Gene therapy
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.
The companies were two years into a four-year, $400 million agreement aimed at developing and marketing gene therapies together.
As FDA seeks to rehire some fired employees, Donald Trump threatens to enact tariffs on pharma companies unless they reshore manufacturing; another lawsuit hits the complex GLP-1 compounding space as Eli Lilly offers expanded Zepbound options; and struggling gene therapy biotech bluebird bio goes private in an attempt to stay solvent.
At the 2025 National Biotechnology Conference, gene therapies, bispecific antibodies and other novel modalities—relative newcomers to medicine—will be much discussed. In this curtain raiser, BioSpace speaks with conference chair Prathap Nagaraja Shastri of J&J about these highly anticipated topics.
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