Podcasts about Alnylam Pharmaceuticals

In this episode of IDEA Collider, we dive into the world of biotech innovation with John Maraganore, former CEO of Alnylam Pharmaceuticals. Recognized as a trailblazer, John transformed RNA interference from a scientific concept into a revolutionary class of medicines, leading the development of five life-changing drugs. Join us as we explore John's journey from a first-generation American with Greek immigrant parents to a key figure in biomedicine. We'll discuss his education at the University of Chicago, his leadership role at Alnylam, drug delivery challenges for RNAi therapeutics, and the importance of strategic pharmaceutical alliances. John also shares his take on the current biotech environment, the impact of policy and regulation, and the promising future of genetic medicines and AI in drug discovery. Chapter Summaries;00:00 Introduction to John Maraganore: Biotech Trailblazer01:11 John's Early Life and Education03:09 Joining Alnylam and the RNAi Revolution04:56 Challenges and Successes at Alnylam06:47 Current Biotech Funding Environment08:17 Global Competition and US Leadership in Biotech10:25 The Importance of In-Person Collaboration12:01 Overcoming Drug Delivery Hurdles15:21 Maintaining an Entrepreneurial Spirit in Biotech17:00 Balancing Science and Business in Decision Making20:27 Strategic Partnerships in Biotech23:36 The Role of Biotech in Global Challenges26:25 Advocating for Policy Changes in Biotech30:41 Mentoring the Next Generation of Biotech Leaders32:35 Future Frontiers in Biotech Innovation36:08 John's Vision for the Future of Biomedicine37:18 Communicating Innovation and LeadershipDon't forget to Like, Share, Subscribe, Rate, and Review! Keep up with John Maraganore;LinkedIn: https://www.linkedin.com/in/john-maraganore/ Follow Mike Rea On;Website: https://www.ideapharma.com/X: https://x.com/ideapharmaLinkedIn: https://www.linkedin.com/in/bigidea/ Listen to more fantastic podcast episodes: https://podcast.ideapharma.com/

We love to hear from our listeners. Send us a message. On today's episode, Tolga Tanguler, EVP and Chief Commercial Officer at Alnylam Pharmaceuticals, talks about commercializing RNAi therapies, how they fit into health insurance reimbursement systems, the company's use of value-based contracts, and building therapeutic franchise "skyscrapers." Tangular also shares his thoughts on commercializing RNAi therapies for larger patient populations, compared with rare disease populations, and reflects on changes in the biopharmaceutical sales and promotion model.This episode is brought to you by Avantor. For more information, visit avantorsciences.comAccess this and hundreds of episodes of the Business of Biotech videocast under the Business of Biotech tab at lifescienceleader.com. Subscribe to our monthly Business of Biotech newsletter. Get in touch with guest and topic suggestions: ben.comer@lifescienceleader.comFind Ben Comer on LinkedIn: https://www.linkedin.com/in/bencomer/

Alnylam Pharmaceuticals, Inc. v. Moderna, Inc.

In this insightful episode of The New CISO, host Steve Moore sits down with Sanju Misra, Chief Information Security Officer (CISO) at Alnylam Pharmaceuticals, to explore the pivotal moments that have shaped her impressive career in cybersecurity leadership. Sanju shares her strategies for navigating career transitions, the importance of aligning with a company's mission, and how to identify the right time to move on from a role.Listeners will gain valuable insights into:How Sanju built her career by embracing challenging projects and maintaining authentic professional relationships.The decision-making processes behind her moves from GE to Praxair, and eventually to Alnylam Pharmaceuticals.The evolution of her leadership style from a technical expert to a business risk executive.Why aligning with a company's culture and mission is crucial for long-term success.Tips for aspiring CISOs on taking initiative, growing their networks, and articulating risk in business terms.Sanju's reflections on imposter syndrome, professional growth, and the rewards of working in a patient-focused organization offer both inspiration and practical advice for leaders at every stage of their careers. Tune in to hear her story and discover actionable strategies for thriving as a modern CISO.0:00 - Introduction and Show Overview1:10 - Sanju Misra's Career Path: From GE to Praxair4:00 - Building Security Programs and Leadership Growth6:30 - Navigating Mergers and the Role of Culture10:00 - Transitioning from Technologist to Business Risk Leader15:50 - Career Advice for Aspiring CISOs22:00 - Key Takeaways: Communication and Leadership EvolutionLinks: LinkedIn

Alnylam Pharmaceuticals chief scientific officer Kevin Fitzgerald on the past, present & future of RNA interference medicines.

In this episode of The New CISO, host Steve Moore is joined by Sanju Misra, CISO and IT Risk Leader at Alnylam Pharmaceuticals. Sanju shares her journey from a college side hustle on a typewriter to becoming a security leader in the pharmaceutical industry. Along the way, she reflects on the importance of taking risks, embracing roles outside of your comfort zone, and the power of transferable skills.Listen to Steve and Sanju discuss career transitions, building diverse teams, and why not checking every box on a job description might be the best career move:00:00 - Meet Sanju06:15 - The Start of a Tech Journey18:30 - Taking the Leap27:00 - Advice for Job Seekers39:45 - Hiring from Within51:15 - Networking and Career GrowthLinks: LinkedIn

For more information regarding this CME/CE activity and to complete the CME/CE requirements and claim credit for this activity, visit:https://www.mycme.com/courses/questions-and-answers-on-amyloid-cardiomyopathy-and-polyneuropathy-9845SummaryIn this activity – which learners can review as a podcast or a webcast - Drs. Chafic Karam and Michelle Kittleson dive into five key questions from recent live-virtual broadcasts about transthyretin amyloidosis (ATTR). They discuss the role of cardiac MRI, biopsy, and genetic testing in diagnosing ATTR cardiomyopathy and polyneuropathy. They also explore treatment options, including silencing therapies and stabilizers, with insights into when patients should be referred for specialized care. The conversation emphasizes the importance of early diagnosis, collaborative care between neurologists and cardiologists, and the expanding therapeutic landscape for ATTR.Learning ObjectivesAt the conclusion of this activity, participants should be better able to:Describe the correct diagnostic algorithm for suspected ATTR, including interpretation of the monoclonal protein screen and indications for biopsy and genetic testing.Initiate appropriate management, including disease-directed therapy and appropriate cardiac care, or referral when indicated.This activity is accredited for CME/CE CreditAssociation of Black Cardiologists, Inc. (ABC) is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.Association of Black Cardiologists, Inc. designates this enduring material for a maximum of 0.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.This activity has been planned and implemented in accordance with the Accreditation Standards of the American Association of Nurse Practitioners® (AANP) through the joint providership of the National Association for Continuing Education (NACE) and ABC. NACE is accredited by the AANP as an approved provider of nurse practitioner continuing education. Provider number 121222. This activity is approved for 0.25 contact hours (which does not include hours of pharmacology).In support of improving patient care, this activity has been planned and implemented by Haymarket Medical Education (HME) and NACE. Haymarket Medical Education 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.This knowledge-based activity JA4008232-9999-24-043-H01-P qualifies for 0.25 contact hours (0.025 CEUs) of continuing pharmacy education credits.For additional information about the accreditation of this program, please contact NACE at info@naceonline.com.Summary of Individual DisclosuresPlease review faculty and planner disclosures here.Disclosure of Commercial SupportThis educational activity is supported by an educational grant from Alnylam Pharmaceuticals, Inc., and an educational grant from AstraZeneca Pharmaceuticals.Please visit http://naceonline.com to engage in more live and on demand CME/CE content.

Marco Fossatelli is country manager BeNeLux van Alnylam Pharmaceuticals.***Volg Leaders in Life Sciences via de website.Volg Leaders in Life Sciences via LinkedIn.Bezoek www.henkjanout.nl voor meer informatie over de host.***Vragen, suggesties of feedback? Graag! Stuur een email naar: henkjan.out@me.com***Vind jij de Leaders in Life Sciences podcast leuk? Dan zouden we het enorm waarderen als je een recensie zou willen achterlaten op Apple Podcasts of een vijf sterren beoordeling zou willen geven op Spotify.Jouw steun betekent veel voor ons, omdat het anderen kan aanmoedigen om de podcast ook te gaan beluisteren. Heel erg bedankt!***Leaders in Life Sciences wordt mede mogelijk gemaakt door Pivot Park, Pedersen & Partners en Johnson & Johnson Innovative Medicine.

This episode is a discussion of the paper entitled, "Catheter Ablation versus Advanced Therapy for Patients with Severe Heart Failure and Ventricular Electrical Storm." https://www.hrsonline.org/education/TheLead https://doi.org/10.1016/j.hrthm.2024.09.045 Host Disclosure(s): W.Sauer: Honoraria/Speaking/Consulting: Biotronik, Biosense Webster, Inc., Abbott, Boston Scientific, Research: Medtronic   Contributor Disclosure(s): R. Kerley: Nothing to disclose. A. Desai: Research: Novartis, Bayer Healthcare Pharmaceuticals, Abbott Medical, AstraZeneca, Honoraria/Speaking/Consulting: Novartis, Abbott, AstraZeneca, Regerneron, Alnylam Pharmaceuticals, Bayer Healthcare Pharmaceutricals, Cytokinetics, AxonTherapies, Avidity Biosciences, Medpace, Merck, New Amsterdam Pharma, Parexel, Roche Diagnostics, GlacoSmithKline, NovoNordisk, Veristat, Verily/Google, Zydus, River2Renal, Membership on Advisory Committees: BioFourmis

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/EZY865. CME credit will be available until October 9, 2025.Stepping Stones on the Path to a Healthier Future for Patients With Primary Hyperoxaluria Type 1: Expert Perspectives on Ensuring Timely Diagnosis and Appropriate Management In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Alnylam Pharmaceuticals, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/EZY865. CME credit will be available until October 9, 2025.Stepping Stones on the Path to a Healthier Future for Patients With Primary Hyperoxaluria Type 1: Expert Perspectives on Ensuring Timely Diagnosis and Appropriate Management In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Alnylam Pharmaceuticals, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/EZY865. CME credit will be available until October 9, 2025.Stepping Stones on the Path to a Healthier Future for Patients With Primary Hyperoxaluria Type 1: Expert Perspectives on Ensuring Timely Diagnosis and Appropriate Management In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Alnylam Pharmaceuticals, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/EZY865. CME credit will be available until October 9, 2025.Stepping Stones on the Path to a Healthier Future for Patients With Primary Hyperoxaluria Type 1: Expert Perspectives on Ensuring Timely Diagnosis and Appropriate Management In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Alnylam Pharmaceuticals, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/EZY865. CME credit will be available until October 9, 2025.Stepping Stones on the Path to a Healthier Future for Patients With Primary Hyperoxaluria Type 1: Expert Perspectives on Ensuring Timely Diagnosis and Appropriate Management In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Alnylam Pharmaceuticals, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/EZY865. CME credit will be available until October 9, 2025.Stepping Stones on the Path to a Healthier Future for Patients With Primary Hyperoxaluria Type 1: Expert Perspectives on Ensuring Timely Diagnosis and Appropriate Management In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Alnylam Pharmaceuticals, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/EZY865. CME credit will be available until October 9, 2025.Stepping Stones on the Path to a Healthier Future for Patients With Primary Hyperoxaluria Type 1: Expert Perspectives on Ensuring Timely Diagnosis and Appropriate Management In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Alnylam Pharmaceuticals, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME information, and to apply for credit, please visit us at PeerView.com/EZY865. CME credit will be available until October 9, 2025.Stepping Stones on the Path to a Healthier Future for Patients With Primary Hyperoxaluria Type 1: Expert Perspectives on Ensuring Timely Diagnosis and Appropriate Management In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from Alnylam Pharmaceuticals, Inc.Disclosure information is available at the beginning of the video presentation.

In this episode, Ayesha spoke with Kirk Brown, PhD, Vice President of Research at Alnylam Pharmaceuticals. Alnylam is focused on developing RNAi medicines to transform the way diseases like cardiovascular and neurological diseases are treated. Dr. Brown's preclinical work combining stable siRNA designs with alternative conjugation strategies has enabled potent, long-lasting silencing across the CNS following a single intrathecal administration.  In addition to driving RNAi platform innovations, Dr. Brown leads a team of CNS target biologists at Alnylam and serves as the research lead for ALN-APP, the first clinical CNS RNAi program. In this episode, Dr. Brown discusses the promise and evolving landscape of RNAi therapeutics across various therapeutic areas and Alnylam's approach to developing innovative RNAi medicines. For more life science and medical device content, visit the Xtalks Vitals homepage. https://xtalks.com/vitals/ Follow Us on Social Media Twitter: https://twitter.com/Xtalks Instagram: https://www.instagram.com/xtalks/ Facebook: https://www.facebook.com/Xtalks.Webinars/ LinkedIn: https://www.linkedin.com/company/xtalks-webconferences YouTube: https://www.youtube.com/c/XtalksWebinars/featured

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Medtronic's chief medical officer of acute care and monitoring has departed for a new role. The FDA has finalized voluntary malfunction summary reporting guidance. Penumbra is laying off 71 people after axing its virtual reality division. Qiagen and AstraZeneca have expanded their companion diagnostic pact. The Association for Molecular Pathology is suing to block an FDA lab test rule. Layoffs in the medical device industry continue to be a trend. Industry news includes the launch of the first over-the-counter glucose monitor, unexpected partnerships in diabetes tech, and pushback on FDA regulation of lab-developed tests.The text discusses various updates in the biopharma industry, including Alnylam's heart drug data sparking debate, Novo building a heart failure case for semaglutide, and a slump in cell and gene therapy investment. The industry is shifting towards more patient-centric commercialization strategies, with companies like Pfizer and Lilly breaking into the direct-to-consumer market. The summer has been a mixed bag for biotech, with market fluctuations and limited IPOs and acquisitions. Alnylam's full study data on its heart drug vutrisiran showed benefits but also raised skepticism. Novo's analysis showed semaglutide's effectiveness in reducing heart failure risk. Additionally, PBM executives are facing fines for alleged perjury in a house hearing defending their business practices.The text discusses the pricing and sales of gene therapies, particularly focusing on Novartis' Zolgensma, which has been the only gene therapy to cross the blockbuster threshold with sales of $1.2 billion in 2021 and $1.4 billion in 2022. While high price tags have not always resulted in significant returns for pharmaceutical companies with gene therapies, Zolgensma has been an exception. Other companies have faced challenges with new gene therapies, such as Biomarin Pharmaceuticals, which recently announced layoffs due to dismal sales of its hemophilia A gene therapy Roctavian.Alnylam Pharmaceuticals has released detailed data on their drug Vutrisiran, showing its benefits in treating a progressive and fatal heart condition known as ATTR cardiomyopathy. The results were published in the New England Journal of Medicine. While the data confirms the therapy's benefit, there are still questions about how exactly Vutrisiran should be used in treating the disease. The study is expected to continue to fuel debate among doctors.The text discusses the recent slump in investment in cell and gene therapy, with fewer venture funding rounds closed by developers in the first six months of 2024. Two companies, Biomarin and Tome Biosciences, are cutting jobs, with over 200 and over 100 layoffs respectively. Biomarin has made changes to its executive team and drug pipeline to refocus resources. Additionally, Bayer has partnered with RNA drugmaker Nextrna Therapeutics to develop new cancer therapies. The text also highlights the challenges faced in clinical trials, with nearly 80% failing to meet enrollment goals and schedules. The importance of understanding social determinants of health for research is emphasized.Biopharma Dive's Gene Therapy Weekly provides news and insights on gene therapy for biopharma leaders.

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, Sharon Cohen, MD, a behavioral neurologist, sat down to discuss positive data from an ongoing phase 1 study of mivelsiran (Alnylam Pharmaceuticals), the first investigational RNA interference therapeutic targeting amyloid precursor protein for Alzheimer disease (AD). Cohen, who also serves as the medical director of the Toronto Memory Program at the University of Toronto, discussed the potential of RNA therapeutics for treating AD, the unique mechanism of action of mivelsiran, and some of the early promising safety, efficacy, and pharmacokinetic data observed in the phase 1 trial. In addition, Cohen touched upon the idea of how RNA therapeutics could be used in combination with previously approved novel treatments and the benefits mivelsiran brings with no observed amyloid-related imaging abnormalities. Furthermore, the discussion covered some of the potential of this investigational agent, what to expect in the multi-dose part B of the study, and an additional phase 2 study in cerebral amyloid angiopathy.  Looking for more Alzheimer disease/dementia discussion? Check out the NeurologyLive® Alzheimer disease/dementia clinical focus page. Episode Breakdown: 1:15 – Mechanism of action of mivelsiran and how it can be potentially beneficial in AD 4:15 – Growing knowledge of RNA therapeutics and their impact on neurologic conditions 7:00 – Neurology News Minute 10:10 – Phase 1 study data, including efficacy, safety, biomarker, and pharmacokinetic results 19:20 – How mivelsiran fits with other approved therapies for AD; future goals and directions of the drug The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: FDA Clears Indapta Therapeutics' IND for Cell Therapy IDP-023 in Progressive Multiple Sclerosis Muscle-Targeting Therapy Apitegromab Effective in Spinal Muscular Atrophy Over 4 Year Period FDA Approves Medtronic's Deep Brain Stimulation Technology for Asleep Capabilities Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

Discover the cutting-edge world of RNA interference (RNAi) therapeutics with Dr. Akshay Vaishnaw, Chief Innovation Officer at Alnylam Pharmaceuticals. In this episode, Dr. Vaishnaw sheds light on the groundbreaking concept of RNAi therapeutics, offering a powerful new approach to treating a wide range of diseases. Learn how Alnylam Pharmaceuticals is revolutionizing healthcare with its unwavering commitment to innovation, despite facing numerous setbacks and challenges along the way.

Scott Wapner and the Investment Committee discuss whether the rally can last as we approach the second half of the year. The calls of the day include Chipotle, UnitedHealth, and Nike. The experts debate the energy trade. The chart of the day is Alnylam Pharmaceuticals.Investment Committee Disclosures

This Day in Legal History: James McCord ArrestedOn June 17, 1972, James McCord, security director for President Richard Nixon's re-election committee, and four Cuban-Americans were arrested for breaking into the Democratic National Committee Headquarters at the Watergate complex in Washington, D.C. This event marked the beginning of the Watergate scandal, a major political scandal in the United States that ultimately led to President Nixon's resignation. The burglars were caught wiretapping phones and stealing documents, intending to gather information to sabotage Nixon's political opponents. As investigations unfolded, it was revealed that the break-in was part of a broader campaign of political espionage and sabotage conducted by the Nixon administration. The scandal exposed widespread abuse of power, including illegal wiretapping, break-ins, and attempts to cover up these activities. Journalists Bob Woodward and Carl Bernstein of The Washington Post played a crucial role in uncovering the details of the scandal, leading to increased public scrutiny and pressure on the administration.The investigation led to the indictment of several Nixon administration officials and the creation of the Senate Watergate Committee. The most significant outcome was the discovery of the existence of secret tapes of conversations in the Oval Office, which revealed President Nixon's involvement in the cover-up. Faced with the likelihood of impeachment, Nixon became the first U.S. president to resign from office on August 8, 1974. The Watergate scandal had a lasting impact on American politics, leading to increased transparency and reforms aimed at preventing such abuses of power in the future.The rare disease community is advocating for changes to the Inflation Reduction Act's drug price-setting scheme, which is causing drugmakers and investors to reconsider developing drugs for small patient populations. Companies like Pfizer, Alnylam Pharmaceuticals, Eli Lilly, and Protagonist Therapeutics are altering their research strategies due to concerns over recouping costs under the Medicare Drug Price Negotiation Program. This program exempts orphan drugs with a single FDA-approved indication from price negotiations, but those with multiple indications might not qualify for the exclusion, even if they are not yet approved for additional conditions.Opponents are seeking legislative and judicial changes to amend the Inflation Reduction Act, as companies and investors shift their focus away from rare disease and small-molecule drugs. Jamie Sullivan of the EveryLife Foundation for Rare Diseases emphasized the importance of achieving technical fixes to support innovation. Recent legislative efforts aim to protect the development of these drugs, and companies like AstraZeneca are challenging the provisions in court. The Inflation Reduction Act has made the capital environment more challenging for rare disease drugs, according to John Stanford of the Incubate Coalition.Patient groups and some manufacturers argue that the exemption reduces incentives provided by the Orphan Drug Act, which has historically promoted the research and development of rare disease medicines. These drugs often launch with a single indication but can later be approved for additional uses. Concerns are rising that the current policies may hinder further research into rare diseases. Despite industry concerns, some groups argue that drug companies still have substantial resources for robust R&D and can profit from expanding indications for orphan drugs. A CMS spokesperson stated that the negotiation program aligns with the law and won't harm long-term innovation.US Drug Negotiations Plan Shifts Focus for Rare Disease ProgramsAlex Jones' personal bankruptcy has been converted to a Chapter 7 liquidation, meaning a trustee will now manage how he pays the $1.5 billion in defamation judgments against him. Jones was unable to reach an agreement with the families of the Sandy Hook Elementary School shooting victims, whom he defamed by claiming the 2012 massacre was a hoax. US Bankruptcy Judge Christopher Lopez ruled that Jones' case does not qualify for exceptions to prevent its conversion to Chapter 7, despite Jones' objections.During the same hearing, Judge Lopez dismissed the bankruptcy case of Infowars' parent company, Free Speech Systems LLC, allowing the Sandy Hook families to pursue their claims in state court. The judge clarified that there was never a request to shut down Infowars itself. Lopez expressed the difficulty of the case, acknowledging its connection to the tragic 2012 shooting.Jones' defamation debts were deemed non-dischargeable in bankruptcy due to the intentional and malicious nature of his actions. Following state court verdicts, Free Speech Systems filed for Chapter 11 bankruptcy in July 2022, and Jones filed for personal bankruptcy in December 2022. Efforts to reach a consensual bankruptcy plan failed, prompting the recent conversion to Chapter 7.Jones and the Sandy Hook families had recently agreed on the need to convert his case to Chapter 7, but the Free Speech Systems case remained contentious. Ultimately, the court decided that dismissing the Free Speech case was in the best interest of creditors. Despite potential liquidation, Jones may start a new broadcast, according to attorneys for the Sandy Hook families. This case is being managed under Alexander E. Jones, Bankr. S.D. Tex., No. 22-33553, as of June 14, 2024.Alex Jones Loses Financial Control as Trustee Takes Over (4)Alex Jones' assets to be liquidated as his company exits bankruptcy | ReutersAt a star-studded fundraiser in Los Angeles on June 15, President Joe Biden criticized the U.S. Supreme Court as being "out of kilter," emphasizing that it has never been as out of step as it is today. The event, which featured former President Barack Obama and Hollywood celebrities like George Clooney and Julia Roberts, raised over $30 million for Biden's campaign. Biden highlighted the court's conservative leanings, particularly criticizing Justice Clarence Thomas's remarks on reconsidering issues like in vitro fertilization and contraception. He also warned that if Donald Trump wins the 2024 election, he could appoint two more justices, which Biden described as alarming.The fundraiser was framed by a video montage contrasting Biden's record with that of Trump, drawing cheers from the audience. Biden noted that the Supreme Court, now dominated by conservatives appointed by Trump, has made decisions restricting affirmative action, gay rights, gun control, and environmental regulations. Obama reiterated the importance of elections in determining the court's power, linking the current situation to Trump's 2016 victory.The event marked the largest Democratic fundraiser in history, surpassing a previous record set in New York City. Other celebrities present included Jack Black, Jason Bateman, and Kathryn Hahn. Despite Biden's low approval ratings and concerns about his age, the fundraiser aimed to showcase the campaign's strength and momentum. Biden and Trump are currently tied in national polls, with Trump leading in battleground states. Both candidates have raised significant funds in California, demonstrating the state's financial influence on their campaigns.Biden slams Supreme Court at $30 million fundraiser with Obama, Clooney, Julia Roberts | Reuters This is a public episode. If you'd like to discuss this with other subscribers or get access to bonus episodes, visit www.minimumcomp.com/subscribe

Tercera hora de Visión Global en Radio Intereconomía que dedicamos a nuestro consultorio de Wall Street en el que nos acompaña David Leyguarda, analista independiente. Con él hablamos de Meta, Intel, Imperial Petroleum, Alnylam Pharmaceuticals, Walt Disney, Walmart, Verizon, Zebra Technologies, Nike, Bolt, Warrior Met Coal, Ivanhoe Mines, NIO, Porsche Holding y Boeing. Después, último repaso a los mercados el los últimos instantes de cotización y actualizamos la información en titulares. Terminamos con el último repaso a los mercados en el que nos acompaña José Luis Herrera, analista independiente. Con él hacemos balance de la jornada y previsiones de lo que queda de semana en unas jornadas marcadas por las caídas en Wall Street con los inversores pendientes de resultados empresariales.

William H. Sauer, MD, FHRS, CCDS, of Brigham and Women's Hospital is joined by guests Akshay Suvas Desai, MD, MPH of Brigham and Women's Hospital, and Sunil Kapur, MD of Brigham and Women`s Hospital to discuss how the Resynchronization–Defibrillation for Ambulatory Heart Failure Trial (RAFT) showed a greater benefit with respect to mortality at 5 years among patients who received cardiac-resynchronization therapy (CRT) than among those who received implantable cardioverter–defibrillators (ICDs). However, the effect of CRT on long-term survival is not known. We randomly assigned patients with New York Heart Association (NYHA) class II or III heart failure, a left ventricular ejection fraction of 30% or less, and an intrinsic QRS duration of 120 msec or more (or a paced QRS duration of 200 msec or more) to receive either an ICD alone or a CRT defibrillator (CRT-D). We assessed long-term outcomes among patients at the eight highest-enrolling participating sites. The primary outcome was death from any cause; the secondary outcome was a composite of death from any cause, heart transplantation, or implantation of a ventricular assist device.  https://www.hrsonline.org/education/TheLead https://www.nejm.org/doi/full/10.1056/NEJMoa2304542 Host Disclosure(s): W. Sauer: Honoraria/Speaking/Consulting: Biotronik, Biosense Webster, Inc., Abbott, Boston Scientific; Research: Medtronic Contributor Disclosure(s): A. Suvas Desai: Honoraria/Speaking/Consulting: Abbott, Novartis, AstraZeneca, BioFourmis, Regeneron, Alnylam Pharmaceuticals, Bayer Healthcare Pharmaceuticals, Cytokinetics, AxonTherapies, Avidity Biosciences, Medpace, Merck, New Amsterdam Pharma, Parexel, Roche Diagnostics, GlaxoSmithKline, NovoNordisk, Veristat, Verily/Google, Zydus, River2Renal, Research: Bayer Healthcare Pharmaceuticals, Abbott Medical, AstraZeneca, Novartis  S. Kapur: Honoraria/Speaking/Consulting: Medtronic, Novartis, Abbott, Biotronik This episode has .25 ACE credits associated with it. If you want credit for listening to this episode, please visit the episode page on HRS365 https://www.heartrhythm365.org/URL/TheLeadEpisode53

Tercera hora de Visión Global que dedicamos a nuestro consultorio de Wall Street en el que nos acompaña Miguel Ángel Martínez, gestor de mercados en Tiempo de Bolsa. Con él analizamos compañías como AMD, Tesla, The Honest Company, Alnylam Pharmaceuticals, Sea Limited, ZScaler, Berkshire Hathaway, Quantumscape, Alphabet, SoundHound, Palo Alto, Tesla y Phibro. Después, último repaso a los mercados y actualizamos información en titulares. Terminamos la jornada con el análisis de mercados que nos trae Borja de Castro, analista de Banco Big. Con él analizamos una semana marcada por los resultados empresariales y por datos macros que iremos conociendo a lo largo de la semana. El foco especialmente está puesto en el jueves que será cuando se publique el dato de deflactor PCE, el principal indicador que tiene en cuenta la Reserva Federal a la hora de tomar sus decisiones en materia de tipos de interés.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA information, and to apply for credit, please visit us at PeerView.com/SPK865. CME/MOC/AAPA credit will be available until December 12, 2024.Addressing Unmet Needs in Anticoagulation for Thrombosis: Next-Generation Strategies for Improving Care In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from the Bristol Myers Squibb and Janssen Alliance.Disclosure PolicyAll relevant conflicts of interest have been mitigated prior to the commencement of the activity.Faculty/Planner DisclosuresCo-Chair/PlannerRoxana Mehran, MD, has a financial interest/relationship or affiliation in the form of:Grant/Research Support from Abbott; ABIOMED; Affluent Medical; Alleviant Medical; Amgen Inc.; AM-Pharma B.V.; Arena Pharmaceuticals, Inc.; AstraZeneca; AtriCure Inc.; Biosensors; BIOTRONIK SE & Co KG; Boston Scientific Corporation; Bristol Myers Squibb; CardiaWave; Celonova Biosciences Inc.; Chiesi USA, Inc.; Concept Medical; Cytosorbents Europe GmbH; Daiichi Sankyo Inc.; Duke; Element Science, Inc.; Faraday Pharmaceuticals; Humacyte, Inc.; Idorsia Pharmaceuticals Ltd; Janssen Pharmaceuticals, Inc.; Koninklijke Philips N.V.; Magenta Therapeutics; M.A. Med Alliance SA; MediaSphere Medical LLC; Medtelligence; Medtronic; MJH Life Sciences; Novartis Pharmaceuticals Corporation; OrbusNeich Medical Company Group Holdings Limited; Penumbra, Inc.; PhaseBio Pharmaceuticals, Inc.; Pi-Cardia; PLx Pharma Inc.; Protembis GmbH; RenalPro; RM Global Partners; Shockwave Medical Inc.; Vivasure Medical Limited; and ZOLL Medical Corporation.Honoraria from ACC (BOT Member, SC Member CTR Program) and JAMA Cardiology (Associate Editor).Stock Shareholder in Applied Therapeutics and Elixir Therapeutics, LLC.Other Financial or Material Support in the form of personal fees from Affluent Medical; Cardiovascular Research Foundation; Daiichi Sankyo Brasil; ER Squibb & Sons LLC; Esperion Therapeutics, Inc.; Europa Group/Boston Scientific Corporation; Gaffney Events Educational Trust; Ionis Pharmaceuticals; IQVIA Inc; J-CalC; McVeigh Global Meetings and Events; Novartis Pharmaceuticals Corporation; Novo Nordisk Inc.; Oversome; Primer Healthcare of New Jersey; Radcliffe Institute for Advanced Study; SL Solutions LLC; TARSUS Cardiology; Vectura Group Ltd; and Vox Media, LLC.Co-Chair/PlannerJeffrey Weitz, MD, FRCPC, FRSC, FACP, has a financial interest/relationship or affiliation in the form of:Consultant and/or Advisor for Alnylam Pharmaceuticals, Inc.; Bayer AG; Boehringer Ingelheim Pharmaceuticals, Inc.; Bristol Myers Squibb; Daiichi Sankyo Co., Ltd.; Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Honoraria from Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Planning Committee and Reviewer DisclosuresPlanners, independent reviewers, and staff of PVI, PeerView Institute for Medical Education, do not have any relevant financial relationships related to this CE activity unless listed below.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA information, and to apply for credit, please visit us at PeerView.com/SPK865. CME/MOC/AAPA credit will be available until December 12, 2024.Addressing Unmet Needs in Anticoagulation for Thrombosis: Next-Generation Strategies for Improving Care In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from the Bristol Myers Squibb and Janssen Alliance.Disclosure PolicyAll relevant conflicts of interest have been mitigated prior to the commencement of the activity.Faculty/Planner DisclosuresCo-Chair/PlannerRoxana Mehran, MD, has a financial interest/relationship or affiliation in the form of:Grant/Research Support from Abbott; ABIOMED; Affluent Medical; Alleviant Medical; Amgen Inc.; AM-Pharma B.V.; Arena Pharmaceuticals, Inc.; AstraZeneca; AtriCure Inc.; Biosensors; BIOTRONIK SE & Co KG; Boston Scientific Corporation; Bristol Myers Squibb; CardiaWave; Celonova Biosciences Inc.; Chiesi USA, Inc.; Concept Medical; Cytosorbents Europe GmbH; Daiichi Sankyo Inc.; Duke; Element Science, Inc.; Faraday Pharmaceuticals; Humacyte, Inc.; Idorsia Pharmaceuticals Ltd; Janssen Pharmaceuticals, Inc.; Koninklijke Philips N.V.; Magenta Therapeutics; M.A. Med Alliance SA; MediaSphere Medical LLC; Medtelligence; Medtronic; MJH Life Sciences; Novartis Pharmaceuticals Corporation; OrbusNeich Medical Company Group Holdings Limited; Penumbra, Inc.; PhaseBio Pharmaceuticals, Inc.; Pi-Cardia; PLx Pharma Inc.; Protembis GmbH; RenalPro; RM Global Partners; Shockwave Medical Inc.; Vivasure Medical Limited; and ZOLL Medical Corporation.Honoraria from ACC (BOT Member, SC Member CTR Program) and JAMA Cardiology (Associate Editor).Stock Shareholder in Applied Therapeutics and Elixir Therapeutics, LLC.Other Financial or Material Support in the form of personal fees from Affluent Medical; Cardiovascular Research Foundation; Daiichi Sankyo Brasil; ER Squibb & Sons LLC; Esperion Therapeutics, Inc.; Europa Group/Boston Scientific Corporation; Gaffney Events Educational Trust; Ionis Pharmaceuticals; IQVIA Inc; J-CalC; McVeigh Global Meetings and Events; Novartis Pharmaceuticals Corporation; Novo Nordisk Inc.; Oversome; Primer Healthcare of New Jersey; Radcliffe Institute for Advanced Study; SL Solutions LLC; TARSUS Cardiology; Vectura Group Ltd; and Vox Media, LLC.Co-Chair/PlannerJeffrey Weitz, MD, FRCPC, FRSC, FACP, has a financial interest/relationship or affiliation in the form of:Consultant and/or Advisor for Alnylam Pharmaceuticals, Inc.; Bayer AG; Boehringer Ingelheim Pharmaceuticals, Inc.; Bristol Myers Squibb; Daiichi Sankyo Co., Ltd.; Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Honoraria from Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Planning Committee and Reviewer DisclosuresPlanners, independent reviewers, and staff of PVI, PeerView Institute for Medical Education, do not have any relevant financial relationships related to this CE activity unless listed below.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA information, and to apply for credit, please visit us at PeerView.com/SPK865. CME/MOC/AAPA credit will be available until December 12, 2024.Addressing Unmet Needs in Anticoagulation for Thrombosis: Next-Generation Strategies for Improving Care In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from the Bristol Myers Squibb and Janssen Alliance.Disclosure PolicyAll relevant conflicts of interest have been mitigated prior to the commencement of the activity.Faculty/Planner DisclosuresCo-Chair/PlannerRoxana Mehran, MD, has a financial interest/relationship or affiliation in the form of:Grant/Research Support from Abbott; ABIOMED; Affluent Medical; Alleviant Medical; Amgen Inc.; AM-Pharma B.V.; Arena Pharmaceuticals, Inc.; AstraZeneca; AtriCure Inc.; Biosensors; BIOTRONIK SE & Co KG; Boston Scientific Corporation; Bristol Myers Squibb; CardiaWave; Celonova Biosciences Inc.; Chiesi USA, Inc.; Concept Medical; Cytosorbents Europe GmbH; Daiichi Sankyo Inc.; Duke; Element Science, Inc.; Faraday Pharmaceuticals; Humacyte, Inc.; Idorsia Pharmaceuticals Ltd; Janssen Pharmaceuticals, Inc.; Koninklijke Philips N.V.; Magenta Therapeutics; M.A. Med Alliance SA; MediaSphere Medical LLC; Medtelligence; Medtronic; MJH Life Sciences; Novartis Pharmaceuticals Corporation; OrbusNeich Medical Company Group Holdings Limited; Penumbra, Inc.; PhaseBio Pharmaceuticals, Inc.; Pi-Cardia; PLx Pharma Inc.; Protembis GmbH; RenalPro; RM Global Partners; Shockwave Medical Inc.; Vivasure Medical Limited; and ZOLL Medical Corporation.Honoraria from ACC (BOT Member, SC Member CTR Program) and JAMA Cardiology (Associate Editor).Stock Shareholder in Applied Therapeutics and Elixir Therapeutics, LLC.Other Financial or Material Support in the form of personal fees from Affluent Medical; Cardiovascular Research Foundation; Daiichi Sankyo Brasil; ER Squibb & Sons LLC; Esperion Therapeutics, Inc.; Europa Group/Boston Scientific Corporation; Gaffney Events Educational Trust; Ionis Pharmaceuticals; IQVIA Inc; J-CalC; McVeigh Global Meetings and Events; Novartis Pharmaceuticals Corporation; Novo Nordisk Inc.; Oversome; Primer Healthcare of New Jersey; Radcliffe Institute for Advanced Study; SL Solutions LLC; TARSUS Cardiology; Vectura Group Ltd; and Vox Media, LLC.Co-Chair/PlannerJeffrey Weitz, MD, FRCPC, FRSC, FACP, has a financial interest/relationship or affiliation in the form of:Consultant and/or Advisor for Alnylam Pharmaceuticals, Inc.; Bayer AG; Boehringer Ingelheim Pharmaceuticals, Inc.; Bristol Myers Squibb; Daiichi Sankyo Co., Ltd.; Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Honoraria from Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Planning Committee and Reviewer DisclosuresPlanners, independent reviewers, and staff of PVI, PeerView Institute for Medical Education, do not have any relevant financial relationships related to this CE activity unless listed below.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA information, and to apply for credit, please visit us at PeerView.com/SPK865. CME/MOC/AAPA credit will be available until December 12, 2024.Addressing Unmet Needs in Anticoagulation for Thrombosis: Next-Generation Strategies for Improving Care In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from the Bristol Myers Squibb and Janssen Alliance.Disclosure PolicyAll relevant conflicts of interest have been mitigated prior to the commencement of the activity.Faculty/Planner DisclosuresCo-Chair/PlannerRoxana Mehran, MD, has a financial interest/relationship or affiliation in the form of:Grant/Research Support from Abbott; ABIOMED; Affluent Medical; Alleviant Medical; Amgen Inc.; AM-Pharma B.V.; Arena Pharmaceuticals, Inc.; AstraZeneca; AtriCure Inc.; Biosensors; BIOTRONIK SE & Co KG; Boston Scientific Corporation; Bristol Myers Squibb; CardiaWave; Celonova Biosciences Inc.; Chiesi USA, Inc.; Concept Medical; Cytosorbents Europe GmbH; Daiichi Sankyo Inc.; Duke; Element Science, Inc.; Faraday Pharmaceuticals; Humacyte, Inc.; Idorsia Pharmaceuticals Ltd; Janssen Pharmaceuticals, Inc.; Koninklijke Philips N.V.; Magenta Therapeutics; M.A. Med Alliance SA; MediaSphere Medical LLC; Medtelligence; Medtronic; MJH Life Sciences; Novartis Pharmaceuticals Corporation; OrbusNeich Medical Company Group Holdings Limited; Penumbra, Inc.; PhaseBio Pharmaceuticals, Inc.; Pi-Cardia; PLx Pharma Inc.; Protembis GmbH; RenalPro; RM Global Partners; Shockwave Medical Inc.; Vivasure Medical Limited; and ZOLL Medical Corporation.Honoraria from ACC (BOT Member, SC Member CTR Program) and JAMA Cardiology (Associate Editor).Stock Shareholder in Applied Therapeutics and Elixir Therapeutics, LLC.Other Financial or Material Support in the form of personal fees from Affluent Medical; Cardiovascular Research Foundation; Daiichi Sankyo Brasil; ER Squibb & Sons LLC; Esperion Therapeutics, Inc.; Europa Group/Boston Scientific Corporation; Gaffney Events Educational Trust; Ionis Pharmaceuticals; IQVIA Inc; J-CalC; McVeigh Global Meetings and Events; Novartis Pharmaceuticals Corporation; Novo Nordisk Inc.; Oversome; Primer Healthcare of New Jersey; Radcliffe Institute for Advanced Study; SL Solutions LLC; TARSUS Cardiology; Vectura Group Ltd; and Vox Media, LLC.Co-Chair/PlannerJeffrey Weitz, MD, FRCPC, FRSC, FACP, has a financial interest/relationship or affiliation in the form of:Consultant and/or Advisor for Alnylam Pharmaceuticals, Inc.; Bayer AG; Boehringer Ingelheim Pharmaceuticals, Inc.; Bristol Myers Squibb; Daiichi Sankyo Co., Ltd.; Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Honoraria from Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Planning Committee and Reviewer DisclosuresPlanners, independent reviewers, and staff of PVI, PeerView Institute for Medical Education, do not have any relevant financial relationships related to this CE activity unless listed below.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA information, and to apply for credit, please visit us at PeerView.com/SPK865. CME/MOC/AAPA credit will be available until December 12, 2024.Addressing Unmet Needs in Anticoagulation for Thrombosis: Next-Generation Strategies for Improving Care In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from the Bristol Myers Squibb and Janssen Alliance.Disclosure PolicyAll relevant conflicts of interest have been mitigated prior to the commencement of the activity.Faculty/Planner DisclosuresCo-Chair/PlannerRoxana Mehran, MD, has a financial interest/relationship or affiliation in the form of:Grant/Research Support from Abbott; ABIOMED; Affluent Medical; Alleviant Medical; Amgen Inc.; AM-Pharma B.V.; Arena Pharmaceuticals, Inc.; AstraZeneca; AtriCure Inc.; Biosensors; BIOTRONIK SE & Co KG; Boston Scientific Corporation; Bristol Myers Squibb; CardiaWave; Celonova Biosciences Inc.; Chiesi USA, Inc.; Concept Medical; Cytosorbents Europe GmbH; Daiichi Sankyo Inc.; Duke; Element Science, Inc.; Faraday Pharmaceuticals; Humacyte, Inc.; Idorsia Pharmaceuticals Ltd; Janssen Pharmaceuticals, Inc.; Koninklijke Philips N.V.; Magenta Therapeutics; M.A. Med Alliance SA; MediaSphere Medical LLC; Medtelligence; Medtronic; MJH Life Sciences; Novartis Pharmaceuticals Corporation; OrbusNeich Medical Company Group Holdings Limited; Penumbra, Inc.; PhaseBio Pharmaceuticals, Inc.; Pi-Cardia; PLx Pharma Inc.; Protembis GmbH; RenalPro; RM Global Partners; Shockwave Medical Inc.; Vivasure Medical Limited; and ZOLL Medical Corporation.Honoraria from ACC (BOT Member, SC Member CTR Program) and JAMA Cardiology (Associate Editor).Stock Shareholder in Applied Therapeutics and Elixir Therapeutics, LLC.Other Financial or Material Support in the form of personal fees from Affluent Medical; Cardiovascular Research Foundation; Daiichi Sankyo Brasil; ER Squibb & Sons LLC; Esperion Therapeutics, Inc.; Europa Group/Boston Scientific Corporation; Gaffney Events Educational Trust; Ionis Pharmaceuticals; IQVIA Inc; J-CalC; McVeigh Global Meetings and Events; Novartis Pharmaceuticals Corporation; Novo Nordisk Inc.; Oversome; Primer Healthcare of New Jersey; Radcliffe Institute for Advanced Study; SL Solutions LLC; TARSUS Cardiology; Vectura Group Ltd; and Vox Media, LLC.Co-Chair/PlannerJeffrey Weitz, MD, FRCPC, FRSC, FACP, has a financial interest/relationship or affiliation in the form of:Consultant and/or Advisor for Alnylam Pharmaceuticals, Inc.; Bayer AG; Boehringer Ingelheim Pharmaceuticals, Inc.; Bristol Myers Squibb; Daiichi Sankyo Co., Ltd.; Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Honoraria from Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Planning Committee and Reviewer DisclosuresPlanners, independent reviewers, and staff of PVI, PeerView Institute for Medical Education, do not have any relevant financial relationships related to this CE activity unless listed below.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA information, and to apply for credit, please visit us at PeerView.com/SPK865. CME/MOC/AAPA credit will be available until December 12, 2024.Addressing Unmet Needs in Anticoagulation for Thrombosis: Next-Generation Strategies for Improving Care In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from the Bristol Myers Squibb and Janssen Alliance.Disclosure PolicyAll relevant conflicts of interest have been mitigated prior to the commencement of the activity.Faculty/Planner DisclosuresCo-Chair/PlannerRoxana Mehran, MD, has a financial interest/relationship or affiliation in the form of:Grant/Research Support from Abbott; ABIOMED; Affluent Medical; Alleviant Medical; Amgen Inc.; AM-Pharma B.V.; Arena Pharmaceuticals, Inc.; AstraZeneca; AtriCure Inc.; Biosensors; BIOTRONIK SE & Co KG; Boston Scientific Corporation; Bristol Myers Squibb; CardiaWave; Celonova Biosciences Inc.; Chiesi USA, Inc.; Concept Medical; Cytosorbents Europe GmbH; Daiichi Sankyo Inc.; Duke; Element Science, Inc.; Faraday Pharmaceuticals; Humacyte, Inc.; Idorsia Pharmaceuticals Ltd; Janssen Pharmaceuticals, Inc.; Koninklijke Philips N.V.; Magenta Therapeutics; M.A. Med Alliance SA; MediaSphere Medical LLC; Medtelligence; Medtronic; MJH Life Sciences; Novartis Pharmaceuticals Corporation; OrbusNeich Medical Company Group Holdings Limited; Penumbra, Inc.; PhaseBio Pharmaceuticals, Inc.; Pi-Cardia; PLx Pharma Inc.; Protembis GmbH; RenalPro; RM Global Partners; Shockwave Medical Inc.; Vivasure Medical Limited; and ZOLL Medical Corporation.Honoraria from ACC (BOT Member, SC Member CTR Program) and JAMA Cardiology (Associate Editor).Stock Shareholder in Applied Therapeutics and Elixir Therapeutics, LLC.Other Financial or Material Support in the form of personal fees from Affluent Medical; Cardiovascular Research Foundation; Daiichi Sankyo Brasil; ER Squibb & Sons LLC; Esperion Therapeutics, Inc.; Europa Group/Boston Scientific Corporation; Gaffney Events Educational Trust; Ionis Pharmaceuticals; IQVIA Inc; J-CalC; McVeigh Global Meetings and Events; Novartis Pharmaceuticals Corporation; Novo Nordisk Inc.; Oversome; Primer Healthcare of New Jersey; Radcliffe Institute for Advanced Study; SL Solutions LLC; TARSUS Cardiology; Vectura Group Ltd; and Vox Media, LLC.Co-Chair/PlannerJeffrey Weitz, MD, FRCPC, FRSC, FACP, has a financial interest/relationship or affiliation in the form of:Consultant and/or Advisor for Alnylam Pharmaceuticals, Inc.; Bayer AG; Boehringer Ingelheim Pharmaceuticals, Inc.; Bristol Myers Squibb; Daiichi Sankyo Co., Ltd.; Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Honoraria from Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Planning Committee and Reviewer DisclosuresPlanners, independent reviewers, and staff of PVI, PeerView Institute for Medical Education, do not have any relevant financial relationships related to this CE activity unless listed below.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA information, and to apply for credit, please visit us at PeerView.com/SPK865. CME/MOC/AAPA credit will be available until December 12, 2024.Addressing Unmet Needs in Anticoagulation for Thrombosis: Next-Generation Strategies for Improving Care In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from the Bristol Myers Squibb and Janssen Alliance.Disclosure PolicyAll relevant conflicts of interest have been mitigated prior to the commencement of the activity.Faculty/Planner DisclosuresCo-Chair/PlannerRoxana Mehran, MD, has a financial interest/relationship or affiliation in the form of:Grant/Research Support from Abbott; ABIOMED; Affluent Medical; Alleviant Medical; Amgen Inc.; AM-Pharma B.V.; Arena Pharmaceuticals, Inc.; AstraZeneca; AtriCure Inc.; Biosensors; BIOTRONIK SE & Co KG; Boston Scientific Corporation; Bristol Myers Squibb; CardiaWave; Celonova Biosciences Inc.; Chiesi USA, Inc.; Concept Medical; Cytosorbents Europe GmbH; Daiichi Sankyo Inc.; Duke; Element Science, Inc.; Faraday Pharmaceuticals; Humacyte, Inc.; Idorsia Pharmaceuticals Ltd; Janssen Pharmaceuticals, Inc.; Koninklijke Philips N.V.; Magenta Therapeutics; M.A. Med Alliance SA; MediaSphere Medical LLC; Medtelligence; Medtronic; MJH Life Sciences; Novartis Pharmaceuticals Corporation; OrbusNeich Medical Company Group Holdings Limited; Penumbra, Inc.; PhaseBio Pharmaceuticals, Inc.; Pi-Cardia; PLx Pharma Inc.; Protembis GmbH; RenalPro; RM Global Partners; Shockwave Medical Inc.; Vivasure Medical Limited; and ZOLL Medical Corporation.Honoraria from ACC (BOT Member, SC Member CTR Program) and JAMA Cardiology (Associate Editor).Stock Shareholder in Applied Therapeutics and Elixir Therapeutics, LLC.Other Financial or Material Support in the form of personal fees from Affluent Medical; Cardiovascular Research Foundation; Daiichi Sankyo Brasil; ER Squibb & Sons LLC; Esperion Therapeutics, Inc.; Europa Group/Boston Scientific Corporation; Gaffney Events Educational Trust; Ionis Pharmaceuticals; IQVIA Inc; J-CalC; McVeigh Global Meetings and Events; Novartis Pharmaceuticals Corporation; Novo Nordisk Inc.; Oversome; Primer Healthcare of New Jersey; Radcliffe Institute for Advanced Study; SL Solutions LLC; TARSUS Cardiology; Vectura Group Ltd; and Vox Media, LLC.Co-Chair/PlannerJeffrey Weitz, MD, FRCPC, FRSC, FACP, has a financial interest/relationship or affiliation in the form of:Consultant and/or Advisor for Alnylam Pharmaceuticals, Inc.; Bayer AG; Boehringer Ingelheim Pharmaceuticals, Inc.; Bristol Myers Squibb; Daiichi Sankyo Co., Ltd.; Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Honoraria from Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Planning Committee and Reviewer DisclosuresPlanners, independent reviewers, and staff of PVI, PeerView Institute for Medical Education, do not have any relevant financial relationships related to this CE activity unless listed below.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA information, and to apply for credit, please visit us at PeerView.com/SPK865. CME/MOC/AAPA credit will be available until December 12, 2024.Addressing Unmet Needs in Anticoagulation for Thrombosis: Next-Generation Strategies for Improving Care In support of improving patient care, PVI, PeerView Institute for Medical Education, 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.SupportThis activity is supported by an educational grant from the Bristol Myers Squibb and Janssen Alliance.Disclosure PolicyAll relevant conflicts of interest have been mitigated prior to the commencement of the activity.Faculty/Planner DisclosuresCo-Chair/PlannerRoxana Mehran, MD, has a financial interest/relationship or affiliation in the form of:Grant/Research Support from Abbott; ABIOMED; Affluent Medical; Alleviant Medical; Amgen Inc.; AM-Pharma B.V.; Arena Pharmaceuticals, Inc.; AstraZeneca; AtriCure Inc.; Biosensors; BIOTRONIK SE & Co KG; Boston Scientific Corporation; Bristol Myers Squibb; CardiaWave; Celonova Biosciences Inc.; Chiesi USA, Inc.; Concept Medical; Cytosorbents Europe GmbH; Daiichi Sankyo Inc.; Duke; Element Science, Inc.; Faraday Pharmaceuticals; Humacyte, Inc.; Idorsia Pharmaceuticals Ltd; Janssen Pharmaceuticals, Inc.; Koninklijke Philips N.V.; Magenta Therapeutics; M.A. Med Alliance SA; MediaSphere Medical LLC; Medtelligence; Medtronic; MJH Life Sciences; Novartis Pharmaceuticals Corporation; OrbusNeich Medical Company Group Holdings Limited; Penumbra, Inc.; PhaseBio Pharmaceuticals, Inc.; Pi-Cardia; PLx Pharma Inc.; Protembis GmbH; RenalPro; RM Global Partners; Shockwave Medical Inc.; Vivasure Medical Limited; and ZOLL Medical Corporation.Honoraria from ACC (BOT Member, SC Member CTR Program) and JAMA Cardiology (Associate Editor).Stock Shareholder in Applied Therapeutics and Elixir Therapeutics, LLC.Other Financial or Material Support in the form of personal fees from Affluent Medical; Cardiovascular Research Foundation; Daiichi Sankyo Brasil; ER Squibb & Sons LLC; Esperion Therapeutics, Inc.; Europa Group/Boston Scientific Corporation; Gaffney Events Educational Trust; Ionis Pharmaceuticals; IQVIA Inc; J-CalC; McVeigh Global Meetings and Events; Novartis Pharmaceuticals Corporation; Novo Nordisk Inc.; Oversome; Primer Healthcare of New Jersey; Radcliffe Institute for Advanced Study; SL Solutions LLC; TARSUS Cardiology; Vectura Group Ltd; and Vox Media, LLC.Co-Chair/PlannerJeffrey Weitz, MD, FRCPC, FRSC, FACP, has a financial interest/relationship or affiliation in the form of:Consultant and/or Advisor for Alnylam Pharmaceuticals, Inc.; Bayer AG; Boehringer Ingelheim Pharmaceuticals, Inc.; Bristol Myers Squibb; Daiichi Sankyo Co., Ltd.; Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Honoraria from Ionis Pharmaceuticals; Janssen Inc.; Merck & Co., Inc.; Pfizer; and Servier Canada.Planning Committee and Reviewer DisclosuresPlanners, independent reviewers, and staff of PVI, PeerView Institute for Medical Education, do not have any relevant financial relationships related to this CE activity unless listed below.

The brain and its vasculature used to be considered separate domains. But in the last decade, we've learned more about the interplay between brain cells and vascular cells. Pathological studies are revealing a surprising degree of overlap between cerebrovascular disease and neurodegenerative disease. All of this has led to the coining of a new word: the neurovasculome. Our guest today is Dr. Eric Smith, an author of a recent scientific statement about the neurovasculome in the journal Stroke. Dr. Smith is a Professor of Neurology, Radiology, and Community Health Sciences at the University of Calgary, and the holder of the endowed Katthy Taylor Chair in Vascular Dementia. He was interviewed about the neurovasculome by Dr. Christoph Stretz, a Vascular and Critical Care neurologist at the Warren Alpert Medical School of Brown University. Series 5, Episode 1 Featuring: Guest: Eric Edward Smith, MD, MPH, FRCPC, FAHA, University of Calgary Interviewer: Christoph Stretz, MD, Brown University Disclosures: Dr. Smith disclosed that he receives personal consulting fees from Alnylam Pharmaceuticals and Eli Lilly

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Bayer has recently opened a new plant in California to produce cell therapies, specifically a Parkinson's disease treatment being tested by its subsidiary Bluerock. This is a significant step forward in the field of cell therapy development.Meanwhile, Sana, another cell therapy developer, is implementing cost-saving measures, including layoffs and delaying certain research projects. These actions are aimed at ensuring the company's financial stability.In the world of biotech, Crispr gene editing technology is gaining increasing importance. Many companies in this field are led by former students of the pioneering scientists in the Crispr field. This shows the significant impact and potential of this technology.After a slow summer, there has been an uptick in biotech M&A activity. One notable acquisition is that of Orchard Therapeutics. This indicates the growing interest and investment in the biotech industry.In regulatory news, Pepgen has received clearance from the FDA to begin a study of its muscular dystrophy drug. This is an important milestone for the company, and we can expect to see the first results of this study next year.On the other hand, Alnylam has faced a setback as the FDA denied expanded approval for its RNA drug for a rare heart condition. This decision has set back the company's plans, highlighting the challenges faced by pharmaceutical companies in gaining regulatory approval for their products.Moving on to other news, Alvotech, a biopharmaceutical company, has received another rejection from the FDA for its biosimilar of Stelara. The FDA cited "deficiencies" at Alvotech's manufacturing plant as the reason for the rejection. This marks the fourth FDA rejection for Alvotech since last year.In a recent acquisition, Biogen has announced layoffs at Reata Pharmaceuticals, which it acquired for $7 billion. These layoffs will impact approximately one-third of Reata's workforce.In regulatory news, the European Commission has ordered Illumina to divest its subsidiary Grail. This decision comes after years of regulatory challenges and investigations.In clinical trial updates, Eli Lilly's mirikizumab has shown promising results in a Phase III trial for Crohn's disease. Over 54% of patients treated with mirikizumab achieved clinical remission at 52 weeks. Eli Lilly plans to submit a marketing application for mirikizumab in Crohn's disease to the FDA in 2024.Overall, these developments highlight the challenges faced by pharmaceutical companies in gaining regulatory approval for their products and the impact of acquisitions on workforce restructuring.The recent rejection by the FDA of a label expansion for Alnylam Pharmaceuticals' drug Onpattro to treat a rare heart condition has caused disappointment for the company. However, CEO Yvonne Greenstreet has outlined the company's future plans.This rejection sheds light on the challenges faced by drugmakers in gaining approval for their treatments. There is a delicate balance between patients and drugmakers wanting more liberal approval processes, while the medical community aims to ensure that drugs provide significant benefits compared to the risks.The FDA has been working towards bridging this gap through programs like Accelerating Rare Disease Cures (ARC). ARC aims to provide education campaigns and resources for developers to ensure all stakeholders are aligned.Alnylam's rejection serves as a reminder that surprises can still arise in the drug development and regulatory arenas. It also highlights the need for continuous innovation and adaptation in this field.Looking ahead, gene editing technology, particularly CRISPR-based therapies, holds great promise and could soon receive US approval. Many startups and scientists are actively working on advancements in this gene editing revolution.In summary, Alnylam's disap

William H. Sauer, MD, FHRS, CCDS, of Brigham and Women's Hospital is joined by Kolade Muchaili Agboola, MD, and Akshay Suvas Desai, MD, MPH, of Brigham and Women's Hospital to discuss the results and implications of the CASTLE-HTx trial.   https://www.hrsonline.org/education/TheLead   Host Disclosure(s): W. Sauer: Honoraria/Speaking/Consulting Fee: Biotronik, Biosense Webster, Inc., Abbott, Boston Scientific; Research (Contracted Grants for PIs Named Investigators Only): Medtronic   Contributor Disclosure(s): K. Agboola: No relevant financial relationships with ineligible companies to disclose.  A. Desai: Honoraria/Speaking/Consulting Fee: Abbott, Novartis, AstraZeneca, BioFourmis, Regeneron, Alnylam Pharmaceuticals, Bayer Healthcare Pharmaceuticals, Cytokinetics, AxonTherapies, Avidity Biosciences, Medpace, Merck, New Amsterdam Pharma, Parexel, Roche Diagnostics, GlaxoSmithKline, NovoNordisk, Veristat, Verily/Google, Zydus, River2Renal; Research (Contracted Grants for PIs Named Investigators Only): Bayer Healthcare Pharmaceuticals, Abbott Medical, AstraZeneca, Novartis

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in the Pharma and Biotech world. Today, we have some exciting news to share with you.## Cytiva's Optimized Method for Analyzing AAV CapsidsCytiva, formerly known as Pall Life Sciences, has developed an optimized method for analyzing adeno-associated virus (AAV) capsids. This method is fast, robust, and cost-efficient, addressing the limitations of current options. The optimized method uses anion exchange chromatography (AEX) with Capto Q chromatography resin to separate empty and full capsids. It has been successfully applied to various serotypes, achieving baseline peak separation. In a recent webinar, Cytiva demonstrated how their method can determine the percentage of full capsids in less than 5 minutes with low sample consumption. This method offers a solution to the challenges faced by process developers and provides a more accurate and cost-effective approach to analyzing AAV capsids.## Real-World Data in Rare Disease ResearchOur next topic is the use of real-world data (RWD) in gaining insights into rare diseases and improving drug development efficiency. RWD expands the insights that clinical trials can provide and helps researchers better understand patients with rare diseases. By analyzing real-world data, researchers can develop new treatments and interventions. However, there are challenges associated with using RWD, such as ensuring data quality and privacy protection. Despite these challenges, leveraging RWD is crucial for optimizing rare disease research.## Johnson & Johnson's Phase III Trial TerminationIn other news, Johnson & Johnson (J&J) has terminated its Phase III trial for Opsumit in pulmonary hypertension based on the recommendation of an independent data monitoring committee. Opsumit is a blockbuster drug for J&J, generating significant revenue for the company. This termination is a setback for J&J, but they continue to explore other opportunities in the field.## Allegations Against Bristol Myers SquibbBristol Myers Squibb (BMS) has been accused by Blue Cross and Blue Shield of Louisiana of conducting patent fraud to sustain a monopoly on its multiple myeloma drug Pomalyst. The healthcare insurance company alleges that BMS fraudulently extended patent protections for Pomalyst, preventing generic versions from entering the market. This case highlights the importance of fair competition and access to affordable treatments.## Roche's Successful Investment in Alnylam PharmaceuticalsOn a positive note, Roche has seen a return on its investment in Alnylam Pharmaceuticals. Alnylam's Phase II trial for its RNA interference therapy for hypertension showed promising results, reducing blood pressure in hypertensive patients. This collaboration between Roche and Alnylam has the potential to lead to innovative treatments for patients.## Astellas Withdraws Lawsuit Against Department of Health and Human ServicesAstellas has withdrawn a lawsuit against the Department of Health and Human Services after its prostate cancer therapy Xtandi was not included in Medicare's initial drug price negotiation list. While disappointed with the decision, Astellas remains committed to working with the government to ensure access to its therapy for Medicare beneficiaries.## Challenges in the Biotech IndustryLastly, the biotech industry is facing challenges as it navigates the post-pandemic markets. The risks are high, with therapeutics potentially failing. It is crucial for the industry to communicate its positive impact and build trust with the public. Recent leadership shakeups at companies like Biogen, Sanofi, and Bristol Myers Squibb highlight the need for stability and improvement within the industry.That's all for today's episode. Thank you for tuning in to Pharma and Biotech Daily, where we bring you the most important news in the world of Pharma and Biotech. Stay tuned for our next episode, and have

Dr. P and Dr. Saraswathy Nochur discuss why it is imperative that people of diverse backgrounds participate in clinical trials. Dr. Nochur explains what happens before, during, and after a clinical trial and notes that  people can find a clinical trial for which they may be qualified by visiting www.clinicaltrials.gov.Saraswathy Nochur is Chief DE&I Officer at Alnylam Pharmaceuticals in Cambridge, MA where she works with various stakeholders to increase diversity, promote equity and foster inclusion at Alnylam, and also co-leads initiatives to increase diversity in clinical trials.  She previously was Head, Global Regulatory Affairs at Alnylam for 14 years, enabling the development and approval of four RNA interference therapeutics for rare diseases.  She has been in the Boston biotechnology industry since 1989.  Sara holds a Ph.D. in Biochemical Engineering from MIT.  Sara is on the Board of Marinus Pharmaceuticals, a company developing innovative therapeutics to treat rare seizure disorders and of Decibel Therapeutics, who are developing regenerative and gene therapy strategies to restore hearing and balance. She is a host and member of the Board of Hospitality Homes, a non-profit organization that provides free/low-cost housing for patients/families who seek medical care in Boston. She is also on the Board of Biomedical Science Careers Program (BSCP), a non-profit organization that aims to increase the representation of underrepresented minority and disadvantaged individuals in all facets of science and medicine.  Please note that the website "clinical trials.gov" was inadvertently referred to as "clintrials.gov" in this episode. Please visit www.clinicaltrials.gov to find out more information about clinical trials being conducted in the United States.

Hereditary ATTR amyloidosis, or hATTR amyloidosis, is a rare, inherited condition that has an increased prevalence among people of African descent. This week, Dr. P is joined by Alnylam Patient Education Liaison (PEL), Valerie Graham, and Cece, who is living with hATTR amyloidosis, to learn more about the condition. We'll talk about the importance of discussing health history within families and the role genetic testing can play in helping to determine someone's risk for developing a disease. For more information about hATTR amyloidosis, and to request to speak with an Alnylam PEL who can answer questions you may have about hATTR amyloidosis, please visit www.hATTRbridge.com.Content sponsored and provided by Alnylam Pharmaceuticals. TTR-USA-00279Valerie Graham Valerie is a Patient Education Liaison (PEL) at Alnylam Pharmaceuticals. In her role, she is responsible for educating individuals and their families about hATTR amyloidosis – a rare, inherited condition. PELs are employees of Alnylam Pharmaceuticals. They do not act as healthcare providers and should not be considered part of your healthcare team.CeceCece's journey with hATTR amyloidosis began when she started to experience symptoms attributed to carpal tunnel syndrome at 55 years old. As time passed and she began to experience additional, unexplained symptoms, including shortness of breath, peripheral neuropathy and leg swelling, she was determined to find the cause. After years of searching for answers, she was eventually diagnosed with hATTR amyloidosis. Today, Cece is focused on educating the community about this inherited condition and encouraging others to advocate for their health and never give up.   

In this podcast, Paul Nioi, Vice President of Discovery and Translational Research at Alnylam Pharmaceuticals discusses the clinical development and manufacturing of RNA interference (RNAi) therapeutics. The post EPR Podcast 20 – RNAi Therapeutics – Paul Nioi, Alnylam Pharmaceuticals appeared first on European Pharmaceutical Review.

John Maraganore, former founding CEO of Alnylam Pharmaceuticals, on his life in biotech and developing RNA interference as a new class of medicine.

This episode is proudly sponsored by our partner, Alnylam Pharmaceuticals, the leading RNAi therapeutics company. When Stan Crooke ran Ionis and John Maraganore ran Alnylam, they were partners that turned into rivals — and not always friendly ones — as they persevered to pursue an entirely new therapeutic space; RNA-targeted drug discovery and development. Now, the pair of drug discovery titans have united once again in support of Dr. Crooke's n-Lorem Foundation—working to provide personalized medicines to the rarest of rare disease patients (nano-rare) using the antisense oligonucleotide (ASO) technology Stan led the creation of at Ionis. In this episode, Stan has a conversation with Dr. John Maraganore about John's past, their former rivalry, and the optimism shared between the two with respect to a better future for nano-rare patients.On This Episode We Discuss:- Being a dream merchant and driving a dream into a real therapeutic platform- Founding of Regulus and the synergy between Ionis and Alnylam throughout the years- What happens when two very competitive CEO-scientists have competing drugs- Overcoming their differences- Giving hope to a patient and their family with nano-rare diseases is powerful- Saving the world one life at a time- Bringing ASO and RNAi technologies together to collaboratively help nano-rare patients- A sustainable non-profit model?- Hope – a powerful thing to lose and an important thing to recover

“We're pioneering the ability to detect genetic disease earlier, and actually intervene in a way that is going to help those patients.” In this jam-packed 45-minute episode, we're joined by Dr Paul Nioi, Vice President of Discovery and Translational Research at Alnylam Pharmaceuticals and Chair of the Founders Board for Our Future Health. Join Patrick and Paul as they expose the two major reasons that 90% of clinical drug development fails, and why many believe that embedding genetics into target discovery and validation can increase the success rate of trials by 2-5 times. They also deep-dive into: the evolution of population genomics: from Iceland's DECODE to the UK Biobank examples of the value of recontact-by-genotype studies in supporting safety studies for novel targets, evidenced by the recontact of an ultra-rare homozygous loss-of-function carrier of the gene HAO1 the next chapter in population genomics that's pioneering the ability to detect genetic diseases earlier the challenges of recontacting participants in biobanks and avoiding overwhelming the healthcare system, whilst responsibly handling patients' sensitive data Alnylam's work to develop therapeutics for genetic diseases based on RNAi, the discovery that was awarded the Nobel Prize in Medicine or Physiology in 2006 Tune in to benefit from Paul's invaluable insights and expertise, which are coloured with practical examples from his career throughout. After listening to this 45 minute episode, you will: recognise the role of genetics in supporting the safety and efficacy of novel targets in clinical development understand the evolution of population genomics and what's next understand how RNAi works, and how it differs from gene editing and other forms of next-generation therapies

“We're pioneering the ability to detect genetic disease earlier, and actually intervene in a way that is going to help those patients.” In this jam-packed 45-minute episode, we're joined by Dr Paul Nioi, Vice President of Discovery and Translational Research at Alnylam Pharmaceuticals and Chair of the Founders Board for Our Future Health. Join Patrick and Paul as they expose the two major reasons that 90% of clinical drug development fails, and why many believe that embedding genetics into target discovery and validation can increase the success rate of trials by 2-5 times. They also deep-dive into: * the evolution of population genomics: from Iceland's DECODE to the UK Biobank * examples of the value of recontact-by-genotype studies in supporting safety studies for novel targets, evidenced by the recontact of an ultra-rare homozygous loss-of-function carrier of the gene HAO1 * the next chapter in population genomics that's pioneering the ability to detect genetic diseases earlier * the challenges of recontacting participants in biobanks and avoiding overwhelming the healthcare system, whilst responsibly handling patients' sensitive data * Alnylam's work to develop therapeutics for genetic diseases based on RNAi, the discovery that was awarded the Nobel Prize in Medicine or Physiology in 2006 Tune in to benefit from Paul's invaluable insights and expertise, which are coloured with practical examples from his career throughout. After listening to this 45 minute episode, you will: recognise the role of genetics in supporting the safety and efficacy of novel targets in clinical development understand the evolution of population genomics and what's next understand how RNAi works, and how it differs from gene editing and other forms of next-generation therapies

In this podcast, you will hear a keynote presentation from Dr John Maraganore from the 2022 PODD Conference regarding lessons learned from his 19 years as CEO of Alnylam Pharmaceuticals, leading a team that conquered some very difficult drug delivery challenges.  To learn more about the PODD Conference, please visit PODDConference.com.  

Rachel Meyers serves as the chief scientific officer at Faze Medicines. She was most recently an entrepreneur-in-residence at Third Rock Ventures (TRV), where she helped drive the creation of Faze Medicines. Rachel brings more than twenty years of drug discovery and development expertise to Faze and is an expert in the development of RNAi therapeutics. Prior to joining Faze and TRV, she served as a biotech consultant in the Greater Boston area and served as SVP Research and RNAi Lead Development at Alnylam Pharmaceuticals where she spent over 13 years. Prior to Alnylam, Rachel was a senior scientist at Millennium Pharmaceuticals. She also serves on several scientific advisory boards, consults on nucleic acid therapeutic development, is listed as an inventor on many patents and patent applications, and has numerous peer-reviewed publications. She completed her postdoctoral training at Harvard Medical School in the field of signal transduction and received her PhD from MIT in the field of in vitro transcription. Rachel also enjoys participating in women-in-science panels and events, teaching at Harvard and Brandeis, contributing in ED&I initiatives and playing a variety of sports, particularly racket sports. When Rachel isn't engaged in one of the many things above, she is traipsing around the world with her husband and 2 kids.

This week on the podcast Agustin addresses the flurry of acquisitions that have precipitated in the recent weeks as well as the growing excitement in the sector as indices rebound from their May lows. He also highlights the recent data from Alnylam Pharmaceuticals regarding their therapy for ATTR-CM and the increasingly competitive market that is developing.If you are a fan of this show make sure to like, comment, subscribe and follow me on Twitter @biotech_bros. If you have any questions regarding the product offerings feel free to explore my website biotechbros.com

Is drug pricing reform really happening this time? Are things finally turning around for biotech? And is it ever wise to tweet your food? Rachel Cohrs, STAT's Washington correspondent, joins us to explain how congressional Democrats are on the verge of a coup in drug pricing — and what could still stand in their way. We also discuss the latest news in the life sciences, including some hotly anticipated data from Alnylam Pharmaceuticals, a $4 billion buyout deal, and other surprisingly good news for biotech.

How digital engagement is empowering patients to make better health care decisions Patient centricity has long been a buzzword in the world of pharma marketers, but what does true patient-centric marketing look like? In the MM&M podcast “Redefining Patient Centricity at the Point of Care,” Julie Cosgrove, director of marketing, Alnylam Pharmaceuticals and Isaac Gealer, lead client experience manager, Phreesia Life Sciences, explored the digital innovations at point of care that help marketers target relevant audiences at the patient level. Moderated by Marc Iskowitz, executive editor, MM+M.

Laurence joined Decibel in January 2020. He previously served as an entrepreneur in residence at Third Rock Ventures, where he focused on novel drug discovery and development opportunities. Before that, Laurence was CEO of Warp Drive Bio, a small molecule company focused on novel oncology and antibiotic drug discovery based on natural products, until its merger with Revolution Medicines in 2018. Prior to that, he was the senior vice president and chief business officer of Alnylam Pharmaceuticals, where he was responsible for the company's business development, finance and legal functions. Before coming to Alnylam, Laurence was the chief business officer at Ensemble Discovery, where he led the company's strategic planning and corporate development efforts. Laurence previously spent ten years at Millennium Pharmaceuticals in a range of general management and business development positions. In addition to his professional interests, Laurence is a board member of the Possible Project and board advisor to Life Science Cares. He earned his Ph.D. from King's College London and his B.A. from Cambridge University.

Apple verliert mit dem iPod auch den Hoheits-Status an der Börse. Terra- und Luna-Investoren verlieren unfassbar viel Geld und schwedischer Tabak seine Unabhängigkeit. Außerdem liebt Disney das Streaming und die Shop Apotheke das E-Rezept. Die Bank of America sucht stabile Firmen für schwierige Zeiten und findet Humana (WKN: 856584), Alnylam Pharmaceuticals (WKN: A0CBCK), Jazz Pharmaceuticals (WKN: A1JS1K) und CVS Health (WKN: 859034). Terra und Luna erleben den schlimmsten Crash der Kryptogeschichte. Wir erklären, was dahintersteckt. Diesen Podcast der Podstars GmbH (Noah Leidinger) vom 12.05.2022, 3:00 Uhr stellt Dir die Trade Republic Bank GmbH zur Verfügung. Die Trade Republic Bank GmbH wird von der Bundesanstalt für Finanzaufsicht beaufsichtigt.

This week, Dr. P will be joined by Rita Lazenby, a Patient Education Liaison (PEL) from Alnylam Pharmaceuticals, to discuss hereditary ATTR amyloidosis and the  importance of learning about genetic health history within families and some tools that may help, like genetic counseling and testing.Content sponsored and provided by Alnylam Pharmaceuticals.

In this episode, Ayesha shed light on National Colorectal Cancer Awareness Month by talking about a new colon cancer screening campaign from Exact Sciences that features a TV ad with Katie Couric. Couric, who is a long-time advocate of colon cancer screening after her husband passed away from colon cancer over 20 years ago, continues her mission to help raise awareness about the importance of early screening for the disease. Exact Sciences' campaign is appropriately called “Mission to Screen” and includes a TV commercial featuring Couric and her daughter, along with other people that Couric is seen speaking to about their reasons for screening for colon cancer. The ad highlights Exact Sciences' Cologuard, a DNA-based stool test for colon cancer detection.The editorial team also learned about Alnylam Pharmaceuticals' lawsuits against mRNA COVID-19 vaccine makers Pfizer and Moderna over patent infringements. Alnylam claims it invented the lipid nanoparticle delivery technology used in the vaccines and is seeking “fair compensation” for its use. Moderna hit back at Alnylam, accusing it of “blatant opportunism” and saying that it has worked for years on a different version of the RNA delivery technology. The editorial team said it was difficult to take sides in this case without all of the evidence/information, but it isn't difficult to “believe” Moderna as it has been developing RNA vaccines for over a decade.Read the full articles here: Alnylam Files Lawsuits Against Pfizer and Moderna Over Patent Infringement of RNA Delivery TechnologyExact Sciences and Katie Couric Partner for Colon Cancer Awareness Month with Cologuard TV AdFor more life science and medical device content, visit the Xtalks Vitals homepage.Follow Us on Social MediaTwitter: @Xtalks Instagram: @Xtalks Facebook: https://www.facebook.com/Xtalks.Webinars/ LinkedIn: https://www.linkedin.com/company/xtalks-webconferences YouTube: https://www.youtube.com/c/XtalksWebinars/featured

Did you know that hearing loss is one of the largest areas of unmet need in medicine? It affects approximately 466 million people worldwide, including 48 million people in the United States. Today's guest Laurence Reid, CEO of Decibel Therapeutics dives deeper into meeting that unmet need. Decibel has architected a whole platform to restore hearing and balance loss. With several treatments in their pipeline now, one in phase 1 clinical trials, they're making great progress. Learn more about how Decibel started, current pipeline, and the future of biotech industry. About Laurence Reid, Ph.D.Laurence joined Decibel in January 2020. He previously served as an entrepreneur in residence at Third Rock Ventures, where he focused on novel drug discovery and development opportunities. Before that, Laurence was CEO of Warp Drive Bio, a small molecule company focused on novel oncology and antibiotic drug discovery based on natural products, until its merger with Revolution Medicines in 2018. Prior to that, he was the senior vice president and chief business officer of Alnylam Pharmaceuticals, where he was responsible for the company's business development, finance and legal functions. Before coming to Alnylam, Laurence was the chief business officer at Ensemble Discovery, where he led the company's strategic planning and corporate development efforts. Laurence previously spent ten years at Millennium Pharmaceuticals in a range of general management and business development positions. In addition to his professional interests, Laurence is a board member of the Possible Project, a board member of Garuda Therapeutics and board advisor to Life Science Cares. He earned his Ph.D. from King's College London and his B.A. from Cambridge University.About DecibelDespite the overwhelming burden of hearing loss and balance disorders, the search for therapeutics to treat these conditions remains one of the largest areas of unmet needs in medicine. At Decibel, we are exclusively focused on discovering and developing transformative treatments to restore and improve hearing and balance. We have assembled a highly experienced scientific team and built a unique platform to achieve this goal.Show notesDecibel website: https://decibeltx.com/Pictures of the inner ear: https://www.decibeltx.com/our-approach/QualioPrevious episodes: https://www.qualio.com/from-lab-to-launch-podcastApply to be on the show: https://forms.gle/uUH2YtCFxJHrVGeL8Music by keldez

I've talked about companies that have decided to stop doing business with/in Russia. Here, I want to talk about those that have not cut ties with Russia as of 5 March 2022: McDonald's tops the list as it's trending on American Twitter. Other companies are: Burger King, PepsiCo, Coca Cola and Estee Lauder. Hilary Russ, a writer with Reuters, writes about New York state pension's fund chief sending several letters to companies that are yet to cut ties with Russia: Mondelez International, Kimberly-Clark Corp, Coty Inc, Trimble Inc and Alnylam Pharmaceuticals.

Educational Objectives • Review the clinical and economic burdens associated with transthyretin amyloidosis (ATTR) • Recall key characteristics supporting the use of current and emerging therapies for the treatment of ATTR including mechanisms of action, clinical data, dosing, administration, and potential adverse effects Faculty: Svetlana Goldman, PharmD, BCACP, CDCES Clinical Pharmacist UC Davis Health Sacramento, California Yvette Hellier, PharmD, BCACP Clinical Pharmacist UC Davis Health Sacramento, California The following contributors have no relevant financial relationships with commercial interests to disclose:  Faculty Svetlana Goldman, PharmD, BCACP, CDCES, and Yvette Hellier, PharmD, BCACP Pharmacy Times Continuing Education™ is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This activity is approved for 0.75 contact hour (0.075 CEU) under the ACPE universal activity number 0290-0000-22-045-H01-P. The activity is available for CE credit through February 28, 2023. This activity is supported by an educational grant from Alnylam Pharmaceuticals. Learn more about your ad choices. Visit megaphone.fm/adchoices

Educational Objectives • Review the clinical and economic burdens associated with transthyretin amyloidosis (ATTR) • Recall key characteristics supporting the use of current and emerging therapies for the treatment of ATTR including mechanisms of action, clinical data, dosing, administration, and potential adverse effects Faculty: Svetlana Goldman, PharmD, BCACP, CDCES Clinical Pharmacist UC Davis Health Sacramento, California Yvette Hellier, PharmD, BCACP Clinical Pharmacist UC Davis Health Sacramento, California The following contributors have no relevant financial relationships with commercial interests to disclose:  Faculty Svetlana Goldman, PharmD, BCACP, CDCES, and Yvette Hellier, PharmD, BCACP Pharmacy Times Continuing Education™ is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This activity is approved for 0.75 contact hour (0.075 CEU) under the ACPE universal activity number 0290-0000-22-045-H01-P. The activity is available for CE credit through February 28, 2023. This activity is supported by an educational grant from Alnylam Pharmaceuticals. Learn more about your ad choices. Visit megaphone.fm/adchoices

Educational Objectives • Review the clinical and economic burdens associated with transthyretin amyloidosis (ATTR) • Recall key characteristics supporting the use of current and emerging therapies for the treatment of ATTR including mechanisms of action, clinical data, dosing, administration, and potential adverse effects Faculty: Svetlana Goldman, PharmD, BCACP, CDCES Clinical Pharmacist UC Davis Health Sacramento, California Yvette Hellier, PharmD, BCACP Clinical Pharmacist UC Davis Health Sacramento, California The following contributors have no relevant financial relationships with commercial interests to disclose:  Faculty Svetlana Goldman, PharmD, BCACP, CDCES, and Yvette Hellier, PharmD, BCACP Pharmacy Times Continuing Education™ is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. This activity is approved for 0.75 contact hour (0.075 CEU) under the ACPE universal activity number 0290-0000-22-045-H01-P. The activity is available for CE credit through February 28, 2023. This activity is supported by an educational grant from Alnylam Pharmaceuticals. Learn more about your ad choices. Visit megaphone.fm/adchoices

Call Nigel Coffey a change agent, or call him a serial innovator. Everywhere Nigel takes on a new leadership challenge, he executes a program that adds tangible bottom-line value to his new organization. Starting with industry benchmarking, following with an audit of payments to suppliers to identify root cause issues and control weaknesses, and progressing through a systematic program of automation, Nigel has succeeded time and time again in moving each company to world-class procure-to-pay processes and technologies.In his career Nigel has held senior business service, finance and transformation roles at Pfizer, PepsiCo, Shire Pharmaceuticals/Takeda and now Alnylam Pharmaceuticals. As the Head of Finance operations at this rapidly-growing biotech, he's helping optimize the processes to help this company produce leading-edge therapies based on the latest RNA interference science.Hear Nigel and Danny Thompson, SVP of Market & Product Strategy at apexanalytix, as they discuss:How fresh industry benchmarking information on finance and procure-to-pay best practices can be leveraged to establish goals and inform business cases for talent and technology.The one-two punch of recovery audit: improving AP profitability and uncovering weak controls, supplier issues and root causes of overpayments.Optimising prior to automating: why improving and rationalising end-to-end processes, policies, procedures and controls is critical before selecting and implementing technology. Featuring: The latest 2021 benchmarking results from apexanalytix's P2PKey Practices Benchmarking survey. The newest P2P survey data and insights from top tier global companies.Webinar slides: https://bit.ly/3dFttvnWebinar video: https://www.apexanalytix.com/resources/webinars/one-leader-s-proven-formula-dramatically-improve-finance-and-gbs-operationsSpeakers:Nigel Coffey, Head of Finance Operations, Alnylam PharmaceuticalsDanny Thompson, SVP, Market and Product Strategy at apexanalytix

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Manish Thapar, MD, AGAF, FAASLD, FACG Acute hepatic porphyria (AHP) is a rare genetic condition characterized by severe, diffuse abdominal pain and a broad range of seemingly unrelated symptoms. AHP presents differently in each patient, with symptoms that mirror those of more common conditions, making it difficult to diagnose. Although AHP can be misleading, it can be easy to detect once you know what to look for. Joining Dr. Charles Turck to discuss AHP signs and symptoms and when a doctor should consider AHP in a patient is porphyria expert Dr. Manish Thapar, gastroenterologist and associate professor of medicine in Philadelphia. ©2021 Alnylam Pharmaceuticals, Inc. All Rights Reserved. AS1-USA-01069

This was recorded live on 12/08/21 Welcome to the Cyber Fire Fight podcast where we discuss “Fight Fire with Fire: Proactive #Cybersecurity Strategies for Today's Leaders,” a new book offering collective advice from industry experts to improve cyber strategies. Join #Fortinet's Renee Tarun, Deputy CISO, and Sanju Misra, CISO and VP, Information Security at Alnylam Pharmaceuticals as they discuss the transition from technologist to strategist and their experiences in the #cybersecurity industry. #CyberFireFight

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Manish Thapar, MD, AGAF, FAASLD, FACG Acute hepatic porphyria (AHP) is a rare genetic condition characterized by severe, diffuse abdominal pain and a broad range of seemingly unrelated symptoms. AHP presents differently in each patient, with symptoms that mirror those of more common conditions, making it difficult to diagnose. Although AHP can be misleading, it can be easy to detect once you know what to look for. Joining Dr. Charles Turck to discuss AHP signs and symptoms and when a doctor should consider AHP in a patient is porphyria expert Dr. Manish Thapar, gastroenterologist and associate professor of medicine in Philadelphia. ©2021 Alnylam Pharmaceuticals, Inc. All Rights Reserved. AS1-USA-01069

Host: Charles Turck, PharmD, BCPS, BCCCP Guest: Manish Thapar, MD, AGAF, FAASLD, FACG Acute hepatic porphyria (AHP) is a rare genetic condition characterized by severe, diffuse abdominal pain and a broad range of seemingly unrelated symptoms. AHP presents differently in each patient, with symptoms that mirror those of more common conditions, making it difficult to diagnose. Although AHP can be misleading, it can be easy to detect once you know what to look for. Joining Dr. Charles Turck to discuss AHP signs and symptoms and when a doctor should consider AHP in a patient is porphyria expert Dr. Manish Thapar, gastroenterologist and associate professor of medicine in Philadelphia. ©2021 Alnylam Pharmaceuticals, Inc. All Rights Reserved. AS1-USA-01069

STAT's Rachel Cohrs drops in to talk about the latest updates in drug pricing reform. Then, John Maraganore joins us to talk about his decision to step down as CEO of Alnylam Pharmaceuticals after almost 20 years with the company. We also discuss the latest news in biotech, including the Covid-19 vaccine for kids and Moderna's disappointing earnings call.

When Caribou Biosciences (NASDAQ: CRBU) became the seventh publicly-traded CRISPR stock in July 2021, I saw an exchange on social media. One person asked why the company sported a market valuation of $900 million when another newly-public CRISPR stock, Verve Therapeutics (NASDAQ: VERV), was valued near $2.3 billion. "Is there any reason for this other than the timing of the IPOs?", asked the individual. The thread received multiple responses confirming the seemingly large valuation difference between the two companies, with others "agreeing" or responding that they were buying Caribou Biosciences because of it. That was 100% the wrong take. I've observed similar arguments among individual investors within the gene editing space. However, it's important to acknowledge that there are significant differences between gene editing approaches and technology platforms. Caribou Biosciences and Verve Therapeutics might both be using CRISPR systems, but that's where the overlap ends. They're developing completely different tools that have almost nothing in common. Individual investors don't necessarily need to have a deep technical understanding of gene editing tools, but I would argue that there's a minimum level of information required to responsibly invest in the field. Unfortunately, the way the internet works means most investors aren't provided with the information they need. Let's fix that. In this episode of the podcast, 7investing Lead Advisors Maxx Chatsko (me) and Dan Kline introduce simple frameworks for evaluating opportunities and challenges in gene editing. These can be summarized as follows: The Emerging Approaches: There's first-generation tools (gene editing), second-generation tools (base editing), and third-generation tools (prime editing). These approaches are not limited to any specific system. For example, there are CRISPR, TALEN, ARCUS, and other tools capable of performing base editing. The Major Applications: There are knock outs, insertions, activations, precise corrections, knock ins, and other uses of gene editing tools. Each has advantages and disadvantages. The Major Administration Routes: This primarily comes down to in vivo (inside the body) and ex vivo (outside the body). Each has advantages and disadvantages. In addition to this podcast introducing the three frameworks, 7investing Lead Advisor Maxx Chatsko has written an in-depth article explaining these frameworks and how each gene editing stock fits into each -- and it's free to read! Publicly-traded companies mentioned in this podcast include Alnylam Pharmaceuticals, Beam Therapeutics, Caribou Biosciences, Cellectis, CRISPR Therapeutics, Editas Medicine, Graphite Bio, Intellia Therapeutics, Precision BioSciences, Sana Biotechnology, and Verve Therapeutics. 7investing Lead Advisors may have positions in the companies that are mentioned. This interview was originally recorded on August 2nd, 2021 and was first published on August 3rd, 2021. --- Send in a voice message: https://anchor.fm/7investing/message Support this podcast: https://anchor.fm/7investing/support

"10-Baggers" are prime examples of the amazing power of compounding in investing. The term was originally coined by legendary investor Peter Lynch in his book One Up on Wall Street. It refers to a company that goes on to return 10 times your initial investment, turning $1,000 into $10,000 or $100,000 into $1 million. Lynch went on to become one of the greatest money managers of all time. He ran Fidelity's Magellan Fund for 13 years and achieved an incredible compound average growth rate of 29.2%. During his time at the helm, Lynch stocked the Magellan Fund with more than 100 companies that went on to become 10-Baggers. Stocks that generate those types of returns can completely change your entire financial future. Buying into just a few 10-Baggers can supercharge the overall returns of even a diversified investment portfolio. But finding these high-flying performers is no easy feat. It requires diligent upfront research, a methodical investing process, and a boatload of patience. Are there future 10-Baggers available in the stock market today? If so, how do we go about finding them? And if we're fortunate enough to find and invest in them, how should we deal with them becoming an outsized position? In our June Team Podcast, our 7investing advisors share our approach to finding 10-Baggers. We reveal the different metrics and factors that we each look for, as well as several of the personal lessons we've learned along the way. Publicly-traded companies mentioned in this interview include Alnylam Pharmaceuticals, Apple, Costco, CrowdStrike, FireEye, MongoDB, Repligen, Tesla, and Ubiquiti Inc. 7investing's advisors may have positions in the companies mentioned. --- Send in a voice message: https://anchor.fm/7investing/message Support this podcast: https://anchor.fm/7investing/support

Dr. Angelika Erwin, a board‐certified clinical geneticist and internist at Cleveland Clinic, as well as a porphyria expert and member of the Porphyrias Consortium discusses acute hepatic porphyria (AHP), when a doctor should suspect AHP in a patient and how to test for it. Dr. Angelika Erwin is a medical geneticist in Miami, FL, affiliated with Cleveland Clinic, who specializes in clinical genetics and is experienced in lysosomal storage diseases, cancer genetics, general medical genetics, lipodystrophy, and porphyrias. She is a member of the Porphyrias Consortium, comprised of the nation's foremost experts in porphyria, and serves as a consultant of Alnylam Pharmaceuticals to help educate about the condition. #AHP #Porphyria

Alnylam Pharmaceuticals, Inc., Q1 2021 Earnings Call, Apr 29, 2021

On this episode of the podcast, Patrick is joined by Josh Friedman, Senior Director of Research at Alnylam Pharmaceuticals. Alnylam works on the discovery and development of RNA interference therapeutics; treatments for genetic diseases which work by ‘switching off' the specific genes associated with a given condition. In this fifth episode of our precision pioneers miniseries, we explore how RNA interference can be applied to treat common and rare genetic diseases, and dive deep into the genetics of non-alcoholic fatty liver disease and NASH.

On this episode of the podcast, Patrick is joined by Josh Friedman, Senior Director of Research at Alnylam Pharmaceuticals. Alnylam works on the discovery and development of RNA interference therapeutics; treatments for genetic diseases which work by ‘switching off’ the specific genes associated with a given condition. In this fifth episode of our precision pioneers miniseries, we explore how RNA interference can be applied to treat common and rare genetic diseases, and dive deep into the genetics of non-alcoholic fatty liver disease and NASH.

Every month, our team of Lead Advisors is each asked the same question, which is, "what is your very best idea in the stock market right now?"If you are a 7investing subscriber who has seen our reports answering that question, you know we look at metrics beyond just the reported earnings per share and the company's revenue. We love to dig into the profitability ratios, operating margins, and other metrics that will drive that business over time.But how exactly do we learn about those metrics and transparently relay our findings to our subscribers? To help, we partnered with YCharts, a full-service fundamental research platform for investors. While we use YCharts here at 7investing almost every day, we thought it would be fun to bring our partnership to the listeners of our Livestream! We dug into several companies, including Alnylam Pharmaceuticals, Criteo, CVS, Facebook, NVIDIA, Rocket, Square, 2U, and Veeva - and greatly appreciated having the exceptional team from YCharts on hand to spin up graphics on the spot. Our mission at 7investing is to empower individual investors, and our friends at YCharts are a great resource to supplement your 7investing subscription. For a limited time, YCharts is offering a discount on their service to all 7investors! Use code "7investing" at checkout for a 20% discount. Welcome to 7investing. We are here to empower you to invest in your future! We publish our 7 best ideas in the stock market to our subscribers for just $17 per month. Start your journey toward's financial independence: https://www.7investing.com/subscribe Stop by our website to level-up your investing education: https://www.7investing.com Follow us on Social Media ► https://www.facebook.com/7investing/ ► https://twitter.com/7investing ► https://instagram.com/7investingofficial --- Send in a voice message: https://anchor.fm/7investing/message

This week, Dr. P will be joined by Joanna Pearson, a Patient Education Liaison (PEL) from Alnylam Pharmaceuticals, to discuss a rare, genetic condition that disproportionately affects African Americans. Known as hereditary ATTR amyloidosis, or hATTR amyloidosis, the disease is more common for people of certain ethnicities, including those of African (West African: Ghana, Ivory Coast, Nigeria, and Sierra Leone) and Afro-Caribbean descent. For more information about hATTR amyloidosis, and to request to speak with an Alnylam PEL who can answer questions you may have about hATTR amyloidosis, please visit www.hATTRbridge.com. Content sponsored and provided by Alnylam Pharmaceuticals. ABOUT OUR GUEST: JOANNA PEARSON, RN, BSN, CPRH Joanna Pearson is a Patient Education Liaison at Alnylam Pharmaceuticals. In her role, she is responsible for educating individuals and their families about hereditary transthyretin (hATTR) amyloidosis – a rare, life-threatening condition. Joanna received her Bachelor of Science in Nursing from Ohio University and has worked in patient education at all levels of her career.

Morie A Gertz, MD, MACP, Professor of Medicine at the Mayo Clinic College of Medicine and Science in Rochester, Minnesota discusses signs and symptoms that are suspicious of ATTR amyloidosis. This CME activity is possible through an educational grants from Akcea Therapeutics and Alnylam Pharmaceuticals. To obtain credit for this activity, please visit https://checkrare.com/learning/p-managing-attr-module1-suspecting-attr-amyloidosis/

Morie A Gertz, MD, MACP, Professor of Medicine at the Mayo Clinic College of Medicine and Science in Rochester, Minnesota discusses best practices to diagnose ATTR amyloidosis. This CME activity is possible through an educational grants from Akcea Therapeutics and Alnylam Pharmaceuticals. To obtain credit for this activity, please visit https://checkrare.com/learning/p-managing-attr-module2-diagnosing-attr-amyloidosis/

Alnylam Pharmaceuticals, which take its name from the center and brightest star in Orion’s Belt, is working on an RNAi therapy to destroy the genome of the novel coronavirus — and with it, its ability to replicate and kill. Hosted by Jim Greenwood.

Our senior producer, Mariel Carr, talks with John Maraganore, the CEO of Alnylam Pharmaceuticals, a company developing an antiviral medication for COVID-19. When news broke in January about the new coronavirus, John Maraganore made the decision to pause other drugs in development and pivot to working on an antiviral medication for this new and alarmingly infectious virus. He says it was a difficult decision, but this virus had all the ingredients to become a pandemic. “And when you have a public health crisis like this, that’s what you do.”

Alnylam Pharmaceuticals, which take its name from the center and brightest star in Orion’s Belt, is working on an RNAi therapy to destroy the genome of the novel coronavirus — and with it, its ability to replicate and kill.

Co-Host: Amy E. Krambeck, MD Michael O. Koch Professor of Urology, Indiana University and IU Health At the conclusion of this activity, participants will be able to: 1. Interpret metabolic evaluations for patients with stones 2. Determine when to genetically test patients with stones 3. Analyze current gene panels available and options for patients This educational series is supported by an independent educational grant from Alnylam Pharmaceuticals, Inc.

RNA should be a terrible drug target. It’s long, noodle-like structure lacks the nooks and crannies that small molecule drugs use to grab onto proteins and thereby control them. But a decades-old disregard for RNA is starting to change. In August 2018, the US Food and Drug Administration approved the first-ever RNA interference (RNAi) drug, which uses a double-stranded RNA molecule to prevent the production of disease-related proteins. In the past two years, several startups have launched to show that some RNAs can, just like proteins, be drugged with small molecules. And a third group of companies recently emerged with plans to drug proteins that make modifications to RNA, part of the budding field of epitranscriptomics. In this episode, C&EN visits Alnylam Pharmaceuticals, Novartis, and Accent Therapeutics to discuss these three strategies, and to understand how RNA-modulating therapies will compete in the wider world of drug discovery. Visit our website for a script of this podcast. Music credits: “And...(Insert Problem Here)” by GR∑Y is licensed under CC BY-NC 4.0.  “Raccoon Family Robinson”, “Robot Park”, and "The Confrontation" by Podington Bear are licensed under CC BY-NC 3.0.  “Wireless” by Lee Rosevere is licensed under CC BY-NC 4.0.  Image credit: J. Am. Chem. Soc.

Dave Hennessy interviews Karen Anderson live from the NEHRA Conference this past September. Karen talks to Dave about how it is important to her to involve many non-HR folks in the culture building she is working on at Alnylam and much more. The live atmosphere makes for a more engaged and exciting tone - Enjoy!

Karen Anderson-Chief Human Resources Officer at Alnylam Pharmaceuticals. Karen has an extensive (20+ year) career in Human Resources having held a variety of Executive leadership roles with major life sciences corporations such as Bayer, Baxter, and Biogen prior to joining Alnylam. During the show she discusses and what organizations need to do in order to build an agile organization. Just a few of the Q&A’s Karen covers: -How do you retain culture, vision, and values during times of rapid growth -How to gain a competitive recruitment edge in a sea of fierce, local competition -How does HR add value outside of traditional HR responsibilities (comp, benefits, etc)

PharmaPills - Pillole dal farmaceutico: Novità, Curiosità e Lavoro dal mondo del farmaceutico. A cura di Stefano LagravineseIn questa puntata parliamo di:Aziende: Abpi, AIFA, GSK, FDA, Abbvie, Neurocrine Biosciences, Roche, Eisai, Biogen, Siga Technologies, Alnylam Pharmaceuticals, Fidia Farmaceutici, Medpace.Persone: Beatrice Lorenzin (ex Ministro della Salute), Maria Luisa Moro (Centro di Coordinamento Nazionale), Carlo Petrini (Centro di Coordinamento Nazionale).Nuove terapie: elagolix, atezolizumab, Ban2401, tecovirimat.Patologie: endometriosi, tumore del polmone, Alzheimer, vaiolo.Lavoro: Medical Science Liaison, Clinical Project Manager, Clinical Research Associate.Il mercoledì alle h 12.00 su Spreaker.com e iTunes.Seguici su: www.telegram.me/pharmapillswww.facebook.com/pharmapills/

PharmaPills - Pillole dal farmaceutico: Novità, Curiosità e Lavoro dal mondo del farmaceutico. A cura di Stefano LagravineseIn questa puntata parliamo di:Aziende: Abpi, AIFA, GSK, FDA, Abbvie, Neurocrine Biosciences, Roche, Eisai, Biogen, Siga Technologies, Alnylam Pharmaceuticals, Fidia Farmaceutici, Medpace.Persone: Beatrice Lorenzin (ex Ministro della Salute), Maria Luisa Moro (Centro di Coordinamento Nazionale), Carlo Petrini (Centro di Coordinamento Nazionale).Nuove terapie: elagolix, atezolizumab, Ban2401, tecovirimat.Patologie: endometriosi, tumore del polmone, Alzheimer, vaiolo.Lavoro: Medical Science Liaison, Clinical Project Manager, Clinical Research Associate.Il mercoledì alle h 12.00 su Spreaker.com e iTunes.Seguici su: www.telegram.me/pharmapillswww.facebook.com/pharmapills/

John Maraganore is CEO of Alnylam Pharmaceuticals. In his talk with Nature Biotechnology he discusses his decade at Biogen, running an RNAi company when skepticism covered the field, and growing up the son of Greek immigrants in Chicago. See acast.com/privacy for privacy and opt-out information.

Fifteen years ago, folks in the industry were buzzing about RNAi the way they talk about CRISPR today. Then things went quiet for the technology, at least in the news. Until last year. In September of 2017, Alnylam Pharmaceuticals, the leader in the RNAi space, announced such positive phase III study results that most experts in the business expect an FDA approval soon. It will be the first for an RNAi drug.

Today in FirstWord:

Show description/summary:1) This variant alters protein function, but is it pathogenic? (Neurology® Genetics)2) What’s Trending: PFO UpdatesThis podcast begins and closes with Dr. Robert Gross, Editor-in-Chief, briefly discussing highlighted articles from the November 7, 2017 issue of Neurology. In the first segment, Dr. Michelle Mauermann talks with Dr. Massimo Pandolfo about his recent Neurology® Genetics editorial, entitled “This variant alters protein function, but is it pathogenic?”. In the second part of the podcast, Dr. Nicole Chiota focuses her interview with Dr. Steven Messe on recent PFO/stroke updates. Disclosures can be found at Neurology.org.DISCLOSURES: Dr. Mauermann serves on the editorial board of Mayo Clinic Proceedings; receives publishing royalties from Oxford, and has received research support from Ionis Pharmaceuticals, Inc. and Alnylam Pharmaceuticals.Dr. Pandolfo has served on the scientific advisory boards of Apopharma and Voyager Therapeutics; has served on the editorial boards of Acta Neurologica Belgica, Orphanet Journal of Rare Diseases, and Neurology Genetics; is co-holder of a patent for Direct molecular diagnosis of Friedreich’s ataxia; has been a consultant for Biomarin and UCB; has received research support from Biomarin, Fonds National de la Recherche Scientifique (Belgium), Offrez-moi-la-lune, Friedreich's Ataxia Research Alliance, and Association Belge contre les Maladies neuro-Musculaires, and receives royalty payments from Athena Diagnositcs for a Friedreich's ataxia diagnostic test.Dr. Chiota reports no disclosures.Dr. Messe has served on the scientific advisory board of the Yale University Cardiovascular Research Group; has received travel funding from the American Academy of Neurology; has received publishing royalties from Up To Date; has been a consultant for Claret Medical; and has received research support from WL Gore, Glaxo Smith Kline, Bayer, Mallinkrodt, and NIH.

Today in FirstWord:

Today in FirstWord:

Adapted from the 2016 Chief Medical Officer Summit keynote address, Dr Akshay Vaishnaw, who serves as EVP of R&D and CMO at Alnylam Pharmaceuticals, tackles the challenges and opportunities in pursuing a new therapeutic modality from its inception. The Chief Medical Officer Summit is designed for CMOs and R&D Leadership from small to mid-size life science companies. The 6th Annual CMO Summit will be held May 7-8, 2018, in Boston.

1) The Effects of Orthostatic Hypotension on Cognition in Parkinson's Disease 2) What's Trending: Carotid surgery trials3) Topic of the Month: Neuromuscular topicsThis podcast for the Neurology Journal begins and closes with Dr. Robert Gross, Editor-in-Chief, briefly discussing highlighted articles from the print issue of Neurology. In the first segment, Dr. Matthew Barrett interviews Dr. Justin Centi about his paper on the effects of orthostatic hypotension on cognition in Parkinson disease. Dr. Andy Southerland is interviewing Dr. Seemant Chaturvedi for our “What's Trending” feature of the week about urgency for carotid surgery trials. In the next part of the podcast, Dr. Ted Burns interviews Dr. Michelle Mauermann about neuromuscular topics. Disclosures can be found at www.neurology.org.DISCLOSURES: Dr. Barrett has received research support from Axovant Sciences, Inc., the Virginia Center of Alzheimer's and Related Diseases, and the Department of Defense.Dr. Centi has been an employee of Commonwealth Psychology Associates LLC and Harvard Medical School/Partners Consortium in Neuropsychology; and has received research support from NIH-NINDS.Dr. Southerland serves as Podcast Deputy Editor for Neurology; receives research support from the American Heart Association-American Stroke Association National Clinical Research Program, American Academy of Neurology, American Board of Psychiatry and Neurology, Health Resources Services Administration and the NIH; has a provisional patent application titled: “Method, system and computer readable medium for improving treatment times for rapid evaluation of acute stroke via mobile telemedicine;” and gave legal expert review. Dr. Chaturvedi has been a consultant for Merck; has served on the executive committee of ACT I study and CREST 2 study; serves on the editorial boards of Neurology, the Journal of Stroke & Cerebrovascular Disease, Stroke, and NEJM Journal Watch Neurology; has received research support from Boehringer-Ingelheim, NINDS, and the FDA; and has received compensation for expert witness testimony.Dr. Burns serves as Podcast Editor for Neurology®; and has received research support for consulting activities with UCB, CSL Behring, Walgreens and Alexion Pharmaceuticals, Inc. Dr. Mauermann serves on the editorial board of Mayo Clinic Proceedings; receives publishing royalties for book Autonomic Neurology; and receives research support from Ionis Pharmaceuticals, Inc. and Alnylam Pharmaceuticals.All other participants have no disclosures.

In this episode, we take a look back at a historic summer that saw the National Hemophilia Foundation's (NHF) Annual Meeting and the World Federation of America's (WFH) World Congress convene in Orlando for the largest gathering ever of people with bleeding disorders. In addition to featuring some conference related content, our Like Share Comment segment highlight a story on Elijah Warren, a high school swimming star with hemophilia as well as a recent article from John Hopkins University on the power of humor in healthcare. Finally, we close this month's episode with a remembrance of Matt Stinger, a community member and advocate who passed unexpectedly this month at 33 years old. Headlines Novo Nordisk’s B-HERO-S study has found, to the surprise of some, that many patients with mild or moderate hemophilia B in the United States experience their condition negatively impacting their career, education, and recreational activities- something that can be overlooked in those affected by mild or moderate hemophilia as opposed to severe. Link. High schooler Federico Parres is one of five team members on the VEX Robotics World Championship Team F, responsible for creating Caerus-7, a ball-flinging robot. Federico lives with severe hemophilia A and plans on becoming an engineer one day. Link. Bayers’ Hemophilia Awards Program recently announced 16 physicians and researchers from 11 countries who will receives grant money totaling about two million dollars, including 6 individuals from the United States. This is the fourteenth year Bayer has run this program. Link A study published in the European journal Haematologica examines a new potential model for how to treatment patients with hemophilia A who undergo surgery. Something I personally find interesting given our ongoing need to learn more about aging with hemophilia. Link The FDA has approved a new needleless reconstitution device called Baxject III for Shire’s long-lasting factor FVIII therapy Adynovate. For those familiar, it is a very similar device to that which is found in Advate. Link Two medical advisory updates have been announced by the National Hemophilia Foundation regarding existing products: NHF Medical Advisory 419 (four-one-nine) states that Bayer has announced a voluntary recall of two lots of Kogenate FS for a loss of potency. The lot numbers are 270TN1C and 270R978. For more information or to arrange for an exchange please call the Recall Processing Center at 855-838-5782. Link And NHF Medical Advisory 420 (four-two-zero) states that CSL Behring has announced a voluntary recall of two lots of Helixate FS for a loss of potency. Those lot numbers are 270TN1G and 270R979. For more information or to arrange an exchange for these products, call CSL Behring’s Customer Service at 1800-683-1288. Link Product Related Headlines (not mentioned in the show) Genentech/Roche has released encouraging Phase 1/2 extension results for their investigational subcutaneous medicine, emicizumab-previously referred to as ACE910. Link The Netherlands-based uniQure, a company self-described as a leader in human gene therapy, announced encouraging clinical data from their ongoing Phase 1/2 trail of AMT-060, an investigational gene therapy for patients with hemophilia B. Link​ BioMarin has presented positive interim data from a Phase 1/2 clinical trial of BMN 270, an investigational gene therapy for people severe hemophilia A. The company has announced intent to begin another study with an eye toward regulatory approval in both the US and Europe. Link CSL Behring’s hemophilia B therapy Idelvion shows promise after encouraging Phase 3 trial results. Link The company also posted positive results from a Phase III study from its FDA approved, hemophilia A treatment AFSTYLA, unique for being the first and only single-chain recombinant product available in the US. Spark Therapeutics and Pfizer received Breakthrough Therapy Status from the FDA for expedited development of a hemophilia B gene therapy treatment SPK-9001. The treatment is currently also undergoing a Phase 1/2 clinical trial. Link. Alnylam Pharmaceuticals announced positive results of a Phase 1 trial for their subcutaneous therapy fitusiran, designed to treat patients with both hemophilia A and B, with or without inhibitors. Link. Like Share Comment Segment NHF Daily, a digital recap of Annual Meeting. Link WFH The Congress Daily. Link High school swimming star Elijah Warren. Link John Hopkins University article on therapeutic humor. Link BloodStream on Social BloodStream Facebook Page BloodStream Twitter Account BloodStream Host Patrick James Lynch on Twitter and Instagram.