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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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By inhibiting the APRIL cascade, Otsuka Pharmaceuticals’ Voyxact slowed kidney function decline in patients with IgAN, opening up a potential path for full approval while also reading through to Vertex and others with similar assets.
The FDA hasn’t been transparent and open enough with how it has implemented the Commissioner’s National Priority Voucher program, patient, industry and trade groups said Thursday.
Rather than getting hung up on what to call DEI in the workplace, leaders should take three specific actions to help their employees embrace and engage with it. Companies and the patients they serve will benefit.
Massachusetts’ life sciences jobs declined 1% in 2025, according to a new MassBioEd report. However, the report is projecting industry employment will increase 9.7% by 2030. It also noted reskilling needs for some roles, including scientists.
With Germany moving to curb healthcare spending, Eli Lilly and Boehringer Ingelheim have rethought plans to invest in facilities, including a manufacturing plant for GLP-1 drugs.
In addition to a high rate of deaths, ADC Therapeutics’ Zynlonta plus rituximab showed no overall survival benefit in patients with diffuse large B cell lymphoma, casting doubt on its value as a second-line treatment in this indication.
All told, CytomX Therapeutics now stands to receive up to $4 billion over the course of its partnership with Regeneron, if all milestones are met.
Alnylam Pharmaceuticals will leverage Inceptive Nucleics’ generative machine learning models to accelerate the development of RNA interference therapies.
In this episode of Denatured, you’ll be hearing from Daniel Gil, CEO of Pelage Pharmaceuticals and Francisco Ramírez-Valle, senior vice president of immunology discovery at Eli Lilly. We dive into the long-overlooked hair loss space, exploring why true innovation has lagged, how a regenerative approach aims to reactivate dormant follicles and what early proof-of-concept means for patients.
Looking for a biopharma job? Check out the BioSpace list of 12 top companies hiring life sciences professionals like you.