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Disc Medicine’s leadership tried to express optimism that its rare disease therapy bitopertin can be approved based on a Phase 3 trial set to begin shortly. However, analysts are worried that the protocol was developed with former FDA leaders.
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The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is “part of a disturbing pattern” of moving regulatory goalposts, according to Clay Alspach, executive director of the Alliance for mRNA Medicines. Meanwhile, streamlined communications with regulators in other countries pave the way for rapid uptake of novel modalities.
The current state of political affairs in the U.S. does not bode well for the direction of that turn. The country is at real risk of losing its long-held lead in biotech innovation.
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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The FDA issued a rare Refusal-to-File letter to Moderna over its mRNA-based influenza vaccine application, in an unusual move that sent the biotech’s shares tumbling.
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California’s life sciences manufacturing jobs dipped 3.7% in 2024, according to a new Biocom California report. Still, several companies made—and continuing making—significant manufacturing investments in the state as key trends shape the discipline.
The high court sides with HHS on HIV PrEP drugs; Health Secretary RFK Jr.’s newly appointed CDC vaccine advisors discuss thimerosal in flu vaccines, skip vote on Moderna’s mRNA-based RSV vaccine; FDA removes CAR T guardrails; AbbVie snaps up Capstan for $1.2B to end first half; and psychedelics take off again with data from Compass and Beckley.
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.
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.
In this episode presented by IQVIA, BioSpace’s head of insights Lori Ellis discusses the FDA’s first draft guidance for AI in drug development, published in January 2025, with Archana Hegde, senior director, pv systems and innovations at IQVIA.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.