Nanoscope Therapeutics has announced the publication of highly encouraging Phase 1/2a clinical trial data in the esteemed journal Molecular Therapy, showcasing significant vision restoration in patients suffering from retinitis pigmentosa (RP), a debilitating...
Capsida Biotherapeutics’ experimental gene therapy, CAP-004, for Friedreich’s ataxia (FA), has demonstrated promising preclinical results in nonhuman primates. Research presented by Capsida at the 2025 MDA Clinical & Scientific Conference showed that a...
Epicrispr Biotechnologies today announced the first close of its Series B financing, securing $68 million to support the clinical development of EPI-321, a first-in-class, disease-modifying therapy for facioscapulohumeral muscular dystrophy (FSHD), a genetic...
SineuGene Therapeutics Co., Ltd. has received U.S. FDA clearance for its Investigational New Drug (IND) application for SNUG01, a first-in-class TRIM72-targeted gene therapy for amyotrophic lateral sclerosis (ALS). This clearance allows for a global Phase I/IIa...
Regenxbio has presented biomarker data from its Duchenne muscular dystrophy gene therapy trial, demonstrating near-normal microdystrophin expression in a 3-year-old patient. The company reported at the Muscular Dystrophy Association’s annual scientific...
