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In late May, a patient died after receiving Rocket Pharmaceuticals’ investigational gene therapy for Danon disease, spurring the hold. After lowering the dose and changing the regimen of immune modulators patients receive, the company has received FDA clearance for the trial to continue.
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Long considered resistant to economic downturns, the pharmaceutical industry may face a greater challenge this time around as GLP-1s dominate and the population grows older.
Researchers in pharma and beyond have historically glommed onto a limited number of disease targets, limiting innovation. AI could change that.
Akero Therapeutics, 89bio, Boston Pharmaceuticals and more are working to bring novel treatment options for metabolic dysfunction-associated steatohepatitis to a market that could reach $16 billion by 2033.
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4D Molecular Therapeutics today announced updated interim safety and efficacy data on six adults with Fabry disease cardiomyopathy treated with a single intravenous (IV) infusion of 4D-310 (1E13 vg/kg) with follow-up of 12-33 months overall, including 12-24 month follow-up data on cardiac contractility, exercise capacity, quality of life and cardiac biopsy data.
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Read our takes on the biggest stories happening in the industry.
Unpredictable communication and a lack of transparency are eroding the industry’s and the public’s trust. The FDA, experts agree, needs to take control of the narrative.
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The high court’s order blocks a May decision by a California court that temporarily blocked the efforts of Health Secretary Robert F. Kennedy Jr. to drastically reduce the size of his agency’s workforce.
Leerink Partners called the announcement a ‘positive’ given the delayed timeframe and the uncertainty that the administration will implement tariffs at all.
The FDA will allow a new dosing schedule for Eli Lilly’s Alzheimer’s drug Kisunla that could lessen a known side effect of the monoclonal antibody drug class that has led to several deaths.
H2 2025 catalysts to watch, biopharma implications of President Trump’s tax law, KalVista’s new hereditary angioedema drug that Marty Makary reportedly tried to reject, another lawsuit aimed at Health Secretary Robert F. Kennedy Jr. and a plea from patients with ALS for access to BrainStorm’s NurOwn.
Actithera’s radiopharma assets irreversibly bind to their targets, allowing for longer retention of the drug inside tumors.
Armed with the latest biological knowledge and cutting-edge computational techniques—and, of course, investor dollars—these six biotechs are playing in the largely underappreciated longevity space, developing therapies that may improve the quality of aging.
Analysts said the deal with Novo was likely giving Hims “‘credibility’ or increased consumer traffic,” adding that the “litigation risk is back on the table” now that the Danish pharma has stepped away.
The industry sector focused on aging is only about 10 years old, but acting on what scientists already know, a new crop of biotechs, backed by investors, are taking a disease-centric approach to extending the human lifespan.
BrainStorm Cell Therapeutics issued a statement Tuesday supporting a Citizens’ Petition submitted to the FDA requesting the approval of its cell therapy NurOwn, whose BLA was withdrawn in 2023. A Phase IIIb trial was scheduled to begin last month.
The deal marks an end for CAR T company Cargo Therapeutics, which has been slashing its workforce and cutting programs since the January decision to halt its lead candidate for a certain type of aggressive large B cell lymphoma.