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

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 ...

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 ...

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

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

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

Intellia is now able to continue both Phase III trials of nex-z. Credit: Piotr Swat / Shutterstock.com (Piotr Swat / Shutterstock.com) The US Food and Drug Administration (FDA) has released the ...

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

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 ...

UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.

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

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