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. 

This episode continues the discussion regarding the rapid evolution of mRNA technologies since COVID-19. Guests discuss the improvements that have occurred within just a few years, which are making these therapies more reliable, cost-effective and viable for personalized cancer, rare disease chronic disease treatment.

Today’s episode is sponsored by Eclipsebio. From AI-ready datasets to sequencing validation, they drive RNA success.

Explore their solutions at ⁠https://eclipsebio.com/⁠.

Host

⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace

Guests

⁠Andy Geall⁠, Co-founder and Chief Development Officer, Replicate Bioscience; Chair of the Board, Alliance for mRNA Medicines

⁠Pad Chivukula⁠, Co-founder, CSO & COO, Arcturus Therapeutics

Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

This episode focuses on how AI is transforming pharmacovigilance (PV) on a global scale, particularly focusing on the evolving role of local qualified persons for pharmacovigilance in the EU.

This episode is presented in partnership with IQVIA.

Host

⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace

Guest

Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

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.  

In this discussion, BioSpace’s vice president of marketing ⁠⁠Chantal Dresner⁠⁠ and careers editor ⁠⁠Angela Gabriel⁠⁠ take a look at job market performance in the second quarter of 2025. 

They discuss job posting trends, application rates and the most significant layoffs of 2025 so far, plus wider trends impacting biopharma including Massachusetts’ $30M tax incentives and factors affecting California’s success.  

Want to receive our latest quarterly job market reports as soon as they’re published? ⁠⁠Subscribe⁠⁠ to Career Insider for our job market updates, job trends, career advice and more. 

This discussion focuses on how mRNA and self-replicating RNA (srRNA) technologies are expanding far beyond COVID vaccines into revolutionary therapeutic applications for cancer and rare diseases. It is clear that mRNA therapeutics offer three major application areas: infectious disease vaccines, therapeutic vaccines for oncology, and protein replacement for monogenic rare diseases.

Today’s episode is sponsored by Eclipsebio. From AI-ready datasets to sequencing validation, they drive RNA success.

Explore their solutions at https://eclipsebio.com/.

Host

⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠, Head of Insights, BioSpace

Guests

Andy Geall, Co-founder and Chief Development Officer, Replicate Bioscience; Chair of the Board, Alliance for mRNA Medicines

Pad Chivukula, Co-founder, CSO & COO, Arcturus Therapeutics

Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

While most of the U.S. was celebrating the 4th of July holiday, President Donald Trump was busy signing the One Big, Beautiful Bill into law. This wide-ranging tax law has a few implications for the biopharma industry, including expanded IRA exemptions for orphan drugs.  

Looking ahead to the second half of 2025, BioSpace reviews some of the upcoming catalysts highlighted by Jefferies’ “Halftime Show” report,including a highly anticipated Phase III readout for Eli Lilly’s oral obesity candidate orforglipron and an eye on rare disease decisions under the “new” FDA.  

Speaking of FDA decisions, this week kicked off with a surprise approval—that of KalVista’s Pharmaceuticals’ Ekterly for hereditary angioedema. The road to approval for Ekterly was not a smooth one, after the FDA delayed its target action date and Endpoints News reported that FDA Commissioner Marty Makary tried to have the application rejected.  

More regulatory controversy is afoot as Health Secretary Robert F. Kennedy Jr. is facing yet another lawsuit. A group of medical organizations have sued Kennedy and other health leaders in an attempt to reverse Kennedy’s recent decision to remove COVID-19 shots from the routine immunization guidelines for healthy children and healthy pregnant women. In other vaccine news, Kennedy endorsed the expanded use of RSV vaccines for people 50 through 59 years old who are at risk of severe disease—following the recommendation of the CDC vaccine advisory committee he turfed last month.  

This seeming reversal of sentiment largely mirrors the Secretary’s massive HHS overhaul, which has already seen several of these layoffs reversed. In ClinicaSpace this week, we take a deep dive into the numbers. Also in ClinicaSpace, we feature four therapies hanging tough in a troubled TIGIT space that has seen several companies burn billions of dollars on failed assets. And BrainStorm Cell Therapeutics is back in the news after signaling support for a Citizens’ Petition submitted to the FDA requesting the approval of its cell therapy NurOwn, whose Biologics License Application was withdrawn in 2023.  

Finally, in BioPharm Executive, we take a deep dive into the burgeoning longevity space and unpack the short-lived marriage between Novo Nordisk and Hims & Hers Health.   

The U.S. Supreme Court closed out its session before breaking for summer on Friday, ruling that decisions around coverage of HIV PrEP drugs should be left up to the U.S. Preventive Services Task Force. The ruling thwarted a lawsuit brought by a group of insurance providers who challenged the Affordable Care Act’s requirement that they cover preventive medicines, such as HIV drugs, recommended by the task force. The high court also determined that members of this task force can be removed at will by HHS Secretary Robert F. Kennedy Jr.

Speaking of HHS committees from which members have been removed at will by Kennedy, the CDC’s revamped Advisory Committee on Immunization Practices convened last week for its first meeting since the roster overhaul, where a vote on Merck’s recently approved RSV-targeting monoclonal antibody Enflonsia and a discussion around the inclusion of preservative thimerosal in influenza vaccines was on the docket, but an expected vote on Moderna’s mRNA-based RSV shot mRESVIA was not.

Also dominating the headlines on Friday was the FDA’s decision to remove the Risk Evaluation and Mitigation Strategies (REMS) program from the six approved CAR T therapies it was applied to. Analysts and patient groups lauded the move, which is expected to potentially double access to these life-saving therapies, saying it reflects “thoughtful consideration of real-world evidence” and “regulatory trust.”

Willliam Blair noted that it could be a “positive signal” to companies developing CAR T therapies for autoimmune diseases, which now includes AbbVie. In an all-cash buyout on Monday, the Illinois-based pharma dropped $1.2 billion for Capstan Therapeutics and its in vivo edited CAR T therapy for B cell–mediated autoimmune diseases. M&A has been on an uptick of late, and private equity companies—such as those that snapped up bluebird bio—are also getting in on the game.

Finally, after Lykos Therapeutics’ high profile failure last summer, the psychedelics space is heating up once again. While a Phase III readout of Compass Pathways’ psilocybin drug last week in treatment-resistant depression received a muted reaction from investors, the response to a Phase II readout for Beckley Psytech and atai Life Sciences’ intranasal psychedelic was more positive.

In this episode of Denatured, Archana Hegde from IQVIA discusses the practical challenges faced by pharmacovigilance (PV) professionals as they navigate the FDA's first draft guidance for AI in drug development, published in January 2025. Hegde explains that the vagueness of the current framework is like “a recipe with no pictures and mystery ingredients." During the discussion, she highlights areas of existing confusion.

This episode is presented in partnership with IQVIA.

Host

⁠⁠⁠⁠⁠⁠Lori Ellis⁠⁠⁠⁠⁠⁠, Head of Insights, BioSpace

Guest

Archana Hegde, Senior Director, PV Systems & Innovations, IQVIA

Disclaimer: The views expressed in this discussion by guests are their own and do not represent those of their organizations.

Robert F. Kennedy Jr. testified in front of the House Committee on Energy and Commerce Tuesday, where Democrats confronted the health secretary on hot button issues ranging from his recent overhaul over the CDC’s Advisory Committee on Immunization Practices (ACIP) committee, Kennedy’s recently published—and error-ridden—MAHA report, and his threat to ban government scientists from publishing in certain medical journals.  

Meanwhile, at the FDA, the mass exodus of senior leadership continues. On Monday, Jacqueline Corrigan-Curay, acting head of the Center for Drug Evaluation and Research (CDER), announced her retirement as of July. This follows the ouster of Nicole Verdun—the wildly popular director of the FDA’s Office of Therapeutic Products—and her deputy, Rachael Anatol. Their involuntary departure sent shock waves through the biopharma industry, as Verdun had been considered a stabilizing force at the rapidly reshaping agency.  

Speaking of the revamped ACIP, the new panel will meet for the first time Wednesday and Thursday to discuss COVID-19 vaccine safety, maternal and pediatric RSV vaccines and more, as experts question the experience and anti-vaccine views of some of Kennedy’s recently appointed members and others express concern about the potential politicization of the committee.   

On the clinical front, Eli Lilly, Novo Nordisk and others presented new data from their next-generation obesity programs at the American Diabetes Association’s 85th Scientific Congress. After failing to impress investors—and meet its own high expectations—with CagriSema, Novo sought to reassure investors by touting a safety profile “in line with the GLP1-RA class,” and Eli Lilly reported that bimagrumab, when used alongside Novo’s Wegovy, led to additional weight loss while also preserving muscle mass.  

Finally, we recap BIO2025, where Jef Akst, Lori Ellis and Heather McKenzie moderated panels on cell and gene therapy, cybersecurity and AI, and accelerating market entry for rare disease treatments. Relevant to the latter discussion, congressional Republicans dropped the Orphan Cures Act from their version of President Donald Trump’s “One Big Beautiful Bill Act,” and congresspeople, including Rep. Gus Bilirakis (R-Fla.) at Tuesday’s hearing asked Kennedy to commit to supporting the priority review program for rare pediatric diseases, which expired at the end of last year.