Delandistrogene moxeparvovec-rokl shows significant motor function improvements in 8- and 9-year-olds with DMD, countering expected motor decline. Statistically significant differences were observed ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

FDA RMAT boosts inhaled gene therapy KB707, delivering IL‑2/IL‑12 to lung tumors; early trial shows responses with manageable ...

There are currently no approved disease-modifying therapies for MPS IIIA.

ROCKVILLE, Md., July 10, 2025 /PRNewswire/ -- REGENXBIO Inc. today announced the publication of preclinical results comparing a microdystrophin gene therapy construct that included the C-terminal (CT) ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain disorders known as SYNGAP1-related disorders, or SRDs. Researchers were able ...

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...

This article is authored by Amit Mookim, CEO, Immuneel Therapeutics Pvt. Ltd. and member, NATHEALTH.

A U.K. center of excellence for gene and cell therapies is promoting the benefits of digital tools to aid the manufacturing.

After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by ...