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 ...

Cure Rare Disease (CRD), a non-profit biotechnology organization, today announced a landmark partnership with the LGMD2L ...

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 ...

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 ...

New review highlights evolving Pompe disease management, from screening to next-generation therapies and monitoring. Read ...

The MarketWatch News Department was not involved in the creation of this content. -- Itvisma (onasemnogene abeparvovec-brve) demonstrated improved motor function and stabilization in patients ...

Enrollment for liMeliGhT, the first and largest gene therapy registrational trial for broad retinitis pigmentosa (RP) patients, was completed, ...

About the FALCON Clinical Trial FALCON is a first-in-human, open-label, multi-center Phase 1b clinical trial designed to evaluate the safety and tolerability of SGT-212 in participants aged 18-40 who ...

Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...

Novartis has received approval from the U.S. Food and Drug Administration for Itvisma, a treatment for spinal muscular atrophy. Dr. John Day is professor of neurology and pediatrics, director of the ...

Spinal muscular atrophy (SMA) is a disease that causes progressive degeneration in the nerve cells that control muscles, thereby causing muscle weakness and eventually death. SMA affects approximately ...