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Former FDA, CDC and NIH leaders convene at the BIO International Convention to discuss the dismantling of the Department of Health and Human Services under the Trump administration—and where we go from here.
If cell and gene therapy makers are going to achieve their mission to improve patients’ lives, the industry must come together to share information across stakeholders, from regulators to manufacturers to payers.
While drugmakers and other stakeholders want to see faster approvals, experts say the FDA’s Commissioner’s National Priority Voucher program is still bereft of important details, with candidate selection and interference from the agency’s senior leaders topping the list.
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Read our takes on the biggest stories happening in the industry.
UniQure’s planned third-quarter submission for its Huntington’s disease gene therapy may be a harbinger of a more flexible FDA under acting commissioner Kyle Diamantas—but how long will it last? And how can companies be sure these positive decisions won’t just be reversed?
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Weeks after Boehringer Ingelheim and Eli Lilly retracted billions of dollar in German commitments, the nation’s government is reportedly changing a contentious element of its planned healthcare reforms.
Elicio Therapeutics’ investigational cancer immunotherapy failed to meet the primary endpoint of disease-free survival in a Phase 2 trial—a result the company attributed mostly to a disproportionate number of patients with higher residual disease.
Sanofi makes no mention of the Commissioner’s National Priority Voucher. Tzield was awarded the ticket in October 2025, but Sanofi requested withdrawal from the program after former CDER head Tracy Beth Høeg reportedly expressed skepticism of the drug.
Rhythm Pharmaceuticals’ Imcivree reduced fat—while boosting muscle—in patients with Prader-Willi syndrome.
Eli Lilly’s new JAK2 inhibitor—which it obtained from the recent acquisition of Ajax Therapeutics—reduced spleen volume by more than a third in 70% of patients with myelofibrosis.
Intellia Therapeutics’s Senior Vice President Maria Natale discusses why the most successful launches are shaped long before approval, with strategy, structure and patient insight at the core.
DemeRx is launching the first U.S. clinical trial of an ibogaine-derived drug candidate, marking a pivotal moment for a controversial psychedelic long sidelined by safety concerns.
After suffering the market withdrawal of its only product, Amylyx is gearing up for a pivotal Phase 3 readout in post-bariatric hypoglycemia. But the company’s driving ethos is still to treat “debilitating, devastating” neurodegenerative diseases, co-CEO Justin Klee told BioSpace.
Ibogaine’s unconventional “matrix pharmacology” may underlie both its therapeutic promise and unpredictable cardiac risks. Unraveling this mechanism could help drug developers hoping to bring ibogaine analogs to market.
Officials sanctioned Dabur India months after FDA inspectors found bird droppings and data integrity deficiencies during an inspection of the plant.