News
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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Read our takes on the biggest stories happening in the industry.
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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Analysts believe that Gilead’s new PrEP drug Yeztugo could reach peak sales of $4.5 billion. Not if GSK has anything to say about it.
Why did two private equity firms with more than $460 billion under management want a little old gene therapy biotech called bluebird bio? We wanted to know.
In this episode presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses the FDA’s first draft guidance for AI in drug development, published in January 2025, with Archana Hegde, senior director, pv systems and innovations at IQVIA.
Cell and gene therapy leaders say the agency’s decision to remove the Risk Evaluation and Mitigation Strategies that had been attached to approved CAR T cancer therapies reflects “thoughtful consideration of real-world evidence” and “regulatory trust.”
BPL-003 showed “robust” efficacy data in treatment-resistant depression, according to analysts from Jefferies, who noted that the asset could hit peak market sales of $1 billion. The results clear the way for the asset’s late-stage development and for the completion of a proposed merger with atai Life Sciences.
Macrocyclic peptides are designed to engage complex targets like biologics but pass through cell membranes like small molecule drugs.
The pivotal trial for Neurogene’s Rett syndrome gene therapy makes use of baseline controls and a rigorous endpoint that could help ensure a broader label for the drug product, if approved, according to analysts.
With PN-477, Protagonist is directly going up against Eli Lilly, which is advancing retatrutide, also a triple-G agonist, in a Phase II trial.
Pfizer insists that the discontinuation of the Phase II study was due to recruitment difficulties and was not linked to maplirpacept’s safety or efficacy.
Big Pharmas like Eli Lilly, Sanofi and Novartis headed back to the dealmakers table multiple times, with 32 total deals counted across the industry for the first half.