Yentli Soto Albrecht has a rare genetic form of ALS and is working on research at Penn that could one day prove life-saving.

RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

UniQure’s AMT-130 is a gene therapy to reduce levels of the Huntingtin protein in the brain. ・The FDA recently said that data ...

In a strikingly blunt briefing, a senior FDA official accused uniQure of pushing “distorted” data to mask a “failed” therapy ...

Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...

U.S. Cell and Gene Therapy CDMO Market is projected to grow from USD 2.09 Billion in 2025 to USD 10.28 Billion by 2035 due to expanding gene and cell therapy pipelines globallyAustin, March 06, 2026 ...

Developers of gene therapies to treat knee osteoarthritis are taking the familiar intra-articular route to deliver proteins and molecules directly to the site of the disease.

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...

Clinical-stage biotechnology company Jaguar Gene Therapy has completed dosing in Cohort I of its first-in-human clinical trial investigating JAG201 for patients with SHANK3 haploinsufficiency, a ...