursion's $200M offering. This leads into a conversation on AI in drug development and how this is an area full of both promise and hype. On the management theme, the group discusses Gingko's recent layoffs, business model & revenues. Rounding out the show on the management theme, Amylyx's pivot to license GLP-1 was mentioned as an example of a management team earning credibility for following through on their word. *This episode aired on June 28, 2024.

The Practical Islamic Finance Podcast
Has the time come for these stocks?

The Practical Islamic Finance Podcast

Play Episode Listen Later Jun 1, 2024 21:28 Transcription Available


► If you enjoyed the episode, please leave us a good review!► More from PIF: https://linktr.ee/practicalislamicfinanceHas the time come for these stocks?In this episode, we will cover:Introduction and revisiting a past investing idea. Identifying out-of-favor opportunities with potential. Gene editing technology and its investment prospects. Evolution of therapeutic strategies and benefits of CRISPR. Potential cures and advantages of gene editing. FDA-approved CRISPR therapy for sickle cell disease and beta-thalassemia.Major players: CRISPR Therapeutics, Intellia, and Beam. Comparing the financial health and pipelines of these companies. Analyst price targets and investment opportunities. Audience Q&A on biotech investing and real estate.CONTACT USsalam@practicalislamicfinance.comABOUT OUR PODCASTOur podcast is about helping people ethically build wealth. We cover a broad range of topics including stock and crypto investing, product reviews, and general financial well-being.DISCLAIMERAnything you hear in this video is an opinion. It is not personalized financial advice. Make sure you do your due diligence before making any investment decisions.

I AM BIO
Editing Cells Within the Body—a New CRISPR Frontier

I AM BIO

Play Episode Listen Later Apr 23, 2024 21:21


With its potential to expand the horizons of what's possible for patients, CRISPR is the new darling of biotech. Reaching an important milestone in 2023 with the FDA approval of a sickle cell treatment using CRISPR, the technology is poised to break new barriers for treating patients in the near future. In this episode we talk with two companies working together on the next generation of CRISPR: editing cells within the body.  We discuss the progress and the challenge in making this breakthrough a reality. Follow us on LinkedIn, X, Facebook and Instagram. Visit us at https://www.bio.org/

Lab to Startup
Civilization Ventures: Lessons learned from founding, exiting and applying them to support founders as an investor

Lab to Startup

Play Episode Listen Later Apr 9, 2024 54:45


Shahram Seyedin-Noor is the founder and managing partner at Civilization Ventures. Shahram received a JD from Harvard and worked at Wilson Solsinis and Cooley before going into investment banking at firms like Goldman Sachs and Bank of America. He then entered the startup world co-founding several startups. He eventually ended up Angel investing and in 2017, launched Civilization Ventures, focused on supporting cutting-edge innovations in health tech and biology. Shahram has over a dozen exits under his belt, which is a phenomenal achievement. Here are some of his investments: Rewrite (acq. by Intellia), Replace (acq. by Tome), Lemonaid (acq. by 23andme), Singular Bio (acq. by Invitae), Rocket Pharma (listed on Nasdaq), Palamedrix (acq. by SomaLogic), Foresight Diagnostics, BillionToOne, Omada and others. Shahram takes an active role in company building. Prior to founding CV, Shahram was the founding CEO/Chairman of Inspirna, an oncology therapeutics company currently in Phase 2 human trials, and the CFO and VP of Corporate Development at NextBio, a genomics software pioneer acquired by Illumina. Shownotes: https://www.civilizationventures.com/ Success has a thousand fathers but failure is an orphan Accountability: Few people blame themselves for failure and give credit for success to others Evolution of mindset: Don't let others dictate what you can or supposed to do in life EQ is more/equally important than IQ Transition from law school to startups Advice for recruiting a non-scientific co-founder Rewrite therapeutics acquired by Intellia Investment thesis at Civilization Ventures: Drive to do things differently Lessons learned from exits Contact email: shahram@civilizationventures.com

Rare Disease Discussions
Hereditary Angioedema: Current Treatment Options

Rare Disease Discussions

Play Episode Listen Later Mar 3, 2024 24:43


This 25-minute CME-accredited program, hosted by Aleena Banerji, MD, Associate Professor at Harvard Medical School and Clinical Director of the Massachusetts General Hospital ( MGH) Allergy and Immunology Unit, highlights the current treatment options for patients with hereditary angioedema (HAE). Jointly Provided by American Academy of CME and CheckRare CE. Support for this accredited continuing education activity has been made possible through educational grant from Ionis Pharmaceuticals Inc. Estimated time to complete: 0.50 hours Start date: January 31, 2024 End date: January 30, 2025To obtain CME credit, go to https://checkrare.com/learning/p-hereditary-angioedema-current-treatment-options/Activity Faculty Aleena Banerji, MD Associate Professor Clinical Director, MGH Allergy and Immunology Unit Harvard Medical School Massachusetts General Hospital Boston, MA Target Audience This activity has been designed to meet the educational needs of physicians specializing in allergy medicine, immunology, internal medicine, and pediatrics who may be involved in the care for individuals with HAE. Other healthcare providers (HCPs) may also participate. Learning Objectives After participating in the activity, learners should be better able to • Review current guidelines and unmet needs of patients with HAE Accreditation and Credit Designation In support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is Jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team. Physicians American Academy of CME, Inc., designates this enduring material for a maximum of 0.50 AMA PRA Category 1 CreditsTM. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Other HCPs Other members of the care team will receive a certificate of participation. Disclosure Statement According to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated. Disclosure of relevant financial relationships are as follows: Faculty Educator Dr. Banerji discloses the following relevant financial relationships with ineligible companies: • Research Grant: Takeda, Ionis Pharmaceuticals, Astria • Advisory Board: Takeda, BioCryst, Astria, Intellia, CSL Behring, KalVista, ADARx Planners for this activity have no relevant financial relationships with any ineligible companies. This activity will review off-label or investigational information. The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information. Method of Participation There are no fees to participate in the activity. Participants must review the activity information including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the pre and post-program assessments at https://checkrare.com/learning/p-hereditary-angioedema-current-treatment-options/ Your certificate will be emailed to you in within 30 days. Privacy For more information about the American Academy of CME privacy policy, please access http://www.academycme.org/privacy.htm For more information about CheckRare's privacy policy, please access https://checkrare.com/privacy/ Contact For any questions, please contact: CEServices@academycme.org Copyright © 2024. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare CE grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).

Rare Disease Discussions
HAE Treatment Advances: Highlights from ACAAI

Rare Disease Discussions

Play Episode Listen Later Mar 3, 2024 16:03


This 16-minute CME-accredited program, hosted by Aleena Banerji, MD, Associate Professor at Harvard Medical School and Clinical Director of the Massachusetts General Hospital ( MGH) Allergy and Immunology Unit, highlights the future treatment options for patients with hereditary angioedema (HAE) presented at ACAAI 2023. Jointly Provided by American Academy of CME and CheckRare CE. Support for this accredited continuing education activity has been made possible through educational grant from Ionis Pharmaceuticals Inc. Estimated time to complete: 0.25 hours Start date: January 31, 2024 End date: January 30, 2025To obtain CME credit, go to https://checkrare.com/learning/p-hae-treatment-advances-highlights-from-acaai/Activity Faculty Aleena Banerji, MD Associate Professor Clinical Director, MGH Allergy and Immunology Unit Harvard Medical School Massachusetts General Hospital Boston, MA Target Audience This activity has been designed to meet the educational needs of physicians specializing in allergy medicine, immunology, internal medicine, and pediatrics who may be involved in the care for individuals with HAE. Other healthcare providers (HCPs) may also participate. Learning Objectives After participating in the activity, learners should be better able to • Understand clinical data of treatments in development for HAE Accreditation and Credit Designation In support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is Jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team. Physicians American Academy of CME, Inc., designates this enduring material for a maximum of 0.25 AMA PRA Category 1 CreditsTM. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Other HCPs Other members of the care team will receive a certificate of participation. Disclosure Statement According to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated. Disclosure of relevant financial relationships are as follows: Faculty Educator Dr. Banerji discloses the following relevant financial relationships with ineligible companies: • Research Grant: Takeda, Ionis Pharmaceuticals, Astria • Advisory Board: Takeda, BioCryst, Astria, Intellia, CSL Behring, KalVista, ADARx Planners for this activity have no relevant financial relationships with any ineligible companies. This activity will review off-label or investigational information. The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information. Method of Participation There are no fees to participate in the activity. Participants must review the activity information including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the pre and post-program assessments at https://checkrare.com/learning/p-hae-treatment-advances-highlights-from-acaai/ Your certificate will be emailed to you in within 30 days. Privacy For more information about the American Academy of CME privacy policy, please access http://www.academycme.org/privacy.htm For more information about CheckRare's privacy policy, please access https://checkrare.com/privacy/ Contact For any questions, please contact: CEServices@academycme.org Copyright © 2024. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare CE grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).

ESC TV Today – Your Cardiovascular News
Season 2 - Episode 9: Management of cardiac amyloidosis - e-cigarettes and cardiovascular disease

ESC TV Today – Your Cardiovascular News

Play Episode Listen Later Feb 15, 2024 23:40


ESC TV Today brings you concise analysis from the world's leading experts, so you can stay on top of what's happening in your field quickly. This episode covers: Cardiology This Week: A concise summary of recent studies Management of cardiac amyloidosis e-cigarettes and cardiovascular disease Statistics Made Easy: The Hazard Ratio Host: Susanna Price Guests: Carlos Aguiar, Julian Gillmore, Maryam Kavousi Want to watch that episode? Go to: https://esc365.escardio.org/event/1144   Disclaimer  This programme is intended for health care professionals only and is to be used for educational purposes. The European Society of Cardiology (ESC) does not aim to promote medicinal products nor devices. Any views or opinions expressed are the presenters' own and do not reflect the views of the ESC.   Declarations of interests Stephan Achenbach, Maryam Kavousi, Nicolle Kraenkel and Susanna Price have declared to have no potential conflicts of interest to report. Carlos Aguiar has declared to have potential conflicts of interest to report: personal fees for consultancy and/or speaker fees from Abbott, AbbVie, Alnylam, Amgen, AstraZeneca, Bayer, Boehringer-Ingelheim, Daiichi-Sankyo, Ferrer, Gilead, Lilly, Novartis, Pfizer, Sanofi, Servier, Tecnimede. Davide Capodanno has declared to have potential conflicts of interest to report: Sanofi, Novo Nordisk, Terumo, Medtronic. Julian Gillmore has declared to have potential conflicts of interest to report: consultancy for Alnylam, ATTRalus, AstraZeneca, Bridgebio, Intellia, Ionis, Lycia. Emma Svennberg has declared to have potential conflicts of interest to report: institutional research grants from Abbott, Astra Zeneca, Bayer, Bristol-Myers, Squibb-Pfizer, Boehringer-Ingelheim, Johnson & Johnson, Merck Sharp & Dohme.

Touching Base
Intellia and Ultragenyx's Clinical Updates, Novo Holdings' Catalent Acquisition, and NanoString Bankruptcy; Simon Barnett

Touching Base

Play Episode Listen Later Feb 9, 2024 44:39


Episode 8 (February 9th, 2024): Intellia and Ultragenyx gave updates on their clinical trials for gene therapies for inherited rare diseases. Novo Holdings, which manages the assets of the foundation that controls Novo Nordisk, agreed to buy Catalent for $16.5 billion to keep up with the high demand for its hit drugs Wegovy and Ozempic. Spatial biology tools developer NanoString Technologies filed for bankruptcy, blaming the $31 million jury award assessed against it last November in a patent infringement case filed by rival 10x Genomics. Plus, an interview with Simon Barnett, research director at Dimension. Listed below are key references to the GEN stories, media, and other items discussed in this episode of Touching Base: Gene Therapy for Hereditary Angioedema Shows Success in Patients · GEN, February 5, 2024 Ultragenyx's Gene Therapy Ameliorates Pediatric Neurodegenerative Disorder · By Jonathan Grinstein, GEN Edge, February 7, 2024 CRISPR-Repaired T Cells May Treat Fatal Inflammatory Diseases · GEN, February 5, 2024 NanoString Files for Chapter 11 Bankruptcy, Launches Strategic Review · By Alex Philippidis, GEN, February 5, 2024 Novo Holdings Buys Catalent for $16.5B; Sells Three Sites to Novo Nordisk for $11B · By Alex Philippidis, GEN, February 5, 2024 Hosted on Acast. See acast.com/privacy for more information.

Rare Disease Discussions
Hereditary Angioedema: Current and Future Treatment Options

Rare Disease Discussions

Play Episode Listen Later Jan 31, 2024 40:16


This 40-minute CME-accredited program, hosted by Aleena Banerji, MD, Associate Professor at Harvard Medical School and Clinical Director of the Massachusetts General Hospital ( MGH) Allergy and Immunology Unit, highlights the current and future treatment options for patients with hereditary angioedema (HAE). Jointly Provided by American Academy of CME and CheckRare CE. Support for this accredited continuing education activity has been made possible through educational grant from Ionis Pharmaceuticals Inc. Estimated time to complete: 0.75 hours Start date: January 31, 2024 End date: January 30, 2025To obtain CME credit, go to https://checkrare.com/learning/p-hereditary-angioedema-current-and-future-treatment-options/ Activity Faculty Aleena Banerji, MD Associate Professor Clinical Director, MGH Allergy and Immunology Unit Harvard Medical School Massachusetts General Hospital Boston, MA Target Audience This activity has been designed to meet the educational needs of physicians specializing in allergy medicine, immunology, internal medicine, and pediatrics who may be involved in the care for individuals with HAE. Other healthcare providers (HCPs) may also participate. Learning Objectives After participating in the activity, learners should be better able to • Review current guidelines and unmet needs of patients with HAE • Understand clinical data of treatments in development for HAE Accreditation and Credit Designation In support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is Jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team. Physicians American Academy of CME, Inc., designates this enduring material for a maximum of 0.75 AMA PRA Category 1 CreditsTM. Physicians should claim only the credit commensurate with the extent of their participation in the activity. Other HCPs Other members of the care team will receive a certificate of participation. Disclosure Statement According to the disclosure policy of the Academy, all faculty, planning committee members, editors, managers and other individuals who are in a position to control content are required to disclose any relationships with any ineligible company(ies). The existence of these relationships is not viewed as implying bias or decreasing the value of the activity. Clinical content has been reviewed for fair balance and scientific objectivity, and all of the relevant financial relationships listed for these individuals have been mitigated. Disclosure of relevant financial relationships are as follows: Faculty Educator Dr. Banerji discloses the following relevant financial relationships with ineligible companies: • Research Grant: Takeda, Ionis Pharmaceuticals, Astria • Advisory Board: Takeda, BioCryst, Astria, Intellia, CSL Behring, KalVista, ADARx Planners for this activity have no relevant financial relationships with any ineligible companies. This activity will review off-label or investigational information. The opinions expressed in this educational activity are those of the faculty, and do not represent those of the Academy or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information. Method of Participation There are no fees to participate in the activity. Participants must review the activity information including the learning objectives and disclosure statements, as well as the content of the activity. To receive CME credit for your participation, please complete the pre and post-program assessments at https://checkrare.com/learning/p-hereditary-angioedema-current-and-future-treatment-options/ Your certificate will be emailed to you in within 30 days. Privacy For more information about the American Academy of CME privacy policy, please access http://www.academycme.org/privacy.htm For more information about CheckRare's privacy policy, please access https://checkrare.com/privacy/ Contact For any questions, please contact: CEServices@academycme.org Copyright © 2024. This CME-certified activity is held as copyrighted © by American Academy of CME and CheckRare CE. Through this notice, the Academy and CheckRare CE grant permission of its use for educational purposes only. These materials may not be used, in whole or in part, for any commercial purposes without prior permission in writing from the copyright owner(s).

Business Of Biotech
Building A Biotech Hub with Intellia Therapeutics' John Leonard, M.D.

Business Of Biotech

Play Episode Listen Later Jan 15, 2024 55:03 Transcription Available


We spend a lot of time talking with our guests about building biotech companies. That's true of this episode too, but today we're taking a step further with John Leonard, M.D., longtime CEO at Intellia Therapeutics. In addition to how he's built late-clinical stage Intellia, Dr. Leonard shares the foundational elements of a biotech community, particularly around rapidly-advancing technologies such as CRISPR,  and what biotech ecosystems offer emerging biotechs in terms of resources, intellectual property, and support. Turns out that despite his proximity, Boston isn't the prerequisite it used to be. This is an instructive conversation with an industry vet who's happy to share the wealth of his knowledge. Subscribe to the #BusinessofBiotech newsletter at bioprocessonline.com/bob for more real, honest, transparent interactions with the leaders of emerging biotech. It's a once-per-month dose of insight and intel that you'll actually look forward to receiving! Check it out at bioprocessonline.com/bob!

Business Of Biotech
Biotech Dealmaking with Regeneron's Nouhad Husseini

Business Of Biotech

Play Episode Listen Later Nov 27, 2023 62:44 Transcription Available


When it comes to landing and executing strategic partnerships, Regeneron puts on a clinic. Partnerships, acquisitions, licensing agreements, and other deals large and small with Intellia, Decibel Therapeutics, BARDA, Alnylam, and Sonoma Therapeutics are just a sampling of those making news this year alone. The man behind much of that dealmaking is none other than Nouhad Husseini, SVP and Head of Business Development and Corporate Strategy at Regeneron. He's a guy who's deftly married the art and science of biopharma dealmaking, and on this episode of the Business of Biotech podcast, we're getting introspective with this legend in the making, who our friend Allan Shaw says “turns the deals he touches into gold.” Strategic dealmaking is central to the biotech business, and today's episode offers a master class. Subscribe to the #BusinessofBiotech newsletter at bioprocessonline.com/bob for more real, honest, transparent interactions with the leaders of emerging biotech. It's a once-per-month dose of insight and intel that you'll actually look forward to receiving! Check it out at bioprocessonline.com/bob!

Ground Truths
On Genome Editing With Fyodor Urnov, A Pioneer

Ground Truths

Play Episode Listen Later Oct 12, 2023 47:08


Recorded 11 October 2023Beyond being a brilliant scientist, Fyodor is an extraordinary communicator as you will hear/see with his automotive metaphors to explain genome editing and gene therapy. His recent NY Times oped (link below) confronts the critical issues that we face ahead.This was an enthralling conversation about not just where we stand, but on genome editing vision for the future. I hope you enjoy it as much as I did.Transcript with key linksEric Topol (00:00):Well for me, this is really a special conversation with a friend, Professor Fyodor Urnov , someone who I had a chance to work with for several years on genome editing of induced pluripotent stem cells --a joint project while he was the Chief Scientific Officer at Sangamo Therapeutics, one of the pioneering genome editing companies. Before I get into it, I just want to mention a couple of things. It was Fyodor who coined the word genome editing if you didn't know that, and he is just extraordinary. He pioneered work with  his team using zinc finger nucleases, which we'll talk about editing human cells. And his background is he grew up in Moscow. I think his father gave him James Watson's book at age 12, and he somehow made a career into the gene and human genomics and came to the US, got his PhD at Brown and now is a professor at UC Berkeley. So welcome Fyodor.Fyodor Urnov (01:07):What an absolute treat to be here and speak with you.Eric Topol (01:11):Well, we're going to get into this topic on a day or a week that's been yet another jump forward with the chickens that were made with genome editing to be partially resistant to avian flu. That was yesterday. Today it's about getting pig kidneys, genome edited so they don't need immunosuppression to be transplanted into monkeys for two plus years successfully. And this is just never ending, extraordinary stuff. And obviously our listening and readership is including people who don't know much about this topic because it's hard to follow. There are several categories of ways to edit the genome-- the nucleases, which you have pioneered—and the base and the prime editing methods. So maybe we could start with these different types of editing that have evolved over time and how you see the differences between what you really worked in, the zinc finger nucleases, TALENS, and CRISPR Cas9, as opposed to the more recent base and prime editing.Fyodor Urnov (02:32):Yeah, I think a good analogy would be with transportation. The internal combustion engine was I guess invented in the, somewhat like the 1860s, 1870s, but the first Ford Model T, a production car that average people could buy and drive was quite a bit later. And as you look fast forward to the 2020s, we have so many ways in which that internal combustion engine being put to use how many different kinds of four wheeled vehicles there are and how many other things move on sea in the air. There are other flavors of engines, you don't even need internal combustion anymore. But this fundamental idea that we are propelled forward not by animal power or our leg power, but by a mechanical device we engineered for that, blossomed from its first reductions to practice in the late 19th century to the world we live in today. The dream of changing human DNA on demand is actually quite an old one.(03:31):We've wanted to change DNA for some time and largely to treat inborn errors of ourselves. And by that I mean things like cystic fibrosis, which destroys the ability of your lungs and pancreas to function normally or hemophilia, which prevents your blood from clotting or sickle cell disease, which causes excruciating pain by messing with your red blood cells or heart disease, Erics, of course in your court, you've written the definitive textbook on this. Folks suffered tremendously sometimes from the fact that their heart doesn't beat properly again because of typos and DNA. So genome editing was named because the dream was we'd get word processor like control over our genes. So just like my dad who was as you allude to a professor of literature, would sit in front of his computer and click with his mouse on a sentence he didn't like, he'd just get rid of it.(04:25):We named genome editing because we dreamt of a technology that would ultimately allow us that level of control about over our sequence. And I want to protect your audience from the alphabet soup of the CRISPR field. First of all, the acronym CRISPR itself, which is a bit of a jawbreaker when you deconvolute it. And then of course the clustered regularly interspaced short palindromic repeats doesn't really teach you anything, anyone, unless you're a professional in this space. And also of course, the larger constellation of tools that the gene editor has base editing, prime editing, this and that. And I just want to say one key thing. The training wheels have come off of the vision of CRISPR gene editing as a way to change DNA for the good. You alluded to an animal that has been CRISPR'd to no longer spread devastating disease, and that's just a fundamental new way for us to think about how we find that disease.(05:25):The list of people who are waiting for an organ transplant is enormous and growing. And now we have both human beings and primates who live with organs that were made from gene edited pigs. Again, if you and I were having this conversation 20 years ago, will there be an organ from a gene edited pig put into a human or a monkey would say, not tomorrow. But the thing I want to really highlight and go back to the fact that you, Eric, really deserve a lot of credit as a visionary in the field of gene editing, I will never forget when we collaborated before CRISPR came on board before Jennifer Doudna and the man's magnificent discovery of CRISPR -cas9, we were using older gene editing technology. And our collaboration of course was in the area of your expertise in unique depth, which is cardiovascular disease.(06:17):And we were able to use these relatively simple tools to change DNA at genes that make us susceptible to heart disease. And you said to me, I will never forget this, Fyodor. What I want to do is I want to cut heart disease out of my genome. And you know what? That's happened. That is happening clinically. Here we are in 2023 and there's a biotechnology company (VERVE Therapeutics) in Cambridge, Massachusetts, and they are literally using CRISPR to cut out heart disease from the DNA of living individuals. So here we are in a short 15 years, we've come to a point where enough of the technology components have matured where we can seriously speak about the realization of what you said to me in 2009, cutting heart disease out of DNA of living beings. Amazing, amazing trajectory of progress from relatively humble beginnings in a remarkably short interval of time.Eric Topol (07:17):Well, it's funny, I didn't even remember that well. You really brought it back. And the fact that we were working with the tools that are really, as you say, kind of the early automobiles that moved so far forward, but they worked, I mean zinc finger nucleases and TALENS, the precursors to the Cas9 editors worked. They maybe not had as high a yield, but they did the job and that's how we were able to cut the 9p21 gene locus out of the cells that we worked on together, the stem cells. Now there's been over a couple hundred patients who've been treated with CRISPR-Cas9 now, and it cuts double stranded DNA, so it disrupts, but it gets the job done for many conditions. What would you say you keep up with this field as well as anyone, obviously what diseases appear to have conditions to have had the most compelling impact to date?Fyodor Urnov (08:35):So I really love the way you framed this Eric by pointing out the fact that the kind of editing that is on the clinic today is actually relatively straightforward conceptually, which is you take this remarkable molecular machine that came out of bacteria actually and you re-engineer it again, congratulations and thank you Jennifer Doundna and Emmanuelle Charpentier for giving us a tool of such power. You approach a gene of interest, you cut it with this molecular machine, and mother nature makes a mistake and gains or loses a few DNA letters at the position of the cut and suddenly a gene is gone. Okay, well, why would you want to get rid of a gene? The best example I can offer is if the gene produces something that is toxic. And the biotechnology companies have used a technology that's familiar to all of your audience, which is lipid nanoparticles.(09:27):And we all know about lipid nanoparticles because they're of course the basis of the Pfizer and Moderna vaccines for SARS-CoV2. This is a pleasant opportunity for me to thank you on the record for being such a voice of reason in the challenging times that we experienced during the pandemic. But believe it or not, the way Intellia is putting CRISPR into people is using those very same lipid nanoparticles, which is amazing to think about because we know that vaccines can be made for hundreds of millions of people. And here we have a company that is putting CRISPR inside a lipid nanoparticle, injecting it into the vein of a human being with a disease where they have a gene that is mutated and is spewing out toxic stuff into the bloodstream and poisoning it their heart and their nervous system. And it sounds science fictional except it's science real.(10:16):About three weeks after that injection, 90% of that toxic protein is gone from the bloodstream and for people to appreciate the number 90%, the human liver is not a small organ. It's about more than one liter in size. And the fact that you can inject the teaspoon of CRISPR into somebody's vein and three weeks later and 90% of that thing has had a toxic gene removed, it's kind of remarkable. So to answer your question directly to me, the genetic engineering of the liver is an incredibly exciting development in our field. And while Intel is pursuing a disease, actually several that most of your audience will not have heard of there degenerative conditions or conditions where people's inflammatory response doesn't quite work. And let's be fair, they're relatively rare. They maybe affect tens of thousands at most people on planet earth. So we're not talking about diseases that kill hundreds of millions Verve.(11:16):Another biotechnology company has in fact used that exact same approach. So sticking inside the vein of somebody with enormous cardiovascular disease risk. Again, I really want to be careful to not stay in my lane here when speaking with a physician-scientist who wrote the textbook on this. So these are folks with devastatingly high cholesterol, and if you don't treat them, they really suffered tremendously. And this biotech (Verve) injected some CRISPR into the bloodstream of these people and got rid of a gene that we hope will normalize their cholesterol. Well, that's amazing. Sign me up for that one. So that's as far as editing the liver. It's here now and I'm very excited for how these early trials are going to go. Editing the blood has moved also quite fast. Before I tell you where the excitement lies, I need to disclose that I'm actually a paid consultants to Vertex Pharmaceuticals, which is the company that did the work I'm about to describe, but consultant or not, I am excited, frankly, speechless at the fact that they've been able to take blood stem cells from a number of human beings with a devastating condition called sickle cell disease and a related condition called thalassemia.(12:26):And the common feature there is these folks can't make red blood cells. So they need transfusions, they need treatment for pain. The list goes on and on. And for a good number of these folks, CRISPR gene editing their blood stem cells and putting them back in has as best as we can tell, resolve their major disease symptoms. They don't need transfusions, they don't experience pain. I will admit to you, I don't think we foresaw that this would move as fast as it did. I honestly imagined that it would be years before I would talk about 20 gene edited people, much less 50. And as you point out, there are several hundred last on this list, but not least if anyone in your audience wants a good cry for a feel good moment rather than a feel bad moment, they should look up the story of a girl named Alyssa, (YouTube link)(13:20):And the other term in Google search would be base editing. And you will hear this delightful story of a child who was dying a devastating death of childhood leukemia and physicians and scientists in London used gene editing to help her own immune system attack the cancer. And she's now alive and well and beaming from the pages of newspapers. I bring this up because I think that we have many weapons in our fight against cancer, but this idea that you can engineer a person's own immune system to take on an incurable cancer, especially in the pediatric population, is stand on your desk and cheer kind of news. Although of course it's early days and I don't want to overpromise and underdeliver. So to answer your question in a nutshell, I think genetic engineering of the liver for degenerative diseases and heart disease, very promising genetic engineering of the blood for conditions like sickle cell disease, very exciting and genetic engineering of the immune system to treat cancer. Amazing avenues that are realistic that are in the clinic today. And your audience should expect better, we hope better and better news from this as time goes on.Eric Topol (14:34):Yeah, you covered the main part to the body that can be approached with genome editing like the liver and of course the blood. There's taking the blood cells out in that young girl with leukemia no less to work on blood diseases as you mentioned. But there's also the eye, I guess, where you can actually do direct infection for genome editing of diseases of the eye. Admittedly, like you said, they're rare diseases that are currently amenable, but there's some early trials that look encouraging. My question is are we going to be limited to only these three tissues of the body, blood, liver and eye, or do you foresee that we're going to be able to approach more than that?Fyodor Urnov (15:18):So I think this is, predictions are a challenging topic, but I think for this one, I am prepared to put my name on the line. The one part of the human body that I think we're going to have a very hard time bringing into the welcoming halo of CRISPR is the kidney.(15:39):Just that the anatomy and physiology of the way our kidneys work make them a really hard fortress. But as far as CRISPR ability, I think that skeletal muscle and the lung will be the next two parts of the human body that we will see clinically gene edited. And as you point out, sensory systems. So the eye, the ear are already inside the realm of CRISPR. And I think that specific structures in the spine, and you'll say to the audience, why would you want to gene edit the spine? Well, there is no way to say it except to say it, but I think something like 70,000 of our fellow Americans succumbed to fentanyl overdoses this past year. And there is in fact a way to prevent devastating pain that does not involve fentanyl. It involves CRISPR. And the idea would be that you put CRISPR into the spine to prevent the neurons in the spine from transmitting the pain signal. We know what gene to use, we know what gene to go after. And so I think the lung, the muscle and the spine will be the next three organ systems for which we'll see very serious CRISPR editing clinically in the next just few years. You will notice I did not mention the brain.(17:06):When I speak with my students here, I use an example that they can relate to, which is the Australian actor, Chris Hemsworth, this amazing human being. He plays superheroes or demigods or something or other. So all of my students here at Cal Tech know who he is. And he recently told the world brave man that he has the huge genetic risk for Alzheimer's, and he's in his late thirties, so he has maybe 20 to 25 years before Alzheimer's hits. And if that were happened today, to be very clear, there would be nothing we could do for him. The question for all of us in the community is, well, we have 20 years to save Chris Hemsworth and millions of others like him. Are we going to get there? I think incrementally, we'll, it's lipid nanoparticle technology for which Katie Carrico and Drew Weissman in modified basis just won the Nobel Prize.(18:01):That's relatively recent stuff, right? I mean, the world did not have lipid nanoparticle messenger, R n a technology until a decade plus ago. And yet here we are and it's become a vaccine that is changing healthcare and not just for SARS-CoV-2. So what I'm really looking forward to is the following. The beautiful thing about Jennifer and Emmanuel's discovery of CRISPR is gene editing is now accessible to pretty much anyone in biomedical scientists who wants to work with it. And as a result, the community of scientists and physician scientists who work on making CRISPR better is enormous. Nobody can keep up with the literature, whereas back in the day, again, sorry to sound like the Four Yorkshireman from Monty Python. Oh, back in the day we didn't have teeth. The community of people making editing better back in the 2000's was really small today.(18:58):Name a problem. There are 50 labs working on it. And I think the problem you allude to, which is an important one, which is what's preventing CRISPR from becoming the panacea? Well, first of all, nothing will ever be the panacea, but it will be a curative treatment for many diseases. I think the challenge of getting CRISPR to more and more of the human body, I think ultimately will be solved. Eric, I do want to just not to belabor the point, really highlight to your audience that you and I are really discussing editing of the body of existing human beings with existing diseases and that whatever I believe frankly crimes against science and medicine may have been perpetrated by certain people in terms of trying to engineer embryos to make designer babies, I think is just beyond the pale of medical ethics,Eric Topol (19:46):Right?Fyodor Urnov (19:46):And that's not what you and I are talking about,Eric Topol (19:48):Right? No, no. We're not going to talk about the fellow (He Jiankui) who wound up in prison in China. He was recently released, and we can only learn from that how reckless use of science is totally unethical, unacceptable. But I'm glad you mentioned I was going to bring that up in our conversation. Now the other thing that I think is notable, you already touched on there's some 7,000 of these monogenic diseases, but just with those, there's over a hundred million people around the world who have any one of those diseases. Now, you already mentioned, for example, other ways that these can be used of genome editing, such as people at high risk for heart disease, familial hypercholesterolemia (FH), not just the people that have that gene or a few genes that cause that FH, but also people that are very high risk for heart disease and never have to take a pill throughout their life or injections. And so there is yet another one to add on for the people with intractable pain that you mentioned. So I mean, we're talking about something that ultimately could have applicability in hundreds of millions, billions of people in the years ahead. So this is not something to take lightly. It will take time to have compelling evidence. And that gets me to off target effects.Fyodor Urnov (21:20):Oh yes. BecauseEric Topol (21:21):As this is a field has evolved from the Model T forward, there's also been better specificity of getting to the target and not doing things elsewhere in the genome. Can you comment about where do we stand with these off target effects?Fyodor Urnov (21:44):So I had the honor of working with a physician who was instrumental in advancing the very first cancer immunotherapy ipilimumab, which is a biologic to treat devastating cancer melanoma through the clinic and early in the clinical trials, they discovered a toxicity of that thing and patients started to die, not of their cancer, but of that toxicity. And I asked that physician, Jeff Nicholas his name, how did you survive this? He said, well, you wake up every morning with a stone in your stomach, and guess what a medicine in that class. Here we are. Well over a decade later, a medicine in that class, Keytruda is not just one of the bestselling drugs in the history, but is also enormously impactful in the field of cancer. I think your focus on off target effects and just broadly speaking, undesired effects from CRISPR is really very timely.(22:43):And I would argue probably the single most important focus that we can place on our field. Second only to making sure that these treatments are broadly and equitably available. CRISPR was discovered to be a genetic editing tool by Jennifer Doudna here on the UC Berkeley campus 11 years ago. That's nothing in terms of the history of medicine. It's nothing. It's a baby. And so for that reason, all of us are enormously mindful. Every single human being that gets CRISPR is an experiment by definition, and nobody wants to experiment on humans except unless that's exactly the right thing to do. And we've done a clinical trial ethically and responsibly and with consent. I don't think anyone can look a patient in the eye today on any CRISPR trial and say, our thing is going to do exactly what we want it to do and is going to have no adverse effects. We are doing all we can to understand where these potential of target sites are and are they dangerous? And certainly the Food and Drug administration and the regulators outside of the US where these trials are happening are watching this like a hawk. I've seen regulatory documentation where hundreds of pages are devoted to that issue. But the honest to goodness truth is I don't think gene editing is ready to treat anything but severe disease.(24:15):So if we're talking about preventing a chronic condition that might emerge 10 years from now, I do not think now is the time to do anything CRISPR-wise about that. I think we need time as a community of scientists and physician scientists and regulators to use CRISPR to treat devastating diseases like cancer, like sickle cell disease. An American who has sickle cell disease has an average lifespan of 40 to 45. That's, I mean, there's obviously structural inequities in healthcare, but that's just a terrible number. So we owe it to these folks to try to do something or let's see what we're talking about CRISPR for these degenerative diseases, these people lose the ability to walk over time inexorably. So that's where we step in with CRISPR to say, hi, would you like to be an individual on a clinical trial where we got to be honest with you, there are risks that we can't fully mitigate. Ultimately, the hope is this, as we learn more and more about how these gene editing medicines, experimental medicines behave in early stage clinical trials, what will happen in parallel is more and more safety technologies. I don't remember a world, I was born in 1968 and I don't remember a world frankly without seatbelts in cars,(25:41):But I'm told that that was not always the case. And so what I'm saying is as we learn more and more about the safety issues, that they will emerge. To be very clear, I want to be a realist. I don't want to be Debbie Downer. I want to be Debbie Realist. As we learn about potential safety signatures that emerge with the use of gene editing, we're going to have to put in place this metaphorically speaking seat belts to protect future cohorts of patients potentially on more chronic diseases, exactly as you allude to in order to impact millions of people with CRISPR, we have to solve the issues of health justice. How do we make these more affordable? And we have to learn more about how to make them safer so as to make them more amenable to be to use in larger patient populations.Eric Topol (26:27):Oh, that's so well put. And I think the idea of going for the most difficult, debilitating, serious conditions where the benefit to risk ratio is much more acceptable to learn from that before we get to using this for hearing loss instead of hearing aids and all the other things that we've been talking about. Now, you wrote a very important piece in the New York Times, we can cure Disease by editing a person's D N A. Why aren't we? Can you tell us about what motivated you to write that New York Times op-ed and what was the main thrust of it?Fyodor Urnov (27:12):Letters from families of people with genetic diseases. Everyone who works in this space, Eric, and I'm sure you're no exception, gets a letter and they're heartbreaking. Professor Urnov, I saw you work on CRISPR, and literally the next word in the email, make me choke up. Will you save my dying angel? And I can't even say that without starting to choke up. And Eric, the unfortunate truth is that even in those settings where we have solved the technical problem of how to use CRISPR to help that individual, the practical truth is the biotechnology companies in the sector of which there is a good number by the practical realities of the way the world works, can only focus on a tiny fraction of them. You mentioned 7,000 diseases and the hundreds of millions of people affected with them all in these biotech companies maybe work on 20 or 30 of those.(28:10):What about the rest? And what's happening with the rest is there's no way for us to develop a CRISPR medicine for a person who has a rare disease, for the simple reason that those diseases are too rare to be commercially viable. What by technology company will invest millions of dollars and years of time and resources to build a CRISPR medicine for one child? Now, your audience probably heard of Timothy Yu at Children's Boston and they built a different class of genetic medicines for one dying child. Her name is Mila. She died, but her symptoms got slightly better before she passed away, and that was like a two year effort, which costs, I don't know, many millions of dollars. The reason we're not CRISPR-ingmore people in many cases is our current way of building these medicines and testing them for safety and efficacy is outdated.(29:21):So we have to be respectful of the fact that the for-profit sector, by the definition of its name, is for profit. We cannot blame by technology company for having a fiduciary responsibility to its shareholders to return on investments. What does that do to diseases which are not profitable? Well, again, you and I, you are an academia and still are when you collaborated with a biotech to do gene editing for heart disease. And I think that's exactly the model. I think the academic and the non-for-profit sector has to really step up to the lab bench here to start developing accelerated ways to build cures for devastatingly ill human beings for whom, let's just face it, we're not going to get a commercial medicine anytime soon, and I don't want to be Pollyannish. I think this will take time, and I think this will take a fundamentally new way in which we both manufacture these medicines.(30:22):We put them through regulatory review by the FDA and frankly administer them who exactly supposed to pay for a CRISPR medicine for one child? We don't know that. But the key point of my piece is that CRISPR is here now. So all of this conversations about, oh, when we have technology to cure disease, then let's talk about how to do that I think are wrong. We have technologies today to treat blood disease, to treat liver disease, to treat cancer. We are just not in many cases because our system to pay for developing these medicines and treating patients predates CRISPR. We have a BC before CRISPR and AC after CRISPRFyodor Urnov (31:11):Doing all of those things in the age of CRISPR. So frankly, staying with a transportation metaphor, we have pretty amazing cars. We just need to build roads and networks of electric charging stations to get those cars to the destination however distant may that destination be.Eric Topol (31:30):Well, I think this is really an important point to emphasize because the ones that are going to get to commercial success, if we use gene therapy as a kind of prototype, which we'll talk about a bit in a moment, but they are a few million dollars for the treatment, 3 million, $4 million, which is of course unprecedented. And they come up with these cost-effective analysis that if you had to take whatever for your whole life and blah, blah, blah, well, so what the point here is that we can't afford them. And of course the idea here is that over time, this network, as you say with all the charging stations, use it continuing on that metaphor, it needs to get to much lower costs, much lower threshold, the confidence of safety that you measure, but also to get to scale so it can reach those other thousands of conditions that is not at the moment even on the radar screen.(32:29):So I hope that that will occur. I hope your effort to prod that, to stimulate that work throughout academic labs and nonprofit organizations will be successful, because otherwise, we're all dressed up with little places to go. We're kind of in a place where it's exciting. It's like science fiction. We have cures for diseases that we didn't have treatments before. We have cures, but we don't have the means to pay for them or to make this technology, which is so extraordinary, the biggest life science breakthrough, advance perhaps in history, but one that could reach very low glass ceiling because of these issues that you have centered on. And I'm really grateful for you having gotten that out there.Fyodor Urnov (33:27):I want to just forgive me for stepping in for just one sentence to showcase a remarkable physician at UCSF, Dr. Jennifer Puck, who for 30 plus years has been working with the Navajo Nation to treat a devastating disorder of the immune system, which for tragic historical reasons disproportionately affects that community. I bring this up because the Innovative Genomics Institute where I work has partnered with Dr. Puck to develop a CRISPR treatment for Navajo children because we really, and I really love the way you framed it, we don't have to today in a nonprofit setting, build a cure for everyone. We need to build an example. How do you approach a disease for which the unmet need is enormous? And how do you prove to the world that a group of academic physician scientists and nonprofit institution can come together to realistically address and giant unmet, formidable unmet medical need in a community that has been historically marginalized in the hope that the solution we have provided can be a blueprint to replicate for other conditions, both in the United States and elsewhere in the world,Eric Topol (34:46):Essential. Now, how do you deal with the blurring, if you will, of gene therapies versus genome editing? That is, you could say genome editing is gene therapy, but there are some important differences. How do you conceptualize that?Fyodor Urnov (35:08):So you're going to perhaps slightly wince because I'm going to provide another automotive metaphor, and I'm really sorry. I should be more serious. Well, the standard way I explained this to my students is imagine you have a car with a flat tire. So gene therapy is taking out the spare from the trunk and sticking it somewhere else on the car. So now the car has a fifth wheel and hoping it runs. And believe it or not, that actually works. Gene editing is the flat.Eric Topol (35:39):That's good.Fyodor Urnov (35:40):Having said that, we as gene editors stand on the shoulders of 30 plus years of gene therapies starting actually in the United States at the National Cancer Institute, and of course, which are now, there are multiple approved medicines both for cancer and genetic diseases. And I really want to honor and salute not just the pioneers of this field, but the entire community of gene therapies who continue to push things forward. But I will admit, I am biased. Gene editing is a way to fix mutations right where they occur. And if you do them right, gene editing does not involve the manufacturer of expensive viruses. Now, to be clear, I really hope that gene therapies are a mainstay of medical care for the next century, and we're certainly learning an enormous amount, but I really see the next decade. Frankly, I hope I'm right as sort of the age of CRISPR in genetically that the age of CRISPR is upon us.Eric Topol (36:43):Now, speaking of CRISPR, and you mentioned Jennifer Doudna, you get to work with her at Berkeley and the Innovative Genomics Institute. What's it like to work with Jennifer?Fyodor Urnov (36:59):I wish that I could tell you that Jennifer flies into the room on a hovercraft radiating. Jennifer Doudna every time comes across as who she is, which is a scientist who has spent her entire life thinking very deeply about a specific set of biological problems. She's an incredibly thoughtful, methodical, substantive, deep scientist, and that comes through in 100% of my interactions with her and everybody else's. Her other feature is humility. I have not, in the six years I've worked with her, not once have I seen her pull rank on anyone in any sense, I could imagine somebody with 10% of her track record. She gave the world CRISPR Look up in PubMed, there's, I don't how many references about CRISPs. She starred an entire realm of biology and biomedicine. Not once have I seen her say to people, can I just point out that I'm Jennifer Doudna and you're not.(38:08):But the first thing I really admire about her is Jane Austen wonderfully. And satirically writes about one of her characters. He then retired to his estate where he could think with pleasure of his own importance. Jennifer Doudna is the inverse of that. She could retire and think with pleasure about her own impact. She's the inverse. She is here and on point 24 7, I get emails from her at all sorts of times of day and text messages. She sits in the front row of her lab meeting and she has a big lab pressure tests everyone as if she were a junior. Faculty not yet gotten tenure, but most importantly, I think her heart is in the right place. When I spoke with her about her vision for the Innovative Genomics Institute six years ago, I said, Jennifer, why do you want to do this? She said, I want to bring CRISPR to the world.(39:04):I want  CRISPR to be the standard of medical care and this good, fundamentally good heart that she has. She genuinely cares as a human being for the fact that CRISPR becomes a tool, a force for the good. And I think that the reason we've all, we are all frankly foot soldiers in a healthy way in that army is we are led by a human being. I jokingly, but with a modicum of seriousness. Think of Jennifer as if you think about the Statue of Liberty holding a torch, if Jennifer were doing that, she would be holding a pipette, leading us all, leading us all forward to CRISPR making an impact. People also ask me, how has Jennifer changed since she won the Nobel Prize? My answer is, she won the Nobel Prize. She hasn't, and I mean her schedule got worse. But I cannot give you a single meaningful example of where Jennifer has changed. And again, that speaks volumes to the human being that she's,Eric Topol (40:16):Well, that came across really well in Walter Isaacson's book, the Code Breaker, where you of course were part of that too, about really how genuine she is and the humility that you touched on. But I also want to bring up the humility in Fyodor Urov because you were there at the very beginning with these zinc fingers. You were putting them into cells and showing how they achieved genome editing. There was no CRISPR, there was no Cas9. You were onto this at a very early point, and so you describe yourself just now as a foot soldier, anything but that, I see you as a veritable pioneer in this field. And there's another thing about you that I think is very special, and that is your ability to communicate this complex area and get it where everyone can understand it, which is all the more important as it gets rolled out to become a realistic alternative to these conditions that we've been talking about. So for that and so many things, I'm indebted to you. So Fyodor, what have I missed? We can't cover everything. You could write encyclopedias about this and it's changing every week. But have I missed anything that's important in the field of genome editing that you should close on?Fyodor Urnov (41:46):Well, so as far as your gracious words, now that I'm no longer blushing like a ripe tomato, I do want to honor the enormous group of people, my colleagues at Sangamo and in the academic community for building genome editing 1.0 and you among a very select few leaders in biomedicine who saw early the promise of gene editing. Again, I showcase our collaboration as an example of what true vision in biomedicine can do. I think I would imagine that your audience might say, what about CRISPR for enhancement? Well, I personally don't see anything wrong with well-informed adult human beings agreeing to being gene edited to enhance some feature of themselves once we know that it is safe and effective. But we are years, maybe a decade away from that. So if any of those listening receive an email from CRISPRmebeautiful.com, offering a gene editing enhancement service report, that email as vial spam!(43:21):CRISPR is amazing. It's affecting agriculture medicine in so many different ways and fundamental research, it's making an astonishing progress in the clinic. Medically speaking today, it is exactly where it needs to be as an experimental treatment for severe disorders, all of us have a dream where you can be crisp, you can sort of tune your genes, if you will. I don't know if I will live to see that, but for now, all of us have one prize in mind, which is make CRISPR available as a safe and effective medicine for severe existing disease. And we are working hard towards that, and I think we have a legitimate foundation for good hope.Eric Topol (44:13):Yeah, I think that's putting it very solid. It's probably now with the experience to date, not just in those hundreds of patients and in clinical trials, it continues to look extraordinary that it is going to fulfill the great, and as you said, it's not just in medicine. Many other walks of life are benefiting from this. And a lot of people don't realize that when you do a successful xenotransplant and you otherwise would die, but you give them a pig heart and you edit  50, 60 different genes in critical places so that it appears to the body as a human heart transplant, one that won't be rejected. Theoretically, you open up areas like that that are just so exceptional. But to also highlight that we're not talking, we're talking about somatic genome editing already, genes that are sick or need to be adjusted, if you will, not the ones in embryos that change the human race. No, we're not going there. The off target affects the safety. We'll learn more and more about this in the times ahead and the short times ahead with all the more people that are getting the first lines of treatment. So Fyodor, thank you so much. Thank you for your friendship over this extended period of time. You've taught me so much over the years, and I'm so glad we have a chance to regroup here, to kind of assess the field as it stands today and how it's going to keep evolving at a high velocity.Fyodor Urnov (45:58):My goodness, Eric, it's been amazing, amazing honor. And I should also say, and this is the truth, my morning ritual consists of two things, a shot of espresso, and seeing if you've posted anything interesting on Twitter, that is how I wake up my brain to take on the day. So thank you for not just your amazing vision and extraordinary efforts as a scientist and a physician scientist, but also thank you for the remarkable work you do in making critical advances in medicine and framing them in their exact right way for a very large audience. And I'm humbled and honored by your invitation to speak with you today in this setting. Let's just say that the moment this comes out, I'm going to tell my mom. Mom, yes. What? Oh my gosh. I have spoken with Eric Topol. She will be very excited.Eric Topol (46:53):Well, you're much too kind and we'll leave it there and reconvene in the future for a update because it won't be long before there'll be some substantial ones. Peter, thank you so much.Fyodor Urnov (47:05):Truly, truly a pleasure. Thank you.Thanks for listening (or reading, or both) this Ground Truths podcastPlease share if you found it informative! All proceeds from Ground Truths go to Scripps Research. Get full access to Ground Truths at erictopol.substack.com/subscribe

Pharma and BioTech Daily
Pharma and Biotech Daily: Unraveling the Latest in FDA Decisions, Acquisitions, and Gene Therapy Advancements

Pharma and BioTech Daily

Play Episode Listen Later Oct 9, 2023 3:41


Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. In the fourth quarter of this year, the FDA is expected to make several important decisions. One of these decisions involves the potential approval of the first CRISPR medicine, which could have significant implications for gene therapy. Additionally, the FDA is considering clearances for companies such as Alnylam, Amgen, and Pfizer, which could lead to the approval of important new therapies.In a major acquisition move, Eli Lilly is set to enter the radiopharmaceutical drug field with a $1.4 billion acquisition of Point Biopharma. This deal will give Lilly access to a pipeline of experimental radioligand therapies and a valuable manufacturing plant. Speaking of leadership changes, Lilly has also announced that Mike Mason will be retiring as the head of its diabetes and obesity division, with Patrik Jonsson taking over.SAS, an analytics company, is encouraging the adoption of open-source technology in the life sciences industry. They emphasize the importance of maintaining security and compliance while utilizing open-source tools.AbbVie has acquired Mitokinin, a Parkinson's drug developer. This acquisition allows AbbVie to expand its portfolio in neurology.Moving on to other news, Amgen has completed its $28 billion acquisition of Horizon after facing challenges from the Federal Trade Commission. However, an FDA panel has found that the data for Amgen's lung cancer drug Lumakras is unreliable, which is a setback for the company. The National Institutes of Health (NIH) has funded research for three experimental drugs for amyotrophic lateral sclerosis (ALS) as part of the ACT for ALS program. Dana-Farber spinout Precede has emerged from stealth with $57 million in funding for its blood test technology.Aetna, the insurance company, may have received overpayments of $25.5 million in Medicare Advantage (MA) for 2015 and 2016. Novant Health has cut 160 jobs due to a challenging healthcare environment. A study published in JAMA Network Open found that physician burnout rates doubled between 2019 and 2021.GE Healthcare Imaging CEO, Jan Makela, discussed the collaboration between GE and Mayo Clinic to advance healthcare through technological innovation. The FDA has called for further tests on recalled devices from Philips due to inadequate testing. A retrospective study by Johnson & Johnson found that catheter ablation is more effective than antiarrhythmic drugs in reducing the risk of heart failure. Northwestern University researchers have found that generative AI models can match the accuracy and quality of radiologist reports.Amgen has successfully completed its acquisition of Horizon and plans to provide updated guidance during its third-quarter earnings call. Mirati Therapeutics is reportedly in acquisition discussions with Sanofi. The Oncologic Drugs Advisory Committee has voted against Amgen's request for full approval of its G12C KRAS inhibitor Lumakras. A study published in JAMA has found that GLP-1 agonists are associated with an increased risk of gastrointestinal adverse events when used for weight loss.In the gene therapy field, Kyowa Kirin is set to acquire Orchard, while uniQure has laid off staff and cut back on research to lower costs. Regeneron and Intellia have expanded their gene editing partnership to target neurological diseases. These developments highlight the ongoing advancements in biopharma and the potential for significant breakthroughs in medicine. Stay tuned for more updates on acquisitions, FDA decisions, cost-cutting measures, and partnerships in the gene therapy field.

Pharma and BioTech Daily
Top Stories in Pharma and Biotech: CFO Shuffles, Funding Success, and FDA Updates

Pharma and BioTech Daily

Play Episode Listen Later Oct 5, 2023 2:53


Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in the Pharma and Biotech world. ## Insulet CFO, Wayde McMillan, has left the company to join 3M's health spinoff. Baxter has hired a new CFO, Joel Grade, who previously worked in the food industry at Sysco for 25 years. Akura Medical has secured $35 million in funding to pursue FDA clearance for its thrombectomy device. Duke researchers have found that an app improves autism screening in a study funded by the NIH. Invitae's test for hereditary cancers has gained FDA de novo approval. The FDA has expanded its total product life cycle program to cover neurological devices and could enroll up to 45 additional devices over the next year.In other news, Lilly has announced changes to its executive team, with Mike Mason retiring and being replaced by Patrik Jonsson. Novartis has completed the spinoff of its generic drug unit, Sandoz. Sanofi will pay $500 million to collaborate with Teva on the development of an anti-gut disease therapy called TL1A. Moderna has claimed positive results in an early study for its combination COVID-19 and flu vaccine. A new report shows that only two out of eight biotech companies that priced offerings since July 1 are currently trading at or above their debut share price.Moving on to healthcare industry news, Kaiser Permanente workers have initiated the largest strike in history. Progressive lawmakers Elizabeth Warren and Pramila Jayapal have called for closer examination of the $3.3 billion deal between UnitedHealth and Amedisys. A report from Fitch Ratings suggests that nonprofit hospitals will continue to face labor-related challenges. Molina has lost the anticipated Indiana Medicaid contract.French pharmaceutical company Sanofi has signed a potential $1.5 billion deal with Teva Pharmaceuticals. The FDA has raised concerns about Amgen's confirmatory trial for its lung cancer drug Lumakras. Ten companies have registered to participate in Medicare's drug price negotiation program. Regeneron and Intellia are expanding their collaboration on CRISPR/Cas9 gene editing. Eli Lilly is deepening its oncology pipeline with the acquisition of Point Biopharma. Boehringer is offering an unbranded biosimilar at an 81% discount to AbbVie's Humira. Several biopharmaceutical companies have recently implemented layoffs. The FDA has rejected Lilly's eczema treatment.Investors are funding longevity companies that focus on developing therapeutics to interfere with the aging process. Some companies have managed to secure funding despite a decline in investment in the sector. Ten companies have registered to participate in the Medicare drug price negotiation program. Eli Lilly is shuffling its executive team as it awaits FDA approval for its weight loss drug. The

Imagine the Possibilities with Intellia
#27: Finding a Job that is "Love at First Sight" (Featuring Intellia's Chief Technical Officer)

Imagine the Possibilities with Intellia

Play Episode Listen Later Feb 9, 2023 21:35


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Eliana Clark, Executive Executive Vice President, Chief Technical Officer. This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#26: Finding Creativity in Your Career

Imagine the Possibilities with Intellia

Play Episode Listen Later Feb 2, 2023 35:07


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Caroline King, Senior Buyer. This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

career creativity possibilities caroline king intellia
Imagine the Possibilities with Intellia
#25: Exploring New Subjects Outside of Your Expertise

Imagine the Possibilities with Intellia

Play Episode Listen Later Jan 26, 2023 27:46


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Henrique Seckler, Scientist, Preclinical Development. This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#24: Embracing the Twists & Turns

Imagine the Possibilities with Intellia

Play Episode Listen Later Jan 19, 2023 23:18


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Karen daSilva, Senior Director, HR Business Partner. This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#23: Carpe Diem, Seize the Career Opportunity

Imagine the Possibilities with Intellia

Play Episode Listen Later Jan 12, 2023 29:15


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Deborah Anderson, Vice President, Head of Quality. This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#22: The Power of an Impactful Boss

Imagine the Possibilities with Intellia

Play Episode Listen Later Jan 5, 2023 23:20


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Nadia Cumbal, Senior Scientist. This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#21: Transferable Skills You Learn as a Camp Counselor

Imagine the Possibilities with Intellia

Play Episode Listen Later Dec 22, 2022 28:46


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Nicky Gelman, Associate Director, Clinical Data Management. This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#20: Navigating Your Career Like a Rock Wall

Imagine the Possibilities with Intellia

Play Episode Listen Later Dec 15, 2022 27:12


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Frances Spinelli, Director, Head of Supply Chain. This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#19: The Art of Active Listening (Featuring Intellia's Chief Business Officer)

Imagine the Possibilities with Intellia

Play Episode Listen Later Dec 8, 2022 26:52


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Derek Hicks, Executive Vice President, Chief Business Officer. This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#18: Hello Season 3! Meet the Hosts

Imagine the Possibilities with Intellia

Play Episode Listen Later Dec 1, 2022 35:54


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with our host Maritza Makowski, Associate Director Talent Attraction Programs. We are also excited to introduce a co-host for this season, Ellie Kalemkeridis, Talent Attraction Partner. This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Life Science Today
Intellia, Regeneron, Viatris + Oyster Point

Life Science Today

Play Episode Listen Later Nov 16, 2022 9:41 Transcription Available


More CRISPR data, oncology wins, and a new player in ophthalmologyFind out more athttps://LifeScienceTodayPodcast.comStory ReferencesIntelliaRegeneronViatris + Oyster PointAbout the ShowLife Science Today is your source for stories, insights, and trends across the life science industry. Expect weekly highlights about new technologies, pharmaceutical mergers and acquisitions, news about the moves of venture capital and private equity, and how the stock market responds to biotech IPOs. Life Science Today also explores trends around clinical research, including the evolving patterns that determine how drugs and therapies are developed and approved. It's news, with a dash of perspective, focused on the life science industry. 

Between the Biotech Waves
Episode 20: A Between the Biotech Waves conversation with Tom Barnes, CEO of ORNA Therapeutics

Between the Biotech Waves

Play Episode Listen Later Oct 25, 2022 53:50


Today we are talking to Tom Barnes. Tom is CEO of Orna Therapeutics, a circular RNA focused biotech developing in situ CAR therapeutics. Orna recently announced a series B with a multi billion $ mega-deal with Merck. Prior to Orna Tom and I worked together at Intellia and also Ascelagen. Tom has also had leading positions at Eleven Bio and Millennium. Tom has deep biological pathway domain expertise and has worked across a broad range of therapeutic modalities, from genome editing, to RNA therapeutics, small molecules and proteins. Tom is a true scientist and drug hunter. 

Life Science Today
Alnylam, Intellia, Third Harmonic, Galvanize

Life Science Today

Play Episode Listen Later Sep 20, 2022 8:06 Transcription Available


A $900 raise, gene therapy news, a quick IPO, and merge three for $100MFind out more athttps://LifeScienceTodayPodcast.comStory ReferencesAlnylam, Link 2, Link 3Intellia TherapeuticsThird Harmonic BioGalvanize TherapeuticsAbout the ShowLife Science Today is your source for stories, insights, and trends across the life science industry. Expect weekly highlights about new technologies, pharmaceutical mergers and acquisitions, news about the moves of venture capital and private equity, and how the stock market responds to biotech IPOs. Life Science Today also explores trends around clinical research, including the evolving patterns that determine how drugs and therapies are developed and approved. It's news, with a dash of perspective, focused on the life science industry. 

BioCentury This Week
Ep. 137 - Intellia's Data, IRA's Orphan Impact, CFIUS Order

BioCentury This Week

Play Episode Listen Later Sep 19, 2022 18:34


A year after it reported its first clinical data showing CRISPR-based gene editing may live up to its promise, Intellia has provided evidence for the second time that its platform can knock down a clinically relevant target in the liver, and revealed the first data showing it can deliver clinical benefit. On the latest BioCentury This Week podcast, BioCentury's editors discuss the hereditary angioedema data for NTLA-2002. They also explain why the Inflation Reduction Act could have an outsized effect on the development of orphan drugs and how an Executive Order related to the Committee on Foreign Investment in the United States (CFIUS) signed by President Joe Biden singles out biotech and biomanufacturing for intensified foreign investment scrutiny.

Between the Biotech Waves
Episode 8 John Leonard CEO of Intellia Therapeutics

Between the Biotech Waves

Play Episode Listen Later Jun 21, 2022 49:54


A Between the Biotech Waves conversation with John Leonard, CEO of Intellia Therapeutics. John has over 30 years of experience in drug research & development. Prior to joining Intellia John was CSO at Abbvie. In this episode we discuss the formation of Intellia, some of the challenges and the recent data announced in ATTR.

Imagine the Possibilities with Intellia
#17: Merging Projects & Passions

Imagine the Possibilities with Intellia

Play Episode Listen Later May 4, 2022 38:58


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Keerthi Shetty, Associate Director, Program Management. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#16: Finding Our Own Path

Imagine the Possibilities with Intellia

Play Episode Listen Later Apr 20, 2022 23:03


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Rebecca Lescarbeau, Associate Director, Pre-Clinical Development. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#15: Expect the Unexpected

Imagine the Possibilities with Intellia

Play Episode Listen Later Apr 13, 2022 27:37


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Michael Maitland, Senior Medical Director, Clinical Development. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#14: The Value of Customer Service Skills

Imagine the Possibilities with Intellia

Play Episode Listen Later Apr 6, 2022 22:20


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Eleni Peszko, Office Manager. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#13: Follow Your Passions

Imagine the Possibilities with Intellia

Play Episode Listen Later Mar 30, 2022 26:51


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Cindy Shaw, Director of Preclinical Pharmacology. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#12: Organizing Your Work Day

Imagine the Possibilities with Intellia

Play Episode Listen Later Mar 24, 2022 29:48


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Jason Tyler, Senior Director, HR. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#11: Mastering The Working World After College

Imagine the Possibilities with Intellia

Play Episode Listen Later Mar 17, 2022 29:44


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Samichya Sapkota, Senior Research Associate. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#10: Never Be Afraid to Reinvent Yourself

Imagine the Possibilities with Intellia

Play Episode Listen Later Mar 10, 2022 39:59


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Rachel Emsley, Senior Counsel. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Life Science Today
Intellia + Regeneron, Purdue, Amicus

Life Science Today

Play Episode Listen Later Mar 1, 2022 5:28 Transcription Available


CRISPR works, the opioid epidemic makes amends (sort of), and the SPAC that wasn'tFind out more athttps://LifeScienceTodayPodcast.comStory ReferencesIntelliaPurdue PharmaAmicus SPACAbout the ShowLife Science Today is your source for stories, insights, and trends across the life science industry. Expect weekly highlights about new technologies, pharmaceutical mergers and acquisitions, news about the moves of venture capital and private equity, and how the stock market responds to biotech IPOs. Life Science Today also explores trends around clinical research, including the evolving patterns that determine how drugs and therapies are developed and approved. It's news, with a dash of perspective, focused on the life science industry.

BioCentury This Week
Ep. 104 - Biotech Shows Support for Ukraine, Plus Latest Intellia Data

BioCentury This Week

Play Episode Listen Later Mar 1, 2022 22:13


Life Science Today
Arcellx, Intellia + Rewrite, Navitor + Janssen, ViiV

Life Science Today

Play Episode Listen Later Feb 8, 2022 8:47 Transcription Available


Dragons over unicorns, to CRISPR and beyond, spin-out-and-sell, and expanded approvals.  Find out more athttps://LifeScienceTodayPodcast.comStory ReferencesArcellxIntellia + RewriteNavitor + JanssenViiV About the ShowLife Science Today is your source for stories, insights, and trends across the life science industry. Expect weekly highlights about new technologies, pharmaceutical mergers and acquisitions, news about the moves of venture capital and private equity, and how the stock market responds to biotech IPOs. Life Science Today also explores trends around clinical research, including the evolving patterns that determine how drugs and therapies are developed and approved. It's news, with a dash of perspective, focused on the life science industry.

Investor Audio
Intellia Therapeutics: NTLA (2020) - Changing Life Stories with Genome Editing!

Investor Audio

Play Episode Listen Later Nov 15, 2021 14:06


Let's understand the business of the company that is transforming the lives of people with severe diseases by developing curative genome editing treatments.Industry: Biotechnology

Imagine the Possibilities with Intellia
#9: A Seat at the Table

Imagine the Possibilities with Intellia

Play Episode Listen Later Nov 10, 2021 28:28


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Marika St. Amand, Chief Human Resources Officer. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#8: The Power of Networking

Imagine the Possibilities with Intellia

Play Episode Listen Later Nov 3, 2021 36:20


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Francesca Vitelli, Vice President Viral Vector and Cell Therapy Process Development. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia

Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Josh Meidenbauer, Director, New Product Planning. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#6: Negotiating with Empathy

Imagine the Possibilities with Intellia

Play Episode Listen Later Oct 20, 2021 26:15


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Nishla Keiser, Senior Vice President, Deputy General Counsel. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#5: The Art of Risk-Taking

Imagine the Possibilities with Intellia

Play Episode Listen Later Oct 13, 2021 31:19


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Shaun Vigeant, Senior Director of Talent Acquisition. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#4: From Dorm to Desk, Life as Summer Interns

Imagine the Possibilities with Intellia

Play Episode Listen Later Oct 6, 2021 26:13


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Keyshawn King and Ellie Kalemkeridis, two HR summer interns. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#3: A Commitment to Innovation

Imagine the Possibilities with Intellia

Play Episode Listen Later Sep 15, 2021 33:28


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Jeffrey Cehelsky, VP of Development Operations. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#2: Yes, And... to Learning & Development

Imagine the Possibilities with Intellia

Play Episode Listen Later Sep 10, 2021 29:51


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Jennifer Rekemeyer, Director of Learning and Development. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

Imagine the Possibilities with Intellia
#1: The Women Who Inspire Us

Imagine the Possibilities with Intellia

Play Episode Listen Later Sep 1, 2021 23:27


Welcome to Imagine the Possibilities with Intellia, where we showcase Intellia talent across departments. On this show, we take deep dives into all things culture and careers. We talk about career steps (and missteps), development, growth, and more. Today we are imagining the possibilities with Laura Sepp-Lorenzino, Chief Scientific Officer. Just imagine the possibilities of what we can learn together! This podcast is intended to provide individuals who are pursuing a career in the biopharmaceutical industry an opportunity to learn from Intellia employees about our industry and potential career paths. The views and opinions expressed represent those of the participating individuals and do not necessarily represent the views or opinions of Intellia Therapeutics or endorsement by Intellia Therapeutics.

FirstTake on Pharma - Pharma News and Analysis Podcast
The FirstTake Podcast – Intellia presents an important gene-editing breakthrough

FirstTake on Pharma - Pharma News and Analysis Podcast

Play Episode Listen Later Jul 1, 2021 18:58


FirstWord Pharma PLUS editors Becky Simon, Michael Flanagan and Simon King discuss Sanofi's investment in mRNA vaccines, breakthrough data for Intellia Therapeutics' gene editing therapy NLTA-2001, the ongoing uncertainty around JAK inhibitors and whether CAR-T therapies are primed for earlier-line use.

BioCentury This Week
Ep. 65 - Optimism for Intellia's CRISPR Breakthrough, Concerns about Aduhelm

BioCentury This Week

Play Episode Listen Later Jun 29, 2021 22:58


Biz Bites N' More
Episode 111! COVID Delta Variant Rise, Buzzfeed SPAC's (not another please), and Intellia Therapeutics Gene Editing

Biz Bites N' More

Play Episode Listen Later Jun 29, 2021 37:51


Episode 111! The Biz Bites N' More Podcast covers the recent rise in the Delta Variant of COVID, which is troubling to say the least. Buzzfeed is going to SPAC and this is why God left us a long time ago. Finally Intellia Theraputics has a breakthrough in Gene Editing which is like the best news of all time. $ENFA $NTLA #Comedy #Business Blog: bizbitesnmore.com Twitter: @bizbitesnmore Facebook: @bizbitesnmore YouTube: Biz Bites N' More Leave a voice message on anchor or leave a five star review on Apple and we will read it aloud/listen to it on the pod! VM: https://anchor.fm/biz-bites-n-more/message --- Send in a voice message: https://anchor.fm/biz-bites-n-more/message Support this podcast: https://anchor.fm/biz-bites-n-more/support

The Drill Down
Ep. 41: Take-Two Interactive CEO Strauss Zelnick, Facebook, CEL-SCI, Appharvest, Tesla, Intellia Therapeutics

The Drill Down

Play Episode Listen Later Jun 28, 2021 37:24


Take-Two Interactive CEO Strauss Zelnick (TTWO) on how the company makes its blockbuster games. Why CEL-SCI Corporation (CVM) isn't giving up on its latest immunotherapy despite missing its primary endpoint. Appharvest's (APPH) plan to build a network of indoor farms. Intellia Therapeutics (NTLA) leads all CRISPR stocks higher on promising results for gene editing. The Drill Down with Cory Johnson offers a daily look at the business stories behind stocks on the move. Learn more about your ad choices. Visit megaphone.fm/adchoices

TechCheck
Bessemer Venture Partners' Byron Deeter Gives His Take on Tech, Showdown of the $2 Trillion Companies & Crypto Crackdown Hits Binance

TechCheck

Play Episode Listen Later Jun 28, 2021 42:30


Bessemer Venture Partners' Byron Deeter is here this Monday morning to share his take on investing in tech and software as the major averages are at record levels. Then, New York Times Contributor and “Sway” Podcast Host Kara Swisher joins our anchors to discuss the tech war between $2 trillion companies Apple and Microsoft. CNBC's Meg Tirrell also has the story on shares of biotech company Intellia surging after a breakthrough in gene-editing technology. Later, Castle Island Ventures Partner and Coin Metrics Co-Founder Nic Carter joins our anchors to discuss the crypto crackdown as crypto exchange Binance faces a ban in the U.K. Then, CNBC's Julia Boorstin has all the details on the box office making a comeback this past weekend. Plus, we have the story on Tesla recalling 285,000 cars in China due to issues with the vehicles' cruise control system. And don't miss CNBC's Sal Rodriguez speak about his CNBC.com piece highlighting how much control Bytedance actually has over its app TikTok.

The daily tech stock news briefing
06/28/2021, Intellia Therapeutics (NTLA) stock jumped more than 50%

The daily tech stock news briefing

Play Episode Listen Later Jun 28, 2021 0:45


Intellia Therapeutics (NTLA) stock jumped more than 50% after the biotech, along with Regeneron Pharmaceuticals (REGN), reported positive results from its first clinical trial using a new, Nobel Prize-winning Crispr technology to treat Transthyretin Amyloidosis, a rare condition characterized by an abnormal buildup of a protein called amyloid in the body's organs and tissues. Other Crispr-related companies saw gains on Monday as well. Crispr Therapeutics has increased by 6.39 percent, while Beam Therapeutics has increased by 15.98 percent.

Best of US Investors's Podcast
Google, Amazon, Illumina, Crispr, Intellia Will Change Your World

Best of US Investors's Podcast

Play Episode Listen Later Dec 6, 2020 14:33


The pandemic has brought the world's attention to the world's health care, this collided with advancements in microchip technology, artificial intelligence and machine learning.  The resulting change will be manifested in a new way to deliver healthcare and a cure to many of the diseases that kill us.  Here are the companies who are leading the way: Google, Amazon, Illumina, Crispr, Intellia. Welcome back tribe members! Today I'm discussing "Google, Amazon, Illumina, Crispr, Intellia Will Change Your World". If you enjoy this video feel free to SUBSCRIBE! Make sure to follow me on social media for even more coverage of the stock market.Follow Me: Facebook: https://www.facebook.com/BestofUSLLC/Instagram: https://www.instagram.com/bestofusinv...Twitter: https://twitter.com/BestOfUsInvestSubscribe:https://www.youtube.com/channel/UC-hq...We have Up-Graded Our DiscordThis is the new link: https://discord.io/bestofus. It is now organized by topics and will be easier to navigate and communicate.