Cell and Gene Therapy
THE LATEST
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.
AstraZeneca has recently been investing heavily in the cell therapy space, including two acquisitions for TeneoTwo and Gracell Biotechnologies.
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.