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The U.S. Congress greenlit a historic $315 million in federal ALS research funding for 2026 amid Rare Disease Month, spotlighting biotech progress like VectorY Therapeutics’ first patient dosing in its TDP-43-targeting PIONEER-ALS trial and EverythingALS’ pharma consortia driving biomarker innovations and trial alignment.
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Many scientists-turned-CEOs paradoxically abandon scientific principles when it comes to commercializing their innovations. But applying the scientific method to business decisions can help life science entrepreneurs avoid common pitfalls, attract investment and ultimately bring transformative technologies to market.
FDA vouchers are normally a coveted prize for biopharma companies, but a surprise rejection for Disc Medicine’s rare disease drug has biopharma reconsidering.
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.
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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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Following a Phase II review, Novartis has cut the development of a gene therapy candidate for geographic atrophy. In June, the company sold a dry eye disease drug to Bauch + Lomb for $1.75 billion.
The California pharma is building up to its first-ever approval with promising late-stage data for its once-daily investigational acromegaly pill paltusotine, an alternative to the injectable standard of care.
The first-in-class antibody-drug conjugate—in combination with Merck’s Keytruda—has shown promising results in a Phase II study of patients with metastatic non-small cell lung cancer.
After its prostate cancer therapy was not included in Medicare’s initial drug price negotiation list, Astellas dismissed its Inflation Reduction Act lawsuit this week, while Illumina got new leadership.
With a potential combined market value of $30 billion, BioSpace takes a deep dive into the Phase III data supporting Eisai and Biogen’s Leqembi and Eli Lilly’s investigational donanemab.
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.