Cell and 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.

This year’s American Academy of Neurology meeting included a presentation that could one day set a new treatment standard for myasthenia gravis.

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.

After entering the CAR T space in February, Eli Lilly is “jumping into in vivo CAR-T with both feet” with the acquisition of Kelonia Therapeutics and its gene delivery technology.

An investigational cocktail was tied to a 0% overall response rate in patients with gastroesophageal cancer, but developers Agenus and MiNK Therapeutics aren’t giving up on the program just yet.

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The draft guidance supports the agency’s new pathway designed to speed up the development of custom gene therapies.