Gene therapy
Shares of REGENXBIO declined 37% on a mixed data readout and other updates from the company’s first quarter earnings call Thursday.
In this episode of Denatured, you’ll be hearing from Miguel Forte, president of the International Society for Cell and Gene Therapy (ISCT), and Jon Ellis, co-founder & CEO of Trenchant Bios, speaking live from the ISCT annual meeting. We dive into mesenchymal stem cells and induced pluripotent stem cells, exploring the science behind them, the manufacturing challenges, and the potential for scalable, engineered next-generation therapies.
Sarepta Therapeutics has put in place several initiatives to help its gene therapy Elevidys return to growth, but recovery will take a long time, according to company executives.
Comprehending the spate of recent rejections in the cell and gene therapy space may require looking no further than early-stage clinical trials of candidates from REGENXBIO, Excision BioTherapeutics and Intellia Therapeutics.
UniQure plans to submit AMT-130 to the U.K.’s Medicines and Healthcare products Regulatory Agency in the third quarter of 2026 based on Phase 1/2 data showing a 75% slowing of disease—the same data the FDA has deemed unacceptable for a biologics license application.
George Church’s Rejuvenate Bio is turning to social networks to help fund its work on one-time gene therapies targeting chronic diseases and root causes of aging.
As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” session focused on the need to expedite more bespoke gene editing treatments like the one that saved young KJ Muldoon.
Approved Thursday via the FDA’s Commissioner’s National Priority Voucher program, Otarmeni is the first gene therapy for hearing loss—and the first treatment to target an underlying cause of the condition.
At Sarepta Therapeutics, we’ve seen it all. Here are the questions I believe we should be asking to move forward in Duchenne muscular dystrophy.
J&J will hand over the rights to bota-vec for $25 million upfront, clearing MeiraGTx to seek regulatory approvals in the U.S. and EU in 2027.
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