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FDA vouchers are normally a coveted prize for biopharma companies, but a surprise rejection for Disc Medicine’s rare disease drug has biopharma reconsidering.
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PitchBook’s 2025 biopharma VC analysis clocked $33.8 billion in capital dispatched in 2025, mainly to companies with later-stage programs ready to roll into the clinic.
Long an R&D company that partnered off assets, RNAi biotech Ionis Pharmaceuticals shifted in 2025 to bring two medicines to market alone. Analysts are already impressed—and there’s more to come in 2026.
Regulatory uncertainty is no longer background noise. It is a material investment risk that reshapes how capital is deployed and pipelines are prioritized.
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The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is part of a larger communications crisis unfolding at the agency over the past nine months that has also ensnarled Sarepta, Capricor, uniQure and many more.
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By far, the largest acquisition of 2024 was Novo Holdings’ yet-to-be-closed buyout of manufacturer Catalent at $16.5 billion. Outside of that, the leading pharmaceutical companies kept to less than $5 billion per deal.
Ending the diagnostic journey for patients, particularly those with rare diseases, improves patients’ quality of life while reducing costs to healthcare systems.
With nearly 90% of patients showing no detectable cancer cells after treatment, J&J and Legend’s Carvykti could stave off competition from emerging CAR T therapies such as Gilead and Arcellx’s anito-cel.
BNT327, now in early-phase trials, is part of a class of drugs that could one day challenge Keytruda’s dominance. BioNTech obtained the candidate when it bought Biotheus last month in an acquisition deal that could reach up to $950 million.
GSK, Gilead and Arcellx, Vertex and more present new data at the American Society of Hematology annual meeting just as sickle cell therapies Casgevy and Lyfgenia have a new outcomes-based payment model; Eli Lilly and Novo Nordisk pump new funds into manufacturing; and AbbVie makes a Cerevel comeback while uniQure clears a path toward accelerated approval in Huntington’s disease.
Carisma’s second workforce reduction this year likely leaves the company with 44 full-time employees as turns its focus to developing therapies for fibrosis, oncology and autoimmune diseases.
In a Type B meeting, the FDA signified that it will allow uniQure to use a natural history control, the composite Unified Huntington’s Disease Rating Scale, and neurofilament light chain levels to support the accelerated approval of its gene therapy AMT-130.
The overall survival edge over J&J’s Darzalex will help GSK strengthen its case as it plots the market comeback of Blenrep, which was pulled after a failed confirmatory study.
Anito-cel has shown no signs of delayed neurotoxicity at around 9 months of follow-up, hinting at a safety profile that could set it apart from J&J and Legend’s Carvykti.
According to Jake Van Naarden, president of Lilly Oncology, the excess deaths could be due to the high rate of crossover in BRUIN CLL-321.