Gene therapy
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.
In this whitepaper, BioSpace reviews the major trends impacting the CDMO sector and the evolving relationship between sponsors and providers. We examine the key qualities pharma and biotech should consider in CDMO selection, and how the macroeconomic and macrodevelopment factors affecting the space play a role in this selection.
The draft guidance supports the agency’s new pathway designed to speed up the development of custom gene therapies.
In this episode of Denatured, you’ll hear from Jason Jones, head of global business development at Cellular Origins and Alexander Seyf, founder & CEO of Autolomous. They discuss how the push to scale cell and gene therapy manufacturing is accelerating interest in automation, digitization, robotics and deeper collaboration across the ecosystem.
As cell and gene therapy developers face rising pressure to produce therapies faster and at lower cost, the industry is leaning on robotics, digital systems and partnerships to bridge the gap between innovation and delivery.
While the accelerated approval unlocks only a small market opportunity for Rocket Pharmaceuticals, it will give the biotech a chance to prepare for future product launches, according to Jefferies.
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