Podcasts about Sarepta

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Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Eli Lilly is facing significant drug pricing pressure, with issues ranging from the pricing of their weight loss pill Orfoglipron to market battles with competitors like Novo Nordisk. Despite the challenges, Lilly's executives see opportunities for their products in the real world. Jazz Pharmaceuticals received FDA approval for Dordaviprone, making it the first new treatment for an ultra-rare brain tumor. In other news, Sarepta beat Q2 estimates despite not holding an investor call, and Trilink Biotechnologies is offering process development services for nucleic acid therapeutic development. Lotte Biologics is expanding its global CDMO operations, while a big pharma-backed mRNA startup raised $153 million in funding. Novo Nordisk's new CEO is focusing on obesity and diabetes, Pfizer is in talks with Trump on most favored nation drug pricing, and Bayer expects more layoffs. These developments in the pharma and biotech world showcase the ongoing challenges and opportunities in the industry. Stay tuned for more updates on the latest news and trends in this rapidly evolving sector.

This episode of GEN's Touching Base spotlights the unexpected relationship between breathing low oxygen and restoration of neuron function in a mouse model of Parkinson's disease. In addition, a single gene therapy injected at birth now offers lasting protection from HIV in infant rhesus macaques. Rounding out our episode, we discuss key factors shaping this year's A-List of Top 10 U.S. Biopharma Clusters and recap the regulatory conflict surrounding Sarepta Therapeutics' Duchenne muscular dystrophy (DMD) gene therapy, Elevidys®—including the departure of Vinay Prasad, MD, the FDA official behind the agency's brief get-tough approach.Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Fay Lin, PhD, and Uduak Thomas for a discussion of the latest biotech and biopharma news. Listed below are links to the GEN stories referenced in this episode of Touching Base:GLP-1 Drug Semaglutide Linked to Reduced Muscle Strength in MiceGEN, August 6, 2025Out of Thin Air: Hypoxia Reverses Parkinson's Symptoms in MiceBy Fay Lin, PhD GEN, August 6, 2025Early Gene Therapy Provides Lasting HIV Protection in Newborn PrimatesGEN, July 30, 2025StockWatch: As Prasad Exits FDA, Analysts See Benefit for Sarepta, CGT StocksBy Alex Philippidis, GEN Edge, August 3, 2025Sarepta to Resume Shipping DMD Gene Therapy to Ambulant PatientsBy Alex Philippidis, GEN Edge, July 28, 2025Sarepta Under Scrutiny, AstraZeneca's Big Bet, and AI vs. IDRsBy Alex Philippidis, Uduak Thomas, Fay Lin, PhD, Corinna Singleman, PhD, GEN, July 25, 2025About Face: Sarepta to Pause Elevidys Shipments TemporarilyBy Alex Philippidis, GEN Edge, July 21, 2025Touching Base PodcastHosted by Corinna Singleman, PhDBehind the BreakthroughsHosted by Jonathan D. Grinstein, PhDThe State of Biotech Summit Registration Hosted on Acast. See acast.com/privacy for more information.

It's nearly impossible to discuss the private health insurance landscape in America without referencing the killing of UnitedHealthcare CEO Brian Thompson last December.The shooting unleashed a wave of antipathy directed at insurers and brought deep-seeded, longstanding grievances against these for-profit health entities to the fore.In the intervening months, UnitedHealth Group and the broader insurer class have suffered significant damage not only to their respective bottom lines, but also their corporate reputations.Given the current state of play, we wanted to hear how medical marketers are navigating this uniquely challenging situation.Reporter Heerea Rikhraj speaks with Shannon Langrand, CEO of her namesake agency, about how health insurers are being marketed in 2025 as well as what challenges are lingering in the payer space.For our Trends segment, we parse through the Sarepta Therapeutics saga and pull out some key takeaways. Step into the future of health media at the MM+M Media Summit on October 30th, 2025 live in NYC! Join top voices in pharma marketing for a full day of forward-thinking discussions on AI, streaming, retail media, and more. Explore the latest in omnichannel strategy, personalization, media trust, and data privacy—all under one roof. Don't wait—use promo code PODCAST for $100 off your individual ticket. Click here to register! AI Deciphered is back—live in New York City this November 13th.Join leaders from brands, agencies, and platforms for a future-focused conversation on how AI is transforming media, marketing, and the retail experience. Ready to future-proof your strategy? Secure your spot now at aidecipheredsummit.com. Use code POD at check out for $100 your ticket! Check us out at: mmm-online.com Follow us: YouTube: @MMM-onlineTikTok: @MMMnewsInstagram: @MMMnewsonlineTwitter/X: @MMMnewsLinkedIn: MM+M To read more of the most timely, balanced and original reporting in medical marketing, subscribe here.Music: “Deep Reflection” by DP and Triple Scoop Music.

Center for Drug Evaluation and Research Head George Tidmarsh will oversee the Center for Biologics Evaluation and Research on an acting basis after Vinay Prasad's abrupt departure hours after the recording of last week's episode of The Weekly. While the situation is being billed as temporary, rumors are swirling that structural changes may be afoot at the FDA with Commissioner Marty Makary looking for better drug, biologic alignment.Meanwhile, the agency has come under criticism for another recent decision—one apparently driven by CDER's Oncology Center of Excellence director Richard Pazdur: the rejection of Replimmune's advanced melanoma drug, RP1. According to reporting by multiple outlets, Pazdur opposed the consensus opinion of CBER staff to approve the drug. The research team behind Replimmune's Phase III study penned an open letter to the FDA on Friday responding to the issues outlined in the agency's complete response letter. Meanwhile, the FDA's Sarepta saga continues, highlighting “unprecedented” FDA leaks and a veritable communications disaster.On the business side of biopharma, Q2 earnings continue to unfurl, with Pfizer, Vertex, BioNTech, Merck and Moderna all reporting this past week. Merck's $3 billion savings push has claimed 6,000 jobs, contributing to a brutal July that saw the entire biopharma industry axe 7,900 employees, a 487% year-over-year increase, based on BioSpace tallies. Meanwhile, Pfizer CEO Albert Bourla confirmed he has been in direct contact with President Donald Trump to negotiate a path forward on Most Favored Nation drug pricing after the president sent letters to 17 Big Pharma companies—and posted on his Truth Social platform—asking them to comply with the policy within 60 days or face potential unspecified consequences.In other policy news, the Centers for Medicare and Medicaid Services is reportedly considering coverage of GLP-1 drugs for weight management and obesity—reviving a Biden era proposal the Trump admin scrapped earlier this year.Finally, in BioPharm Executive this week, we have a special report on the situation in China as international drugmakers swoop into the region to find new drug candidates, while other companies build their therapeutic farm systems from incubators and venture arms. And check out BioSpace's brand new Manufacturing Brief, where we bring you the latest news and analysis in the area of biopharma manufacturing, starting with a feature on how to make cell and gene therapies commercially viable.

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. BioNTech is focusing on infectious diseases as vaccine rates drop, with expectations that the US COVID-19 vaccination rate will decrease slightly. Glioblastoma research is seeing increased funding and interest from companies like Merck and Jazz Pharmaceuticals. The FDA's rejection of Replimmune's melanoma drug, despite staff consensus, has caused a drop in the company's stock. Sarepta and Capricor learned of regulatory decisions through media leaks, causing confusion for patients and families. Trilink Biotechnologies offers process development services for nucleic acid therapeutic development. Lotte Biologics is expanding globally, Allogene is discontinuing an immunosuppressive antibody after a patient death, and NIH funding cuts are impacting biotech. Pharmas are increasing investment in AI despite challenges in the industry. Trump urges pharma companies to address drug pricing, while Merck plans to cut 6,000 jobs to save $3 billion.

Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy Elevidys and growing scrutiny over the company’s transparency following multiple patient deaths. In this week’s episode of "The Top Line," we explore Sarepta’s brief standoff with the FDA over Elevidys, a short-lived marketing pause, and the regulator’s surprise reversal on the treatment. Fierce Pharma’s Fraiser Kansteiner and Angus Liu sit down with Fierce Biotech’s Gabrielle Masson to recap the story and discuss the broader implications the Elevidys saga may have for gene therapy, the FDA and the pharmaceutical industry. To learn more about the topics in this episode: Sarepta pivots to siRNA and lays off 500 staffers as Elevidys gets box warning Sarepta LGMD trials all hit by FDA hold amid newly surfaced safety concerns over gene therapy Sarepta, bowing to FDA pressure, pauses shipments of Duchenne gene therapy Elevidys FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients Analysts demand transparency after Sarepta's roundabout disclosure of 3rd patient death Roche won't throw in the towel after DMD gene therapy Elevidys' rebuff in Europe See omnystudio.com/listener for privacy information.

Empaveli approved to treat two rare kidney diseases; Elevidys available again for ambulatory patients; Mounjaro shows benefit in cardiovascular disease; wearable cardiac monitor gets clearance; upadacitinib trial shows hair regrowth in individuals with alopecia areata.

Howie and Harlan discuss Dr. Vinay Prasad's departure from the FDA after a backlash against his decisions on Sarepta's Duchenne muscular dystrophy gene therapy. Also examined: AI in clinical settings, changes in NIH grantmaking, and the 60th anniversary of Medicare and Medicaid. Links: Sarepta and the FDA “STAT breaks down the confusing, heartbreaking Sarepta saga” “Sarepta Therapeutics' Duchenne therapy faces ‘arduous' path back to market, senior FDA official says” “For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak” “FDA launches probe into new Elevidys death as Sarepta, Roche stress gene therapy not at fault” “FDA Recommends Removal of Voluntary Hold for Elevidys for Ambulatory Patients” “FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients” “Controversial FDA official Dr. Vinay Prasad departs agency” “Top F.D.A. Vaccine Official Resigns, Citing Kennedy's ‘Misinformation and Lies'” “Vinay Prasad Is a Bernie Sanders Acolyte in MAHA Drag” “Timeline: The Rise and Fall of Vioxx” “What have we learnt from Vioxx?” THC in pizza dough “C.D.C. Ties 85 Cases of THC-Related Symptoms to Wisconsin Restaurant” “Tetrahydrocannabinol Intoxication from Food at a Restaurant — Wisconsin, October 2024” NIH funding “NIH is shrinking the number of research projects it funds due to a new Trump policy” NIH: Supporting Fairness and Originality in NIH Research Applications “Fearful of AI-generated grant proposals, NIH limits scientists to six applications per year” “Trump budget draft proposes NIH consolidation and 40% spending cut” Raw milk “Outbreak of Salmonella Typhimurium Infections Linked to Commercially Distributed Raw Milk — California and Four Other States, September 2023–March 2024” “The Dangers of Raw Milk: Unpasteurized Milk Can Pose a Serious Health Risk” AI in healthcare update “Ambience Healthcare Announces $243 Million Series C to Scale its AI Platform for Health Systems” “As ambient scribes face off, Doximity lures doctors with a free option” Aidoc: “AI Empowering Radiologists” “Clinical Implementation of a Combined Artificial Intelligence and Natural Language Processing Quality Assurance Program for Pulmonary Nodule Detection in the Emergency Department Setting” “Large Language Models as an Inexpensive and Effective Extra Set of Eyes in Radiology Reporting” “Current Trends in Remote and Flexible Work Options in Radiology and Perception of Impact on Radiologist Well-being” Medicare and Medicaid 60th anniversary CMS' program history “Medicare and Medicaid turn 60 – and face historic cuts decades in the making” UnitedHealth and Medicare Advantage “UnitedHealth says 2025 earnings will be worse than expected as high medical costs dog insurers” “UnitedHealthcare to exit certain Medicare Advantage markets as costs balloon, impacting 600K enrollees” “Novo Nordisk slides further on US competition, new CEO concerns” “Dems Must Really Fix Medicaid, Not Just Undo Trump's Damage to It”   Learn more about the MBA for Executives program at Yale SOM. Email Howie and Harlan comments or questions.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.Vinay Prasad's departure from CBER has analysts anticipating a more traditional successor, while GSK streamlines its pipeline and pledges billions in US investments. Susan Monarez is now the confirmed CDC director, and the top biopharma venture capital raises of H1 2025 are summarized. Despite challenges like layoffs and market fluctuations, GSK remains committed to investing in the US despite tariffs. Other headlines include Madrigal's potential $2 billion investment, Biogen and Eisai's Alzheimer's drug update, and Novo Nordisk's new leadership. Trilink Biotechnologies is offering self-amplifying RNA constructs for potential therapeutic advancements.AI biotech companies have secured substantial funding in the first half of 2025, with continued investment pouring into startups within the industry. The top five biopharma venture capital raises of this period are highlighted. There is confusion surrounding Ira's definition of 'drug,' potentially hindering companies from pursuing approval for new formulations and indications. Vinay Prasad's departure from the FDA's Center for Biologics Evaluation and Research, following controversies related to Sarepta, has raised concerns among developers. Despite challenges, four biotech companies are successfully launching their products independently.Over 260 million people are impacted by rare diseases, emphasizing the need for faster evidence generation through global real-world data. GSK's commitment to investing in the US, Merck's cost-cutting measures to support its launch schedule, and Novo Nordisk's new leadership are also highlighted. The FDA has updated regulations regarding Sarepta's DMD gene therapy, making it available for ambulatory patients. Adaptimmune anticipates significant staff reductions following a cell therapy asset sale. Stay tuned for more updates on the latest developments in the biopharma industry.

Howie and Harlan discuss Dr. Vinay Prasad's departure from the FDA after a backlash against his decisions on Sarepta's Duchenne muscular dystrophy gene therapy. Also examined: AI in clinical settings, changes in NIH grantmaking, and the 60th anniversary of Medicare and Medicaid. Links: Sarepta and the FDA “STAT breaks down the confusing, heartbreaking Sarepta saga” “Sarepta Therapeutics' Duchenne therapy faces ‘arduous' path back to market, senior FDA official says” “For many Duchenne families, halt to gene therapy is heartbreak upon heartbreak” “FDA launches probe into new Elevidys death as Sarepta, Roche stress gene therapy not at fault” “FDA Recommends Removal of Voluntary Hold for Elevidys for Ambulatory Patients” “FDA takes U-turn on Sarepta's Elevidys, backing Duchenne gene therapy again in ambulatory patients” “Controversial FDA official Dr. Vinay Prasad departs agency” “Top F.D.A. Vaccine Official Resigns, Citing Kennedy's ‘Misinformation and Lies'” “Vinay Prasad Is a Bernie Sanders Acolyte in MAHA Drag” “Timeline: The Rise and Fall of Vioxx” “What have we learnt from Vioxx?” THC in pizza dough “C.D.C. Ties 85 Cases of THC-Related Symptoms to Wisconsin Restaurant” “Tetrahydrocannabinol Intoxication from Food at a Restaurant — Wisconsin, October 2024” NIH funding “NIH is shrinking the number of research projects it funds due to a new Trump policy” NIH: Supporting Fairness and Originality in NIH Research Applications “Fearful of AI-generated grant proposals, NIH limits scientists to six applications per year” “Trump budget draft proposes NIH consolidation and 40% spending cut” Raw milk “Outbreak of Salmonella Typhimurium Infections Linked to Commercially Distributed Raw Milk — California and Four Other States, September 2023–March 2024” “The Dangers of Raw Milk: Unpasteurized Milk Can Pose a Serious Health Risk” AI in healthcare update “Ambience Healthcare Announces $243 Million Series C to Scale its AI Platform for Health Systems” “As ambient scribes face off, Doximity lures doctors with a free option” Aidoc: “AI Empowering Radiologists” “Clinical Implementation of a Combined Artificial Intelligence and Natural Language Processing Quality Assurance Program for Pulmonary Nodule Detection in the Emergency Department Setting” “Large Language Models as an Inexpensive and Effective Extra Set of Eyes in Radiology Reporting” “Current Trends in Remote and Flexible Work Options in Radiology and Perception of Impact on Radiologist Well-being” Medicare and Medicaid 60th anniversary CMS' program history “Medicare and Medicaid turn 60 – and face historic cuts decades in the making” UnitedHealth and Medicare Advantage “UnitedHealth says 2025 earnings will be worse than expected as high medical costs dog insurers” “UnitedHealthcare to exit certain Medicare Advantage markets as costs balloon, impacting 600K enrollees” “Novo Nordisk slides further on US competition, new CEO concerns” “Dems Must Really Fix Medicaid, Not Just Undo Trump's Damage to It”   Learn more about the MBA for Executives program at Yale SOM. Email Howie and Harlan comments or questions.

The morning after the FDA's vaccine regulator Dr. Vinay Prasad announced his resignation, FDA Commissioner Dr. Marty Makary shares the agency's position on the measles vaccine, Sarepta's Duchenne therapy, and the addictive kratom-derived compound OH-7. Booking Holdings CEO Glenn Fogel discusses the impact of geopolitics on travel to the U.S. While Canadians and Europeans are still traveling, the operator of Agoda, Priceline, and Kayak says they're going to Mexico and Asia, instead of the U.S. Plus, Wall Street remembers the lives lost in Monday's shooting, the first tsunami waves from Russia's 8.8 earthquake have hit Hawaii's shores, and CNBC's Eamon Javers reports on his conversation with Treasury Secretary Scott Bessent about trade talks with China. Eamon Javers - 07:24Dr. Marty Makary - 18:51Glenn Fogel - 36:48 In this episode:Eamon Javers, @EamonJaversJoe Kernen, @JoeSquawk Becky Quick, @BeckyQuickAndrew Ross Sorkin, @andrewrsorkinKatie Kramer, @Kramer_Katie

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Novo Nordisk has announced Maziar Mike Doustdar as its new CEO, along with a 5% reduction in its 2025 sales guidance. This decision comes as a response to the slowed growth of its semaglutide franchise in the U.S. Senate Democrats are currently looking into HHS Secretary RFK Jr.'s controversial move to dismiss all 17 members of the CDC's vaccine advisory committee. Astrazeneca CEO Pascal Soriot emphasized the importance of global collaboration in pharmaceutical research and development. Merck has allocated $3 billion to support upcoming product launches. In a surprising turn of events, the FDA has reversed its decision on Sarepta, now permitting DMD gene therapy for ambulatory patients.

The Sarepta saga continued into another week as the FDA recommended that the voluntary hold on the company's Duchenne muscular dystrophy gene therapy be lifted for ambulatory patients, after determining that the death of an 8-year-old Brazilian Duchenne patient who had received Elevidys' was not caused by the drug.  Sarepta's stock has swung wildly and its transparency questioned after it elected not to reveal the death of a third patient—a participant in a trial of a gene therapy for limb girdle muscular dystrophy—during a business update last week.    Speaking of entities—or individuals—who have trouble staying out of the news, Health Secretary Robert F. Kennedy Jr. plans to dissolve the U.S. Preventive Services Task Force because it is “too woke.” Also on Monday, Kennedy addressed what he called the “broken” vaccine injury compensation program. Without offering details, he vowed to “fix” the U.S.'s VICP and return it to its “original Congressional intent.”   On the business side of the biopharma house, Q2 earnings are in full swing, with AstraZeneca announcing estimate-beating numbers and CEO Pascal Soriot saying the world “needs to share” in global pharma R&D, while Merck cut $3 billion to support an aggressive launch schedule. Meanwhile, a week ahead of its own earnings report, Novo Nordisk named a new CEO and lowered its 2025 sales guidance for the second time this year.   In clinical development, the Alzheimer's Association Annual Conference is underway in Toronto, with Roche's trontinemab the standout so far. In a Phase Ib/IIa trial, the next-gen anti-amyloid antibody rapidly cleared amyloid from the brains of patients with Alzheimer's disease after just seven months—besting the 18-month timeframe for Biogen and Eisai's Leqembi and Eli Lilly's Kisunla. While Leqembi and Kisunla have shown some progress is slowing down the progression of Alzheimer's, their effect size is modest and they don't work for all patients—leaving plenty of room for symptomatic treatments, such as those being developed by Bristol Myers Squibb and Acadia Pharmaceuticals. The space is gearing up for several readouts, for both symptomatic and disease-modifying therapies alike.   And in BioPharm Executive this week, we dig into the top VC rounds so far this year and highlight a few scrappy biotechs walking the solo road.  

On this week's episode, Josh Schimmer, Chris Garabedian, Sam Fazeli, Yaron Werber and guest Adam Feuerstein dive into breaking news, including Sarepta's recent updates -- layoffs, a black box warning, and pipeline reorganization -- and the juxtaposition of Amylyx's post-bariatric hypoglycemia and GLP-1 inhibitor following a webinar update at ENDO 2025. In regulatory news, the group discusses the evolving FDA under Makary's leadership, examining agency morale, rapid review processes for favorable drug pricing, and recent CRLs including Ultragenyx. The discussion then explores what these changes mean for CBER and Peter Marks' accelerated approval pathway for orphan disorders, alongside the ODAC panel for GSK's belantamab for multiple myeloma and the broader space. Market sentiment analysis reveals encouraging signs with the second quarter and first half reports on VC investment showing a clear uptick, along with additional positive feedback from a CEO forum on the FDA's listening tour. The group shares some interesting stats around M&A so far this year, demonstrating strong momentum and a possible trajectory to possibly beat, or at least meet, the 2020 numbers. Data updates include, lung cancer survey insights, DiaMedica's preeclampsia results, obesity updates from Hengrui/Kailera and Chinese biotech. The episode wraps with an overview of Novartis and J&J earnings reports. *This episode aired on July 18, 2025.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. GSK has entered a partnership with Hengrui worth up to $12 billion, focusing on the COPD candidate HRS-9821. The FDA's new voucher policy has caused confusion among experts, as it lacks clear definition and was announced without public input. The American Medical Association urges RFK Jr. to maintain the preventive task force, and Sarepta denies a patient death is linked to Elevidys as the FDA launches a probe. Biopharma companies are leaning towards holistic treatments for Alzheimer's, while Atai-partnered schizophrenia drug fails in a mid-stage trial. Boehringer partners with Irish startup Re-Vana in a $1 billion+ eye deal.As the Alzheimer's space becomes more competitive, biopharma companies are focusing on holistic treatments beyond disease-modifying drugs like Leqembi and Kisunla. Companies such as Bristol Myers Squibb, Acadia, Otsuka, and Lundbeck are renewing their search for symptomatic treatments. Five upcoming data drops could potentially lead to more effective therapies for Alzheimer's. Sarepta Therapeutics is facing challenges due to safety concerns surrounding its gene therapies, leading to a drop in stock value. The FDA's new voucher program aims to offer accelerated pathways for drugs meeting certain criteria, but experts criticize the lack of transparency and public input in the policy's announcement. Sarepta's future is uncertain as the FDA considers a new study for Elevidys, and the EU issues a negative opinion on the drug. Other news includes delays in the FDA decision on GSK's Blenrep, AstraZeneca's PIII win with nanobody treatment for myasthenia gravis, and the removal of thimerosal from influenza vaccines. George Tidmarsh has been appointed as the new chief of FDA's CDER.

Audio roundup of selected biopharma industry content from Scrip over the business week ended July 25, 2025. In this episode: Sanofi's Vicebio buy; Sarepta halts US Elevidys shipments; Novartis warning over Europe; US CRL for Genentech's Columvi; and an interview with Novavax. https://insights.citeline.com/scrip/podcasts/scrips-five-must-know-things/quick-listen-scrips-five-must-know-things-U4IN5X7DRVFLVIBJ4Q72VTAJUY/ This episode was produced with the help of AI text-to-voice and voice emulation tools. Playlist: soundcloud.com/citelinesounds/sets/scrips-five-must-know-things

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. The European Medicines Agency's CHMP did not recommend approving Elevidys for ambulatory patients with Duchenne muscular dystrophy, dealing a blow to Sarepta. FDA is rumored to request new data for Elevidys, leading to uncertainty as FDA considers a new study for the drug. In other news, Eli Lilly commits $856 million to Gate Bioscience for a new class of medicines, while Rocket trims headcount and pipeline focus. Roche also drops an early obesity asset as layoffs continue in the biopharma industry, with companies like Adicet optimizing their pipelines. Novartis makes a billion-dollar drug discovery deal with Matchpoint, and the FDA opens a pilot run of the commissioner voucher program.Stay tuned for more updates on the latest developments in the pharmaceutical and biotech world.

The end of a huge week for earnings and record highs on the S&P: Carl Quintanilla, Sara Eisen, and David Faber reacted to breaking commentary from President Trump top of the hour, before getting Apollo Global Chief Economist Torsten Slok's take… who's warning of stagflation ahead tied to tariffs: arguing there's “no free lunch”. Big Tech a key part of the recent rally – longtime investor Dan Niles broke down his top picks ahead of results next week from Apple, and why he sees clouds on the horizon come September. Plus: a number of individual stock movers… The team discussed what's driving huge declines in shares of Charter and Sarepta, Volkswagen's latest warning on tariffs – and what it means for the rest of the automakers, and new numbers out of one luxury retailer who's down big on the year.   

Pink Sheet Executive Editor Derrick Gingery, Senior Writer Sarah Karlin-Smith and Editor-in-Chief Nielsen Hobbs discuss the issues awaiting George Tidmarsh, the new director of the US Food and Drug Administration's Center for Drug Evaluation and Research (:24), as well as the next steps for the agency and Sarepta amid the ongoing problems with its gene therapy Elevidys (delandistrogene moxeparvovec) (23:49). More On These Topics From The Pink Sheet Will Tidmarsh's Industry Experience Translate As US FDA's Chief Drug Regulator?: https://insights.citeline.com/pink-sheet/agency-leadership/us-fda/breaking-news-pharma-ceo-george-tidmarsh-named-director-of-us-fdas-drug-center-2HXJJCPAIVB2RFNGDDU5NZVNQQ/ Business Background Of New CDER Director George Tidmarsh: https://insights.citeline.com/pink-sheet/agency-leadership/us-fda/business-background-of-new-cder-director-george-tidmarsh-BROCYQDUYFE3LIQDJ2JNBDWD6Y/ US FDA Cancer Reviewers Heading For The Exits, Potentially Impacting Review Timelines: https://insights.citeline.com/pink-sheet/agency-leadership/us-fda/us-fda-cancer-reviewers-heading-for-the-exits-potentially-impacting-review-timelines-YMOFTXDFDZBGVGBIKFXHJY5XTY/ US FDA's Biologics Center Departures Grew Before More Than 100 RIFs Exited: https://insights.citeline.com/pink-sheet/agency-leadership/us-fda/us-fdas-biologics-center-departures-grew-before-more-than-100-rifs-exited-2LR3ZFYOBND3DLIWXSFFWWLFYE/ After Sarepta Reversed Course On Elevidys, Could An Adcomm Be Next?: https://insights.citeline.com/pink-sheet/product-reviews/post-marketing-regulation-and-studies/after-sarepta-reversed-course-on-elevidys-could-an-adcomm-be-next-ZA5ODQB3DJF4LBDFODMB52DM3M/ Sarepta Refuses To Stop Elevidys Shipments After Third Death Linked To Gene Therapy Vector: https://insights.citeline.com/pink-sheet/advanced-technologies/cell-and-gene-therapies/us-fda-asks-sarepta-to-stop-elevidys-shipments-after-third-death-linked-to-gene-therapy-vector-GLJ2YJDJGNBPLD3SSKKE6UVM34/

Elevidys shipments paused following third death; FDA committee votes against brexpiprazole combo for PTSD; Rapiblyk now available for SVT; sleep apnea pill looks promising; JUUL e-cigarettes gain marketing authorization.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.Roche has dropped an early-stage obesity asset, CT-173, citing lack of competitiveness. Novartis has entered into a drug discovery deal with Matchpoint Therapeutics, acquiring global rights on all molecules for several inflammatory diseases. AstraZeneca claims a Phase III win with its nanobody treatment for myasthenia gravis. Second-quarter earnings season is approaching, and biotechs to watch include Sarepta and others facing challenges in the biopharma industry. Genentech downsizes as priorities shift, and GSK's comeback for Blenrep is on pause as the FDA delays its decision. The FDA's lack of transparency has tarnished Sarepta's reputation after patient deaths triggered an FDA battle. Opportunities in the industry include roles like Quality Specialist at CSL and Clinical Research Physician at Eli Lilly and Company.

Sarepta has temporarily paused shipments of Elevidys, its FDA-approved treatment for Duchenne muscular dystrophy. In this episode of GEN's Touching Base, we discuss the company's response to the tragedies associated with its DMD therapy as well as with a new therapy for limb-girdle muscular dystrophy. Also in this episode, big updates from AstraZeneca including a $50 billion investment in U.S. manufacturing and R&D, a heartwarming story about preventing mitochondrial disease involving eight babies from the U.K., and from the lab of Nobel Prize winner David Baker, PhD, AI that designs drugs for previously “undruggable” proteins.Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Fay Lin, PhD, and Uduak Thomas for a discussion of the latest biotech and biopharma news.Listed below are links to the GEN stories referenced in this episode of Touching Base:About Face: Sarepta to Pause Elevidys Shipments TemporarilyBy Alex Philippidis, GEN Edge, July 21, 2025StockWatch: Sarepta Shares Nosedive after LGMD Gene Therapy Patient DiesBy Alex Philippidis, GEN Edge, July 20, 2025Sarepta Axes 500, 36% of Workforce, in Restructuring after DMD Patient DeathsBy Alex Philippidis, GEN Edge, July 16, 2025AstraZeneca Commits $50B More to U.S. Manufacturing, R&D ProjectsBy Alex Philippidis, GEN Edge, July 22, 2025AstraZeneca's New $300M Plant Provides Control of Cell Therapy ProductionBy Gareth John Macdonald, GEN, May 14, 2025 Beyond Baby KJ: Next Steps in Manufacturing Genome Editing CuresGEN Live, July 30, 2025Mitochondrial Disease Milestone: Eight Babies Born Free of Disease via Pronuclear TransferBy Julianna LeMieux, PhD GEN, July 16, 2025Undruggable No More: AI Hits Disordered Proteins, Unlocks Therapy TargetsBy Fay Lin, PhD GEN, July 18, 2025 Hosted on Acast. See acast.com/privacy for more information.

Howie and Harlan are joined by public health communicator Katelyn Jetelina for updates on COVID-19 and other issues, and to discuss how her emails to students and colleagues in the early days of the pandemic turned into a platform with global reach. Harlan looks at how AI is being used on both sides of the battle between providers and insurers over claims; Howie reports on a setback with a promising gene therapy for Duchenne muscular dystrophy. Links: Insurance Claims, AI, and Wearables “Elevance Health Reports Second Quarter 2025 Results” “Elevance lowers profit outlook, warns things will get worse for health insurers ““Blue KC wrongfully denied medical diagnoses, hospital alleges in AI-driven claims lawsuit” “Oscar Health cuts full-year guidance, estimates 2025 loss as ACA marketplace stumbles” Whoop “WHOOP Delivers Innovative Blood Pressure Insights for a Deeper Look at Your Well-Being” “Introducing Heart Screener: A smarter way to stay connected to your heart health” “Whoop says FDA is ‘overstepping its authority' with warning about blood pressure feature” FDA: WARNING LETTER, WHOOP, Inc. “RFK Jr. wants everyone to use wearables. What are the benefits, risks?” “Apple to Sell Watches With Blood-Oxygen Feature Disabled After Legal Setback” Your Local Epidemiologist Your Local Epidemiologist “Poll: Trust in Public Health Agencies and Vaccines Falls Amid Republican Skepticism” “Popular epidemiologist lays out future path of public health communication” Yale School of Public Health: PopHIVE Katelyn Jetelina: “NIH: The quiet engine of science is being dismantled” Katelyn Jetelina: “Covid-19 in pockets, sugar cane isn't better, ticks march on, rescission cuts (vs. everything else), bright spots, and more” Mayo Clinic: Norovirus infection Katelyn Jetelina: “The show must go on...” Katelyn Jetelina: “Harassment against scientists is out of control” Katelyn Jetelina: “Medicaid cuts: The how and why” CDC: H5 Bird Flu: Current Situatio CDC: Current Epidemic Trends (Based on Rt) for States” CDC: Measles Cases and Outbreaks Treating Duchenne Muscular Dystrophy Cleveland Clinic: Duchenne Muscular Dystrophy (DMD) “FDA approves Sarepta's Duchenne gene therapy for nearly all patients” “AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial” “Patient dies in Sarepta gene therapy trial, adding to safety concerns” “FDA Requests Sarepta Therapeutics Suspend Distribution of Elevidys and Places Clinical Trials on Hold for Multiple Gene Therapy Products Following 3 Deaths” “In surprise reversal, Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy” “Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks” Learn more about the MBA for Executives program at Yale SOM. Email Howie and Harlan comments or questions.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. The FDA has initiated a voucher program to address lower drug prices and boost domestic investment, with up to five companies being selected based on national priorities. Roche has halted shipments of Sarepta's Elevidys gene therapy after reports of patient deaths. Sarepta is under FDA scrutiny following a third patient death, while the agency welcomes a new top drug regulator and rejects multiple new drugs from entering the market. Insights from women in biopharma highlight the importance of resilience. Zhen Li, an engineer, is playing a crucial role in shaping the field of siRNA. Research suggests that GLP-1s may offer protection against neurodegenerative diseases. Dispatch Bio enters the scene with a substantial $216 million investment for universal CAR T therapy. Sarepta complies with an FDA request to suspend US shipments of Elevidys, raising concerns about transparency and impacting the company's reputation. Replimune experiences a significant drop in stock value following an FDA rejection, while AstraZeneca makes a bold $50 billion investment in US manufacturing to prepare for potential tariffs. Thank you for tuning in to Pharma and Biotech daily for the latest updates in the industry.

Howie and Harlan are joined by public health communicator Katelyn Jetelina for updates on COVID-19 and other issues, and to discuss how her emails to students and colleagues in the early days of the pandemic turned into a platform with global reach. Harlan looks at how AI is being used on both sides of the battle between providers and insurers over claims; Howie reports on a setback with a promising gene therapy for Duchenne muscular dystrophy. Links: Insurance Claims, AI, and Wearables “Elevance Health Reports Second Quarter 2025 Results” “Elevance lowers profit outlook, warns things will get worse for health insurers ““Blue KC wrongfully denied medical diagnoses, hospital alleges in AI-driven claims lawsuit” “Oscar Health cuts full-year guidance, estimates 2025 loss as ACA marketplace stumbles” Whoop “WHOOP Delivers Innovative Blood Pressure Insights for a Deeper Look at Your Well-Being” “Introducing Heart Screener: A smarter way to stay connected to your heart health” “Whoop says FDA is ‘overstepping its authority' with warning about blood pressure feature” FDA: WARNING LETTER, WHOOP, Inc. “RFK Jr. wants everyone to use wearables. What are the benefits, risks?” “Apple to Sell Watches With Blood-Oxygen Feature Disabled After Legal Setback” Your Local Epidemiologist Your Local Epidemiologist “Poll: Trust in Public Health Agencies and Vaccines Falls Amid Republican Skepticism” “Popular epidemiologist lays out future path of public health communication” Yale School of Public Health: PopHIVE Katelyn Jetelina: “NIH: The quiet engine of science is being dismantled” Katelyn Jetelina: “Covid-19 in pockets, sugar cane isn't better, ticks march on, rescission cuts (vs. everything else), bright spots, and more” Mayo Clinic: Norovirus infection Katelyn Jetelina: “The show must go on...” Katelyn Jetelina: “Harassment against scientists is out of control” Katelyn Jetelina: “Medicaid cuts: The how and why” CDC: H5 Bird Flu: Current Situatio CDC: Current Epidemic Trends (Based on Rt) for States” CDC: Measles Cases and Outbreaks Treating Duchenne Muscular Dystrophy Cleveland Clinic: Duchenne Muscular Dystrophy (DMD) “FDA approves Sarepta's Duchenne gene therapy for nearly all patients” “AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial” “Patient dies in Sarepta gene therapy trial, adding to safety concerns” “FDA Requests Sarepta Therapeutics Suspend Distribution of Elevidys and Places Clinical Trials on Hold for Multiple Gene Therapy Products Following 3 Deaths” “In surprise reversal, Sarepta Therapeutics says it will pause shipments of Duchenne gene therapy” “Sarepta to lay off about 500 employees after Duchenne gene therapy setbacks” Learn more about the MBA for Executives program at Yale SOM. Email Howie and Harlan comments or questions.

Just a few weeks ago, it seemed like Sarepta had weathered a spate of bad news, after two patients died from liver injuries from its Duchenne muscular dystrophy gene therapy Elevidys. Then came news of a third patient death. Last Wednesday, the company announced a major restructuring and 500-person layoff.  Then, in just a few days time, Sarepta Therapeutics went from enjoying a notable stock bump in response to that corporate update to its lowest price in nearly 10 years as it halts shipments of Elevidys. In addition to requesting the shipment hold, the FDA revoked the company's technology platform designation and paused all clinical trials for Sarepta's limb-girdle muscular dystrophy (LGMD) gene therapy. The turmoil was set in motion by media reports that a patient who received the LGMD treatment had died—a fact the company chose not to disclose during an investor call. In other news, the FDA's Center for Drug Evaluation and Research gets a new director in biotech veteran George Tidmarsh, also an adjunct professor of pediatrics and neonatology at Stanford University's School of Medicine. Tidmarsh enters the agency at a time of mass layoffs as well as voluntary departures. Meanwhile, Replimmune and Roche suffer FDA rejections as therapies from Otsuka/Lundbeck and GSK fail to earn adcomm support, as the bar for acceptable controls and demonstrations of efficacy continue to change under FDA commissioner Marty Makary and CBER director Vinay Prasad.  Finally, Big Pharmas continue to pump billions into U.S. manufacturing, with Biogen and AstraZeneca joining the list of companies to have made such pledges, pledging $2 billion and $50 billion, respectively. These latest announcements come as President Donald Trump reiterates that pharma-specific tariffs of up to 200% could come as soon as Aug. 1. 

Last week's public disclosure that a gene therapy from Sarepta had caused a third death led FDA to ask the company to stop distributing its DMD gene therapy Elevidys, a move the biotech has resisted. The deaths, and disputes between FDA and Sarepta, raise questions about the future of AAV gene therapies, as well as the future of FDA's platform technology designation. On the latest BioCentury This Week podcast, BioCentury's analysts unpack the events surrounding Sarepta's gene therapies and discuss how FDA, industry and patient groups should come together to learn the lessons from the tragic, avoidable deaths.BioCentury's analysts also assess Monday's appointment of Stanford professor and biotech executive George Tidmarsh to lead FDA's Center for Drug Evaluation and Research, and check in on the latest trends in venture financings. This episode of BioCentury This Week is sponsored by IQVIA Biotech.View full story: https://www.biocentury.com/article/656537#biotech #biopharma #pharma #lifescience #GeneTherapy #AAVTherapy #Sarepta #Elevidys00:01 - Sponsor Message: IQVIA Biotech02:03 - Gene Therapy17:59 - Leading CDER27:00 - Venture ReportTo submit a question to BioCentury's editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

Audio roundup of selected biopharma industry content from Scrip over the business week ended July 18, 2025. In this episode: – J&J delivers in Q2 despite Stelara drop; Sarepta cuts jobs as Elevidys slumps; AI is inevitable but needs to be applied appropriately; China's mid-cap companies thrive; and a look behind Glenmark's landmark trispecific deal. Article links: https://insights.citeline.com/scrip/podcasts/scrips-five-must-know-things/quick-listen-scrips-five-must-know-things-HYQOHZ42CNCQRPF6HCKGLTHUKM/ This episode was produced with the help of AI text-to-voice and voice emulation tools. Playlist: soundcloud.com/citelinesounds/sets/scrips-five-must-know-things

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.George Tidmarsh, a biopharma veteran and adjunct professor at Stanford's medical school, has been appointed as the new head of the FDA's Center for Drug Evaluation and Research (CDER). His extensive industry experience will be valuable in his new government position. Data secrecy among cell and gene therapy developers is hindering progress in the field, causing fragmentation, stalling innovation, and delaying access to treatments. Meanwhile, Sarepta is facing challenges with its elevidys shipments and has lost platform designation for its technology. At the same time, Roche's phase III trial in COPD has failed, impacting the market path for astegolimab. Layoffs are happening at companies like GSK, Sail, and BioNTech. Experts are exploring new ways to overcome barriers in cell therapy production.Data secrecy among cell and gene therapy developers continues to hinder progress and access to treatments. Acadia has introduced a new team and pipeline with ambitious goals. Patients are fighting for access to Brainstorm's ALS drug after promising real-world data. Moderna's withdrawal of its flu vaccine has left combination flu/COVID-19 vaccines in limbo. In other news, Sarepta is facing challenges with its DMD gene therapy, Ultragenyx's gene therapy for Sanfilippo syndrome is rejected by the FDA, GSK's Blenrep loses an adcomm vote, and BMS' anemia drug Reblozyl fails a Phase III trial. The FDA is experiencing layoffs and employee turnover amid an overhaul. Vinay Prasad overruled reviewers on Moderna's COVID-19 shot for kids. Upcoming events include a webinar on AI for real-world research and job opportunities in the biopharma industry. Readers are encouraged to provide feedback and suggest topics for future coverage.

On this week's episode, Chris Garabedian, Daphne Zohar, Sam Fazeli, Tess Cameron, Paul Matteis and Tim Opler share cautious optimism about the biotech market as IPOs and follow-ons gain momentum. Continuing on market sentiment, competitive pressure looms for small and mid-sized U.S. biotechs as the Chinese biotech market grows and big pharma continues to invest in these assets. Next, the group recapped #BIO2025, where sentiment was mixed due to sector uncertainty. In regulatory news, Daphne reflects on her time with Makary and Prasad at the FDA's listening tour, highlighting positive opportunities for biotech as the new administration takes shape. However, a report linking Makary to KalVista's rejection request stirred concerns. At ADA 2025, Lilly dominated the with positive results across its weight loss portfolio; Novo's data was also discussed. The group then covered Novo's cancellation of its partnership with HIMS due to concerns about the compounded versionof Wegovy. The conversation shifts to data, with Cidara's positive flu vaccine results, Compass' psilocybin data and Nektar's atopic dermatitis win. Additionally, Sarepta's Elevidys faces further safety questions and Capricor'scancelled AdCom raises worries of the FDA's flexibility.  The first ACIP meeting was also mentioned. On deals, Revolution Medicines' $2B deal with Royalty Pharma, Illumina's acquisition of SomaLogic and BlueRock's shutdown were overviewed. The episode concluded with a summary of the Royalty Pharma conference. *This episode aired on June 27, 2025.

Google riding high ahead of earnings, with potential for its longest win streak in years. Japan's election results have the Yen strengthening against the dollar. And Sarepta's slide continues as the drug developer rebukes the FDA's request to halt shipments of its gene therapy drug. Why one Wall Street analyst says the stock could head to zero. Fast Money Disclaimer  

Another morning of record highs on the S&P and NASDAQ ahead of a make-or-break week for stocks: Sara Eisen and Carl Quintanilla started the hour with a look at the major earnings coming this week (Alphabet, Tesla, Intel, and more), and what we've heard from key companies so far. Barclays Head of U.S. Equity Strategy arguing this will be the first quarter to show the impact of tariffs – but the headwinds just haven't shown up yet. Plus: a deep dive on new numbers out of Domino's with an analyst who calls the stock a buy, as their CFO says they've seen no geopolitical impact to their business.  Also in focus: hear from the CEO of the credit bureau FICO as his company comes under fire for being a “monopoly” – why he says it's just the most efficient way for the market to operate, how he's using AI in the business, and more… and the latest on the biggest movers of the day, from Sarepta to Apple to LVMH.Squawk on the Street Disclaimer

Aktien hören ist gut. Aktien kaufen ist besser. Bei unserem Partner Scalable Capital geht's unbegrenzt per Trading-Flatrate oder regelmäßig per Sparplan. Alle weiteren Infos gibt's hier: scalable.capital/oaws. Aktien + Whatsapp = Hier anmelden. Lieber als Newsletter? Geht auch. Das Buch zum Podcast? Jetzt lesen. Chevron darf endlich Hess kaufen. Exxon und CNOOC sind sauer. Union Pacific will Norfolk Southern kaufen. Behörden sind skeptisch. Invesco hofft auf ETF-Geld. Salzgitter & Sarepta schmieren ab. Saab boomt weiter. Block geht in S&P 500, Trump verklagt News Corp. Tetra Pak ist leider nicht an der Börse. Aber die Familie dahinter. Sie steckt ihre Milliarden in Linde (WKN:  A3D7VW), Givaudan (WKN:  938427), International Flavors & Fragrances (WKN:  853881), SIG Group (WKN:  A2N5NU) und Sensient Technologies (WKN:  864463). KI, Rüstung und ein KGV von unter 20. Fujitsu (WKN: 855182) macht's möglich. Leider machte die Software von Fujitsu in der Vergangenheit auch den ein oder anderen Skandal möglich. Diesen Podcast vom 21.07.2025, 3:00 Uhr stellt dir die Podstars GmbH (Noah Leidinger) zur Verfügung.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma and Biotech world. Sarepta Therapeutics experienced a significant drop in shares as the FDA considers requesting a halt to shipments of the Duchenne muscular dystrophy therapy Elevidys. This decision comes after a third patient death linked to the underlying platform. The company's stock plummeted by 37% on Friday afternoon as reports of the potential shipment stop circulated in the media. This news adds to the already turbulent week for Sarepta, which has been facing challenges in its gene therapy and siRNA biotech developments. ## Novartis announced positive results from a phase 3 trial of its drug Cosentyx in treating axial spondyloarthritis. The study showed that patients treated with Cosentyx had significant improvements compared to those on a placebo. These results further solidify Novartis' position in the market for treatments of autoimmune diseases. The company plans to submit the data to regulatory authorities for potential approval of this indication.Novartis revealed encouraging outcomes from a phase 3 trial of its drug Cosentyx for treating axial spondyloarthritis. The study demonstrated that patients who received Cosentyx experienced notable enhancements compared to those who were given a placebo. These findings strengthen Novartis' standing in the autoimmune disease treatment market. The company intends to present the data to regulatory bodies for potential approval of this indication.## AstraZeneca faced setbacks as it announced delays in delivering its COVID-19 vaccine doses to the EU. The company cited production issues as the cause of the holdup, leading to frustration among European officials. This news comes at a time when vaccine distribution is crucial in combating the ongoing pandemic, highlighting the challenges faced by pharmaceutical companies in meeting global demand for vaccines.AstraZeneca encountered obstacles when it disclosed delays in distributing its COVID-19 vaccine doses to the EU. The company attributed production problems as the reason for the delay, causing frustration among European officials. This development occurs during a critical period in vaccine distribution to combat the current pandemic, underscoring the difficulties pharmaceutical companies encounter in meeting worldwide vaccine demands.## Pfizer and BioNTech announced plans to test a third dose of their COVID-19 vaccine to assess its effectiveness against new variants of the virus. The study will involve participants who have already received two doses of the vaccine and will evaluate the immune response generated by a booster shot. This initiative reflects ongoing efforts by pharmaceutical companies to adapt their vaccines to combat emerging strains of the virus.Pfizer and BioNTech unveiled intentions to examine a third dose of their COVID-19 vaccine to determine its efficacy against new virus variants. The research will include individuals who have already been administered two doses of the vaccine and will assess the immune response produced by an additional shot. This undertaking demonstrates continuous endeavors by pharmaceutical firms to modify their vaccines in response to evolving virus mutations.

Romanos 13:7 “Pagad a todos lo que debéis: al que tributo, tributo; al que impuesto, impuesto; al que respeto, respeto; al que honra, honra”.Este es uno de temas mas importantes que los hijos de Dios tenemos que entender y comprender: La honra, es uno de los regalos mas grandes que el Señor nos ha dado, es el vínculo protector de las relaciones.La primera relación que el hombre experimenta en la vida es con los padres y la única manera de proteger esta relación es a través de la honra, Éxodo 20:12 “Honra a tu padre y a tu madre, para que tus días se alarguen en la tierra que Jehová tu Dios te da”, Mateo 15:3-4 “Respondiendo él, les dijo: ¿Por qué también vosotros quebrantáis el mandamiento de Dios por vuestra tradición? Porque Dios mandó diciendo: Honra a tu padre y a tu madre”, Efesios 6:2-3 “Honra a tu padre y a tu madre, que es el primer mandamiento con promesa; para que te vaya bien, y seas de larga vida sobre la tierra”.Otra de las relaciones importantes que tenemos en el transcurso de nuestra vida es el matrimonio, con el cual conformamos un hogar; El apóstol Pablo declara en Hebreos 13:4 “Honroso sea en todos el matrimonio, y el lecho sin mancilla; pero a los fornicarios y a los adúlteros los juzgará Dios”. Sino hay honra, los matrimonios colapsan. Pero la honra en la familia no se limita a los cónyuges, va de los padres a los hijos y de los hijos a los padres, debemos honrarlos con las palabras y las acciones. (Podemos extender esta parte hablando de  las expresiones y palabras que usamos contra nuestro cónyuge y con nuestros hijos).Cuando se trata de Dios, Él espera que nosotros vivamos de tal manera que nuestra vida le honre, cuando no hay honra la vida se agrieta y se daña; honrar a Dios es aprender a darle el primer lugar en todo. Cuando le damos el primer lugar a Dios se activa un poder y una unción sobre natural en nuestras vidas, esto no se trata de dinero, se trata de un corazón que dice “No hay nada ni nadie mas importante que Dios”, el que honra a Dios no solo se esta beneficiando asi mismo, está beneficiando a sus próximas generaciones, por tal razón aprende a colocar a Dios en primer lugar y todo en tu vida va a ser transformado.Dios se revela al hombre en su conciencia, en su interior y en la creación de una manera clara y evidente, por tanto es imposible que el ser humano no pueda deducir y concluir que hay un creador detrás de todo lo creado; Romanos 1:20 dice "Porque las cosas invisibles de él, su eterno poder y deidad, se hacen claramente visibles desde la creación del mundo, siendo entendidas por medio de las cosas hechas, de modo que no tienen excusa”, el hombre no tiene excusa para no honrar a Dios.Por todo esto Pablo hace un reclamo a los Romanos, y hoy lo hace directamente a la iglesia “Aunque conocen a Dios, no lo honran como a Dios, ni le dan gracias” y añade que las consecuencias son evidentes: Se hicieron vanos en sus razonamientos y su necio corazón fue entenebrecido. Aquí hay dos aspectos importantes que enseñar a la iglesia, uno de ellos es honrar a Dios y el otro es la gratitud a Dios.¿Que es honrar a Dios? Es reconocerlo como el Dios soberano sobre toda la creación, como el ser de mayor dignidad, de mayor Gloria, de mayor autoridad y de mayor bondad, es glorificar a Dios exaltando sus atributos. Es muy fácil para nosotros ir a alabar a Dios y agradecerle por todo. Pero deshonrar a Dios es aun MÁS fácil; Salmos 36:1-4 “La iniquidad del impío me dice al corazón: No hay temor de Dios delante de sus ojos. Se lisonjea, por tanto, en sus propios ojos, de que su iniquidad no será hallada y aborrecida. Las palabras de su boca son iniquidad y fraude; Ha dejado de ser cuerdo y de hacer el bien. Medita maldad sobre su cama; Está en camino no bueno”.Y quiero mostrar lo que ocurrió tanto en el antiguo testamento con Nabucodonosor, asi como en el nuevo testamento con Herodes por no honrar a Dios.Nabucodonosor deshonró a Dios con solo hablar: “Habló el rey, y dijo: ¿No es ésta la gran Babilonia, que yo edifiqué para casa del reino, con la fuerza de mi poder, y para gloria de mi grandeza?... En la misma hora se cumplió la palabra sobre Nabucodonosor, y fue echado de entre los hombres; y comía hierba como los bueyes, y su cuerpo se bañaba con el rocío del cielo, hasta que su pelo creció como de águila, y sus uñas como de aves”; Daniel 4:29-33 “Al cabo de doce meses, paseando en el palacio real de Babilonia, habló el rey y dijo: ¿No es esta la gran Babilonia que yo edifiqué para casa real con la fuerza de mi poder, y para gloria de mi majestad? Aún estaba la palabra en la boca del rey, cuando vino una voz del cielo: A ti se te dice, rey Nabucodonosor: El reino ha sido quitado de ti; y de entre los hombres te arrojarán, y con las bestias del campo será tu habitación, y como a los bueyes te apacentarán; y siete tiempos pasarán sobre ti, hasta que reconozcas que el Altísimo tiene el dominio en el reino de los hombres, y lo da a quien él quiere. En la misma hora se cumplió la palabra sobre Nabucodonosor, y fue echado de entre los hombres; y comía hierba como los bueyes, y su cuerpo se mojaba con el rocío del cielo, hasta que su pelo creció como plumas de águila, y sus uñas como las de las aves”.Herodes deshonro a Dios y un ángel descendió y lo hirió y fue comido por los gusanos; Hechos 12:21-23 "Y un día señalado, Herodes, vestido de ropas reales, se sentó en el tribunal y les arengó. Y el pueblo aclamaba gritando: ¡Voz de Dios, y no de hombre! Al momento un ángel del Señor le hirió, por cuanto no dio la gloria a Dios; y expiró comido de gusanos”.Cuando en nuestro corazón hay un espíritu de deshonra nos convertimos en hombres amadores de sí mismos, amadores de los placeres más que de Dios, avaros, jactanciosos, soberbios, blasfemos, desobedientes a los padres, ingratos, irreverentes; 2 Timoteo 3:1-5 “También debes saber esto: que en los postreros días vendrán tiempos peligrosos. Porque habrá hombres amadores de sí mismos, avaros, vanagloriosos, soberbios, blasfemos, desobedientes a los padres, ingratos, impíos, sin afecto natural, implacables, calumniadores, intemperantes, crueles, aborrecedores de lo bueno, traidores, impetuosos, infatuados, amadores de los deleites más que de Dios, que tendrán apariencia de piedad, pero negarán la eficacia de ella; a estos evita”.Los cristianos debemos reconocer que todo lo que tenemos es un regalo, Dios nos ha dado todo: la vida, la salvación y todo lo que forma parte de la vida en este mundo y en el venidero. Cada día, cada momento, debería estar lleno de acciones de gracias. Dios es bueno, y todo lo que Él hace y otorga es para nuestro bien.Quiero mostrar a través de un relato Bíblico en el libro de 1 Reyes 17:8-24, el poder de la honra: La viuda de Sarepta, el milagro de la obediencia y la honra.Y quiero comenzar por el final: “La viuda de Sarepta se fue e hizo conforme a la palabra de Elías; y ella, él y su familia comieron durante muchos días. La harina de la tinaja no se agotó, ni se agotó el aceite de la vasija, conforme a la palabra que el Señor había hablado por medio de Elías”.La historia de la viuda de Sarepta comienza mientras está en la puerta recogiendo leña para poder ir a casa y preparar el último bocado de comida para ella y su hijo. En su mente había un pensamiento de muerte: “El fin está cerca, mi hijo y yo comeremos el último bocado de alimento que tenemos y luego moriremos”.Dios decidió tomar la vida del profeta Elías y entrelazarla con la de la viuda, Elías se presentó ante el rey Acab y profetizó: «...en los próximos años habrá una sequía total, sin una gota de rocío ni de lluvia, a menos que y...

Chevron CEO Mike Wirth joined Carl Quintanilla and Sara Eisen at Post 9 in a "First on CNBC" interview. They discussed the oil giant completing its $55 billion acquisition of Hess. Shares of Netflix pull back from all-time highs despite better-than-expected quarterly results. One analyst offers his take on where the stock and the streaming wars go from here. New record intraday highs for the S&P 500 and Nasdaq -- a chief investment strategist joins the discussion about what's next for the rally. Also in focus: Reaction to earnings from the likes of American Express, Sarepta tumbles, President Trump vs. Fed Chair Powell, the House of Representatives passes landmark crypto legislation, CBS cancels Stephen Colbert's "Late Show."   Squawk on the Street Disclaimer

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in the Pharma and Biotech world. Sarepta Therapeutics has recently experienced an 18% increase in its stock value following a significant business overhaul, which included staff layoffs and pipeline shifts. Analysts are cautiously optimistic about the company's future. At the same time, patients are advocating for access to Brainstorm Cell Therapeutics' ALS drug, Nurown, after promising results from an expanded access program. In other news, Novartis is in the process of reshoring its drug manufacturing operations in the US, a move that may take several years to complete. Additionally, a notable number of employees have departed from the FDA's Center for Drug Evaluation and Research amidst an overhaul by the Department of Health and Human Services. These developments underscore the continuous changes and challenges within the pharmaceutical industry. Sarepta Therapeutics recently announced a strategic overhaul, which involved cutting 500 staff members and shifting focus to sirna platform assets. This decision came after two patients passed away following treatment with its Duchenne muscular dystrophy gene therapy, Elevidys. The company has also added a black box warning for acute liver injury and failure to Elevidys as it pivots away from gene therapy programs.

Up to 3,500 FDA staffers received their final walking papers Monday after the U.S. Supreme Court found last week that the government is “likely to succeed” in arguing that its overhaul of HHS is “lawful.” Meanwhile, FDA Commissioner Marty Makary floated policy changes for the agency, including a proposal to lower prescription drug user fees for the next iteration of the program, and one to offer speedier reviews to companies willing to lower the cost of their drugs.  Last week, the regulator opened its cache of complete response letters (CRLs), offering transparency into the rationale behind more than 200 recent rejections for ultimately approved therapies, including those for Eli Lilly's Alzheimer's drug Kisunla and Sarepta's Duchenne muscular dystrophy (DMD) treatment Vyondys 53. The FDA did not, however, release the CRLs for two new rejections: those of therapies from Ultragenyx and Capricor Therapeutics in Sanfilippo syndrome type A and cardiomyopathy associated with DMD, respectively. It was an especially rough week for Ultragenyx, which also, along with partner Mereo BioPharma, released seemingly negative Phase II/III data for their osteogenesis imperfecta therapy.  On a more positive note, two bustling therapeutic spaces continue to see positive data. In obesity, Hengrui Pharma's Kailera Therapeutics–partnered dual GLP-1/GIP receptor agonist elicited 17.7% average weight loss in a pivotal Chinese trial. And the psychedelic therapeutics space is again generating excitement with two recent positive readouts in treatment-resistant depression. BioSpace took a deep dive into the market reaction to these readouts for Compass Pathways and Beckley Psytech and atai Life Sciences, and what exactly investors are looking for in a successful psychedelic therapy.  Finally, we examine the progress of AI biotech unicorns and kick off our series on women in biopharma with profiles on Mayo Venture Partner Audrey Greenberg and the all-female CEO/R&D tandem at Acadia Pharmaceuticals.  

Good morning from Pharma and Biotech Daily: the podcast that gives you only what's important to hear in Pharma and Biotech world.The FDA has rejected Capricor's therapy for Duchenne Muscular Dystrophy (DMD), causing shares to plunge due to insufficient evidence of effectiveness for cardiomyopathy associated with DMD. Lilly and Sarepta also received Complete Response Letters (CRLs) for their therapies. Moderna has won full approval for its COVID-19 vaccine for higher-risk kids, while Pacira has cut staff in San Diego. The FDA has released a cache of CRLs, providing insights into the journeys to market for various drugs. AbbVie has acquired Ichnos Glenmark's lead myeloma antibody, and Ultragenyx's stock plummeted after a late-stage trial disappointment. Kennedy postponed a preventive care panel meeting after a Supreme Court win. Transitioning from one piece of news to the next, let's move on to the webinar exploring digitization strategies for drug development. Stay tuned for more updates in the pharmaceutical industry.

Father Edward Looney reads and comments on The Life of Jesus Christ and Biblical Revelations: From the Visions of Anne Catherine Emmerich.Day 53Volume 1JESUS BEGINS HIS PUBLIC TEACHINGChapter 4: Jesus Journeys Over Libanus To Sidon And SareptaLEARN MORE - USE COUPON CODE ACE25 FOR 25% OFFThe Life of Jesus Christ and Biblical Revelations: From the Visions of Blessed Anne Catherine Emmerich Four-Book Set - https://bit.ly/3QVreIsThe Dolorous Passion of Our Lord Jesus Christ: From the Visions of Anne Catherine Emmerich -https://bit.ly/4bPsxRmThe Life and Revelations of Anne Catherine Emmerich Two-Book Set -https://bit.ly/3yxaLE5The Life of the Blessed Virgin Mary: From the Visions of Anne Catherine Emmerich - https://bit.ly/3wTRsULMary Magdalen in the Visions of Anne Catherine Emmerich -https://bit.ly/4brYEXbThe Mystical City of God Four-Book Set - https://bit.ly/44Q9nZbOur Lady of Good Help: Prayer Book for Pilgrims -https://bit.ly/3Ke6O9SThe Life of Jesus Christ in a Year: From the Visions of Anne Catherine Emmerich is a podcast from TAN that takes you through one of the most extraordinary books ever published. Follow along daily as Father Edward Looney works his way through the classic four-volume set, The Life of Jesus Christ and Biblical Revelations, by reading a passage from the book and then giving his commentary. Discover the visions of the famous 19th-century Catholic mystic, Blessed Anne Catherine Emmerich, a nun who was privileged by God to behold innumerable events of biblical times.Anne Catherine's visions included the birth, life, public ministry, Crucifixion, and Resurrection of Jesus Christ, as well as the founding of His Church. Besides describing persons, places, events, and traditions in intimate detail, she also sets forth the mystical significance of these visible realities. Here is the infinite love of God incarnate and made manifest for all to see, made all the more striking and vivid by the accounts Blessed Anne has relayed.Listen and subscribe to The Life of Jesus Christ in a Year: From the Visions of Anne Catherine Emmerich on your favorite podcast platform or at EmmerichPodcast.com.And for more great ways to deepen your faith, check out all the spiritual resources available at TANBooks.com and use Coupon Code ACE25 for 25% off your next order.

Jamie is out of town, so John's wife, Sarepta, made her debut in the studio. She even came prepared with questions.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.The CDC's new vaccine advisors are meeting with an unexpected agenda today, including discussions on topics such as Sanofi and Gilead's protein degrader deals, the FDA's review of Sarepta's gene therapy, and a contentious hearing with RFK Jr. over vaccines. The meeting marks the beginning of a new era for the influential vaccine committee. The FDA has named a psychedelic proponent as CDER deputy director, while Nektar has declared a Phase IIb win for eczema treatment. Experts say conflicts of interest among axed ACIP members are a "red herring," and the pharma industry is facing turmoil in failed immuno-oncology projects. Prescription drug sales are projected to hit $1.75 trillion by 2030, thanks to GLP-1s. Transitioning to the next news, a major pharmaceutical company has announced a breakthrough in cancer research, potentially changing the landscape of treatment options for patients worldwide. This development comes at a time when the industry is seeing significant advancements in personalized medicine and targeted therapies. Moving on to regulatory updates, the FDA has recently approved a new drug for a rare disease, providing hope for patients who previously had limited treatment options. This decision showcases the agency's commitment to expediting the approval process for innovative therapies that address unmet medical needs. In other news, a biotech startup has secured funding for its groundbreaking technology that aims to revolutionize drug delivery methods. This investment highlights the growing interest in novel approaches to drug development and underscores the importance of innovation in the industry.Wrapping up today's episode, we take a look at the latest trends in digital health, with companies leveraging artificial intelligence and big data analytics to improve patient outcomes and streamline healthcare delivery. These technological advancements have the potential to transform the way healthcare is delivered and pave the way for a more efficient and patient-centric system. That's all for today's episode of Pharma and Biotech daily. Stay tuned for more updates on the latest developments in the pharmaceutical and biotechnology sectors.

This week, BioSpace is at 50% power as Heather McKenzie and Jef Akst are off attending this year's BIO Conference in Boston. The half-team discusses this week's biggest news: the death of another patient who took Sarepta's Duchenne muscular dystrophy gene therapy Elevidys. The patient was a non-ambulatory teenager who experienced acute liver failure after receiving the gene therapy, which is the same cause of death for an Elevidys patient reported in March. Sarepta announced that it was halting treatments to non-ambulatory patients and on a media call discussed new steps in its therapeutic protocol for preventing further liver injuries.  Elsewhere, mergers and acquisitions are surging across biopharma. Eli Lilly picked up the gene editing company Verve Therapeutics for $1.3 billion, which helped bolster the gene editing space —particularly after Sarepta's report of the death. Supernus bought Sage Therapeutics for $795 million, five months after Sage rejected a smaller offer from Biogen.  BioNTech also got in on the dealmaking, buying its German rival—scientifically and in the courtroom—CureVac for about $1.25 billion. The deal seemed focused mostly on CureVac's early-stage cancer immunotherapy pipeline, but analysts were otherwise left scratching their heads on what BioNTech was getting for its money.  Last week on The Weekly the team discussed the sudden dismissal of the CDC's entire ACIP committee, and this week we have a new slate of members. The eight people replacing the 17 members that were removed last week include allies of HHS Secretary Robert F. Kennedy Jr., many of whom are vaccine skeptics who seem to share his skeptical view of vaccination in general.  

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world. Sarepta Therapeutics faced a drop in shares due to a second death from acute liver failure in a patient receiving their DMD gene therapy. On the other hand, Sage Therapeutics had a positive outcome with a $795 million acquisition by Supernus, surpassing a bid from Biogen. Meanwhile, Lilly saw promising results in a phase I study of their amylin/tirzepatide combo for weight loss. J&J's dual-targeting CAR T-cell therapy showed an 80% complete response rate in early lymphoma studies, demonstrating potential in the field. In addition, Intellia Therapeutics is dedicated to developing potentially curative genome editing treatments for severe diseases, showing promise for future advancements in healthcare.

Ohne Aktien-Zugang ist's schwer? Starte jetzt bei unserem Partner Scalable Capital. Alle weiteren Infos gibt's hier: scalable.capital/oaws. Aktien + Whatsapp = Hier anmelden. Lieber als Newsletter? Geht auch. Das Buch zum Podcast? Jetzt lesen. Estée Lauder ist makaber. Kering ist happy über Renault-CEO. Börse ist happy über Friedenshoffnung. Einen Crash gab's bei Sarepta. Übernahmen gab's bei Santos & Sage. Roku & Whatsapp mögen Werbung. AMD fliegt & Trump macht goldene Handys. Walmart (WKN: 860853) ist mit einem KGV von 40 teurer als viele Tech-Aktien. Die Analysten empfehlen die Aktie trotzdem zum Kauf. Warum und wenn ja, wie viele? Wir klären auf. Bei der Art Basel wird diese Woche Kunst für zig Millionen verkauft. Die Firma dahinter ist an der Börse und deutlich weniger wert als die dort verkaufte Kunst. Was da los bei MCH (WKN: A0Q16U)? Diesen Podcast vom 17.06.2025, 3:00 Uhr stellt dir die Podstars GmbH (Noah Leidinger) zur Verfügung.

Reports this morning that Iran has signaled it wants to de-escalate hostilities with Israel – and restart talks with the US: Carl Quintanilla, Sara Eisen, and David Faber broke down the latest along with key market implications ahead of another rate decision from the Fed on Wednesday – and as energy prices remain surprisingly steady. Plus: A live read from the ground in Alberta, as the G-7 summit begins in Canada.  Rockefeller International's Ruchir Sharma brought his outlook for equities – fresh off an Op-Ed in the Financial Times arguing to not underestimate the Chinese as trade talks continue… Plus: hear from Former Deputy Secretary of State – and lead negotiator for the nuclear agreement with Iran under the Obama Administration – Wendy Sherman's take on what comes next here. Also in focus: Meta rolls out ads on Whatsapp, boosting shares; Sarepta stock plummets after a 2nd death tied to an experimental gene therapy; an exclusive with the CEO of aerospace maintenance company StandardAero, live from the Paris Air Show; and the rumors around AWS that are boosting AMD shares to fresh highs. Squawk on the Street Disclaimer

Carl Quintanilla, David Faber and Sara Eisen kicked off a new week of trading with stocks rebounding sharply and oil prices giving back some of Friday's huge gains  -- despite the ongoing Israel-Iran conflict. President Trump approved U.S. Steel's deal with Nippon Steel, with terms requiring a "golden share" for the U.S. government. Guggenheim's Tesla analyst joined the program nearly a week after his "sell" rating on the stock.  Also in focus: Trump at the G7 in Canada, Meta's WhatsApp to roll out ads directly on its platform, Sarepta plummets, Roku surges on its partnership with Amazon, Circle up 400% since its early June IPO.   Squawk on the Street Disclaimer

The tragic death of a second non-ambulatory DMD patient treated with Sarepta's Elevidys gene therapy marks a turning point for the field and should drive all stakeholders to come together to figure out how to safely treat patients with this new modality. On the latest BioCentury This Week podcast, BioCentury's analysts discuss the opportunity FDA, companies and patient advocates have to share data and identify a safe path forward for the gene therapy field, both in DMD and beyond.The analysts also review promising early data for amylin agonists to treat obesity from Metsera and Eli Lilly, and discuss the vision for FDA's future laid out by Commissioner Marty Makary and CBER director Vinay Prasad. This episode of BioCentury This Week was sponsored by ICON Biotech.View full story: https://www.biocentury.com/article/656214#biotech #biopharma #pharma #lifescience #obesity #FDA #DMD00:01 - Sponsor Message: ICON Biotech01:19 - Gene Therapy Safety11:55 - Amylin in Obesity18:17 - FDA's FutureTo submit a question to BioCentury's editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

Superpowers for Good should not be considered investment advice. Seek counsel before making investment decisions. When you purchase an item, launch a campaign or create an investment account after clicking a link here, we may earn a fee. Engage to support our work.Watch the show on television by downloading the e360tv channel app to your Roku, AppleTV or AmazonFireTV. You can also see it on YouTube.Devin: What is your superpower?Nicole: Willingness to take bold leaps and embrace the unknown.Imagine a world where cancer is as treatable as the flu. That's the vision Nicole Paulk, the founder and CEO of Siren Biotechnology, is working tirelessly to bring to life. Driven by groundbreaking science, Nicole's company is on the brink of launching clinical trials for a revolutionary cancer treatment.Nicole's work leverages engineered viruses—those that don't make you sick—to deliver genetic medicines directly to cancer cells. These “good viruses” act as a delivery system for anti-tumor drugs, targeting difficult-to-treat cancers like recurrent high-grade gliomas, a fatal brain cancer. “We started to see data that looked really promising in this space,” Nicole explained, adding, “We felt a moral obligation to go after this cancer where there's just this huge unmet need.”Siren Biotechnology's approach is designed to have broad applications. Unlike treatments that target specific genetic mutations, their method has the potential to work on various tumor types. Nicole describes it as using viruses like a “little FedEx delivery truck” that can be engineered to deliver medicine precisely where it's needed in the body.This innovation didn't happen overnight. Nicole, a former professor of virology at UCSF, took the bold step of leaving academia to launch Siren Biotechnology. “I decided to resign from my faculty position and go be the founder and CEO just because I was so excited about the data and what we had so far,” she shared.In addition to traditional venture capital funding, Siren Biotechnology is inviting the public to invest through a regulated investment crowdfunding campaign. This unique opportunity allows patients, families, and supporters touched by cancer to be part of their journey. “We wanted to bring patients and their families onto our cap table,” Nicole said. “It's a way to involve them in the conversation much earlier.”Siren Biotechnology's work could redefine cancer care and inspire a new model for patient-centered innovation. With clinical trials just months away, the future looks brighter for those battling cancer—and for all of us who dream of a world where cancer is no longer a death sentence.tl;dr:Nicole Paulk's Siren Biotechnology transforms viruses into targeted cancer therapies with universal potential.The company focuses on recurrent high-grade gliomas, a fatal brain cancer with no standard treatment.Nicole left academia to lead Siren, leveraging her groundbreaking virology research to launch the company.Siren's unique crowdfunding campaign lets patients and families invest in the fight against cancer.Nicole's superpower, fearless innovation, drives her bold mission to revolutionize cancer care.How to Develop Fearless Innovation As a SuperpowerNicole's superpower is her willingness to take bold leaps and embrace the unknown. After spending nearly two decades building her academic career, Nicole left her position as a virology professor at UCSF to start Siren Biotechnology. She explained, “I feel like my superpower is just kind of being willing to jump off the cliff without a parachute and be like, we're going to figure it out. We're going to make it work.”Nicole exemplified fearless innovation when her research led to a groundbreaking discovery about the biology of a virus, one that required rewriting existing textbooks. Initially met with skepticism, her work was later validated and became a foundation for Siren Biotechnology's cancer treatment. This pivotal moment highlights Nicole's courage to challenge conventional knowledge and pursue transformative ideas.Tips for Developing This Superpower:Be willing to question conventional wisdom and explore new paths.Take calculated risks, even if it means stepping away from a well-defined career path.Embrace discomfort and uncertainty as a necessary part of innovation.Engage others by communicating your vision in relatable and accessible ways.By following Nicole's example and advice, you can make fearless innovation a skill. With practice and effort, you could make it a superpower that enables you to do more good in the world.Remember, however, that research into success suggests that building on your own superpowers is more important than creating new ones or overcoming weaknesses. You do you!Guest ProfileNicole Paulk (she/her):CEO, Founder, President, Siren BiotechnologyAbout Siren Biotechnology: We are combining two transformative therapeutic technologies – AAV gene therapy and cytokine immunotherapy – into a single, reimagined modality that overcomes key challenges and redefines how we destroy tumor cells and elicit anti-tumor immunity.This is the first AAV gene therapy drug product that can be made once and used in numerous indications – a huge leap forward for the field. This drastically reduces clinical development times, manufacturing timelines, and capital needs for each clinical trial. Most importantly, ‘universal' means countless solid tumor cancer patients – regardless of tumor type or mutations – may benefit from this breakthrough approach.Website: sirenbiotechnology.comX/Twitter Handle: @SirenBioCompany Facebook Page: facebook.com/SirenBiotechnologyOther URL: wefunder.com/siren.biotechnologyBiographical Information: Dr. Nicole Paulk is the CEO, Founder, and President of Siren Biotechnology and has dedicated her career to advancing the field of gene therapy. With nearly two decades of expertise, Nicole has been at the forefront of developing cutting-edge advances to propel the field of gene therapy forward for a wide range of diseases.Before founding Siren, Nicole held various leadership positions in academia and industry and most notably was an Assistant Adj Professor of AAV Gene Therapy in the UCSF Department of Biochemistry & Biophysics before leaving to found Siren. Nicole has a B.S. in Medical Microbiology, a Ph.D. in Viral Gene Therapy and Regenerative Medicine from OHSU, and completed her Postdoctoral Fellowship and Instructorship in Human Gene Therapy at Stanford University prior to starting her lab at UCSF. Nicole is a pioneer in the development of next-generation AAV platforms for gene repair, gene transfer and gene editing, directed evolution for novel engineered capsid evolution, and comparative multi-omic approaches to interrogate translational AAV biology.Nicole is a renowned expert in gene therapy and has consulted extensively for big pharma, written draft CMC guidance for the FDA, and sits on the Scientific Advisory Boards for Sarepta, Astellas, Metagenomi, Dyno Therapeutics, CEVEC, GRO Biosciences, Excision BioTherapeutics, WhiteLab Genomics, Johns Hopkins Gene Therapy Initiative, the Gene Therapy for Rare Disorders Searchlight Program, and several stealth startups. She has been quoted in The Wall Street Journal, The Economist, The Boston Globe, Endpoints, STAT, Phacilitate, GEN, BioPharma Dive, Evaluate Vantage, SF Business Times, WIRED, Drug Discovery World, MIT Tech Review, C&EN, and more. She sits on the Scientific Editorial Boards of the journals Gene Therapy, Human Gene Therapy, and Biopharma International Gene Therapy. She is the Chair of the American Society of Gene and Cell Therapy (ASGCT) Translational Science Committee, and a member of the ASGCT Cancer Cell and Gene Therapy Committee and the Biocom California Cell and Gene Therapy Committee. She has invented numerous AAV gene therapy technologies that have been shared or licensed to dozens of gene therapy companies and nonprofit groups working in rare diseases.Outside of work, you can find Nicole adventure traveling (think whitewater rafting meets backcountry trekking), snowboarding, planning elaborate Halloween parties complete with animatronics and ghoulish menus, tending her vegetable garden, and obsessing over the latest wearable gadgets. If you're trying to track her down at a conference and can't find her, it's because she snuck off to an oyster bar.X/Twitter Handle: @Nicole_PaulkLinkedin: linkedin.com/in/nicolepaulkInstagram Handle: @sirenbioSupport Our SponsorsOur generous sponsors make our work possible, serving impact investors, social entrepreneurs, community builders and diverse founders. Today's advertisers include FundingHope, Kingscrowd and Crowdfunding Made Simple. Learn more about advertising with us here.Max-Impact MembersThe following Max-Impact Members provide valuable financial support:Carol Fineagan, Independent Consultant | Lory Moore, Lory Moore Law | Marcia Brinton, High Desert Gear | Paul Lovejoy, Stakeholder Enterprise | Pearl Wright, Global Changemaker | Ralf Mandt, Next Pitch | Scott Thorpe, Philanthropist | Matthew Mead, Hempitecture | Michael Pratt, Qnetic | Sharon Samjitsingh, Health Care Originals | Add Your Name HereUpcoming SuperCrowd Event CalendarIf a location is not noted, the events below are virtual.Impact Cherub Club Meeting hosted by The Super Crowd, Inc., a public benefit corporation, on June 17, 2025, at 1:00 PM Eastern. Each month, the Club meets to review new offerings for investment consideration and to conduct due diligence on previously screened deals. To join the Impact Cherub Club, become an Impact Member of the SuperCrowd.SuperCrowdHour, June 18, 2025, at 12:00 PM Eastern. Jason Fishman, Co-Founder and CEO of Digital Niche Agency (DNA), will lead a session on "How to Spin $1 of Advertising into $10!" He'll reveal proven strategies and marketing insights drawn from years of experience helping successful crowdfunding campaigns. Whether you're a founder planning a raise or a supporter of innovative startups, you'll gain actionable tips to boost visibility, drive engagement, and hit your funding goals. Don't miss it!Join us on June 25, 2025, at 8:00 PM Eastern for the Superpowers for Good Live Pitch—streaming on e360tv, where purpose-driven founders take the virtual stage to present their active Regulation Crowdfunding campaigns to a national audience of investors and changemakers. Selected startups are chosen for their commitment to community, alignment with NC3's Community Capital Principles, and their drive to create real-world impact. Thanks to sponsors DNA and DealMaker, this event is free to watch and amplifies the voices of underrepresented and mission-aligned entrepreneurs. Don't miss this inspiring evening where capital meets purpose—tune in to discover and support the next wave of impact-driven innovation.SuperCrowd25, August 21st and 22nd: This two-day virtual event is an annual tradition but with big upgrades for 2025! We'll be streaming live across the web and on TV via e360tv. Soon, we'll open a process for nominating speakers. Check back!Community Event CalendarSuccessful Funding with Karl Dakin, Tuesdays at 10:00 AM ET - Click on Events.Devin Thorpe is featured in a free virtual masterclass series hosted by Irina Portnova titled Break Free, Elevate Your Money Mindset & Call In Overflow, focused on transforming your relationship with money through personal stories and practical insights. June 8-21, 2025.Join Dorian Dickinson, founder & CEO of FundingHope, for Startup.com's monthly crowdfunding workshop, where he'll dive into strategies for successfully raising capital through investment crowdfunding. June 24 at noon Eastern. Regulated Investment Crowdfunding Summit 2025, Crowdfunding Professional Association, Washington DC, October 21-22, 2025.Call for community action:Please show your support for a tax credit for investments made via Regulation Crowdfunding, benefiting both the investors and the small businesses that receive the investments. Learn more here.If you would like to submit an event for us to share with the 9,000+ changemakers, investors and entrepreneurs who are members of the SuperCrowd, click here.We use AI to help us write compelling recaps of each episode. Get full access to Superpowers for Good at www.superpowers4good.com/subscribe