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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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Seeking to deepen its neurology and rare disease pipelines, AstraZeneca’s Alexion has joined forces with Verge Genomics to leverage its artificial intelligence platform in drug discovery and development.
The companies have started a collaboration worth up to $3.4 billion to develop a portfolio of degrader-antibody conjugates, a potentially new class of antibodies that selectively kill cancer cells.
To build its ophthalmology portfolio, Japan’s Otsuka Pharmaceuticals has teamed with RNA editing biotech Shape Therapeutics to develop adeno-associated virus gene therapies for ocular diseases.
Analysts said CymaBay’s seladelpar is emerging as the “therapy of choice in the second-line setting” and “could impact the treatment landscape” across a spectrum of primary biliary cholangitis patients.
After a three-month delay, GSK expects an FDA verdict for its myelofibrosis candidate, while Alnylam gears up for an advisory committee meeting discussing patisiran in cardiomyopathy of ATTR amyloidosis.
A thorough reassessment of the confounders between FibroGen’s trials is necessary to salvage the company’s Duchenne Muscular Dystrophy program and regain investor confidence.
The RNAi therapy, which the companies are co-developing and commercializing, reduced blood pressure in hypertensive patients with high cardiovascular risk by 15 mmHg over placebo.
At the recommendation of an independent data monitoring committee, J&J decided to stop the MACiTEPH trial in chronic thromboembolic pulmonary hypertension due to futility.
Blue Cross and Blue Shield of Louisiana has filed a case against Bristol Myers Squibb alleging the company conducted patent fraud to extend protections for its multiple myeloma drug Pomalyst.
The company has withdrawn a suit against the Department of Health and Human Services after its prostate cancer therapy was not included in Medicare’s initial drug price negotiation list.