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The resubmission for RGX-121, expected in the third quarter, comes as the FDA has deemed REGENXBIO’s existing data “sufficient” to support an accelerated filing. It immediately follows a similar reversal of position regarding uniQure’s embattled Huntington’s disease gene therapy.
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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.
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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FDA veteran Peter Marks will now shape the future of Eli Lilly’s vaccines work after the buys of Curevo, LimmaTech Biologics and Vaccine Company for up to $3.8 billion total.
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The forecast makes pharma one of the fastest-growing manufacturing industries in the U.S.
Pfizer showcased multiple late-breakers at the American Society of Clinical Oncology’s annual conference but its biggest data are expected later this year.
Merck may not have had the splashiest presentation at the American Society of Clinical Oncology meeting, but the data show why the Big Pharma remains king of oncology, analysts say.
Eli Lilly and the Haisco Pharmaceutical Group have yet to disclose what specific indications they plan to prioritize.
When used alongside Merck’s Keytruda, Moderna’s personalized cancer vaccine halved the risk of death or disease recurrence in a mid-stage trial of advanced melanoma—a result analysts said could help “instill confidence” in the regimen.
Recent deals from Pfizer and Bristol Myers Squibb hint at Big Pharma’s more collaborative approach to partnering with Chinese companies.
Government backing, deep scientific talent and a robust pharma heritage are helping France punch above its weight, but turning research into investable companies remains a challenge.
For Peter Pitts, a former associate commissioner at the FDA, the appointment to the board of BrainStorm Cell Therapeutics is an opportunity to fulfill a promise he made long ago to a patient with ALS.