Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases ...

REGENXBIO touted data from the Phase III portion of a study that showed higher benefit that Elevidys.

Gemma Biotherapeutics ("GEMMABio"), a clinical‑stage, global, genetic medicines company, today announced the presentation of preclinical data supporting candidate declaration for GB703, a novel, ...

Capricor Therapeutics has fallen out with its commercial partner for Duchenne muscular dystrophy (DMD) cell therapy ...

Companies are adopting diverse approaches to reducing unwanted toxicities associated with cell and gene therapy.

Deramiocel BLA under active FDA review; PDUFA target action date of August 22, 2026; labeling discussions expected to commence soonHOPE-3 Phase 3 ...

Eleven clinical trial sites in BASECAMP currently active; remaining planned sites to be activated throughout Q2 2026BASECAMP remains on track to ...

The FDA turned away Ebvallo in January, taking issue with the design of the registrational trial. In a recent meeting, however, the agency agreed that the study could in fact support the cell ...

The investigational cell therapy deramiocel slowed upper limb functional decline in boys and young men with Duchenne muscular dystrophy, data from the phase III HOPE-3 trial showed. At 12 months, the ...

Co. Ltd. is paying $60 million up front for an option to secure exclusive rights to Aribio Co. Ltd.’s oral phase III-stage Alzheimer’s disease (AD) therapy, AR-1001. The option fee plus license ...

R. Jude Samulski, Ph.D., was honored by the American Society of Gene + Cell Therapy (ASGCT) with its Founders Award at the ...

With an FDA decision date looming for its Duchenne muscular dystrophy (DMD) treatment deramiocel, Capricor Therapeutics has ...