Cell and Gene Therapy
THE LATEST
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.
Having established success in cancer, biopharma is now looking to leverage CAR T therapies against a new target, autoimmune disorders, with several early- to mid-stage readouts expected this year.
Johnson & Johnson and Legend Biotech hope to hit blockbuster status for Carvykti this year.
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.
Abecma made $406 million in 2024, of which BMS paid $43 million to 2seventy bio as part of their profit-sharing agreement.
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.