Gene therapy
The European Medicines Agency asked for three clinical trials to be placed on hold until the exact cause of death of a US teenager, who suffered acute liver failure after receiving Elevidys, can be precisely determined.
Already reeling from years of market chaos, the announced departure of CBER chief Peter Marks sent a ripple across biopharma markets.
The layoffs will take place throughout 2025 and will mostly affect Tenaya’s research and manufacturing operations. The company is continuing to test its hypertrophic cardiomyopathy gene therapy.
Ayrmid’s offer is 50% higher than bluebird’s previously announced deal with Carlyle and SK Capital Partners.
Industry updates and investment insights—including investor behavior and fundraising advice—were among the topics experts explored at Charles River Laboratories’ 2025 Cell & Gene Therapy Summit.
Werner held roles at Bristol Myers Squibb, AstraZeneca and Novartis before landing at Alltrna, where she works to develop tRNA-based treatments for a range of diseases.
After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent Project Muscular Dystrophy stepped up—as they always do.
After a patient taking the Duchenne muscular dystrophy gene therapy Elevydis died of liver injury, Sarepta will update the label to reflect the safety signal.
The gene therapy world is in turmoil, but Arbor, armed with more than $1 billion in partnerships and raises, is going forward.
Dyne is eyeing an accelerated approval filing for DYNE-251 in early 2026 that would pit the asset against Sarepta’s Exondys 51 in a patient population amenable to exon 51 skipping.
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