Gene therapy
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.
Facing declining valuations and funding challenges, public biotechs like bluebird bio are going private to restructure, reduce regulatory burdens and refocus on long-term growth.
Roche acquired Spark Therapeutics in 2019 for $4.8 billion.
In this deep dive, BioSpace explores the diverse therapeutic modalities now in development, as well as the opportunities and battles for market dominance in this emerging space.
On the agenda for the FDA this month are two RNA-based treatments for rare diseases.
The search for a partner for zerlasiran is ongoing, according to Silence. In the meantime, the biotech will focus its resources on divesiran, which it is testing for polycythemia vera and other hematologic indications.
ITF, IntraBio and Orchard are among the companies that have won FDA nods in the past year for Duchenne muscular dystrophy, Niemann-Pick disease type C, metachromatic leukodystrophy and more.
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