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 ...

Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | Bayer has discontinued an early-stage clinical gene therapy for a rare ...

UniQure needs to run another trial to prove its gene therapy "actually helps people with Huntington's disease," said a senior FDA official.

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

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 ...

A new clinical trial led in part by a Lurie Children’s Hospital doctor is showing promise for children with Dravet syndrome, a severe form of epilepsy, and could become the first gene regulation ...

While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be ...

Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed ...

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

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 ...