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After a patient safety signal and then death, the FDA in October 2025 placed holds on two of the company’s CRISPR programs for hereditary transthyretin amyloidosis.
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Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.
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The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
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Protara is advancing a cell therapy that triggers both adaptive and innate antitumor immune responses, while CG Oncology’s approach makes use of an oncolytic immunotherapy that preferentially targets cancer cells and proliferates inside them, destroying them from the inside.
Eli Lilly is aggressively ramping up its manufacturing capacity for tirzepatide as compounding pharmacies continue to challenge an FDA decision to formally end the shortage of the obesity and diabetes drug.
It is not clear what specifically the new facility will produce, though Amgen could be ramping up its manufacturing capacity as it prepares to enter the obesity space with MariTide.
Iskra Reic will continue to serve as the pharma’s senior vice president for Vaccines and Immune Therapies as she steps in for Leon Wang, who was detained by Chinese authorities in November.
California-based biotech Nuvig Therapeutics scooped up $161 million from the investment arms of Sanofi, Bayer, Novo Holdings, BMS and others for its Fc fragment immunomodulator aimed at improving autoimmune dysregulation.
The Muna partnership will give GSK access to Muna’s MiND-MAP platform, which it will apply to postmortem brain samples to identify potential therapeutic targets for Alzheimer’s disease.
Novartis is seeking to prevent the entry of generics for its blockbuster heart failure drug Entresto, its top-selling asset that brought in more than $6 billion in net global sales last year.
The payment scheme will tie gene therapy payments to improvements in health outcomes—and could potentially boost the uptake of these sickle cell disease treatments.
In recent months Novo Nordisk has invested several billions of dollars to boost its manufacturing capacity—including its highly contested $16.5 billion merger with CDMO giant Catalent.
In this episode of Denatured, BioSpace’s Head of Insights Lori Ellis, Miguel Forte and Ali Pashazadeh discuss how a slow and steady pace is a continuation of the pattern we have seen throughout the last three years.