Nanoscope Therapeutics Publishes Breakthrough Clinical Data Demonstrating Significant Vision Restoration in Retinitis Pigmentosa with Novel AAV-based MCO-010 Gene Therapy
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’s AAV CAP-004 Gene Therapy Shows Promise for Friedreich’s Ataxia in Primate Study at MDA Conference
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 single...
FDA Grants Fast Track to Sanofi’s Chlamydia mRNA Vaccine
Sanofi is making significant strides in the fight against chlamydia, the most common sexually transmitted bacterial infection, for which there are currently no approved preventative vaccines. The French pharmaceutical group has recently been granted a fast-track...
Epicrispr Biotechnologies Announces $68M Series B to Advance First-in-Class FSHD Epigenetic Therapy to Clinic
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...
Turn Bio Acquires Vesigen’s Drug Delivery Tech to Expand mRNA Therapy Reach
Turn Biotechnologies, a startup focused on mRNA therapies for cellular rejuvenation, has acquired an experimental drug delivery technology from Harvard University and Vesigen Therapeutics, according to an exclusive report by Endpoints News. The acquired technology,...
SineuGene’s SNUG01: First TRIM72 Gene Therapy for ALS Enters Clinical Trials
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...
Verve Therapeutics Expands Gene Editing Trial for Cholesterol Treatment to the U.S.
Verve Therapeutics has received clearance from the U.S. Food and Drug Administration (FDA) to expand its ongoing clinical trial of VERVE-102, an experimental gene editing therapy aimed at lowering cholesterol and reducing the risk of heart disease, the company...
Regenxbio Presents Positive Duchenne Gene Therapy Data
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 conference that...
Optieum Biotechnologies Receives $39M AMED Grant for CAR-T Development
Optieum Biotechnologies Inc., a biopharmaceutical company focused on developing innovative cancer immunotherapies, has secured a significant funding boost through a grant of up to $39 million from the Japanese Agency for Medical Research and Development (AMED). This...
Sickle Cell Patient Achieves Remission with Gene Therapy
Sebastien Beauzile, a 21-year-old from New York, has achieved remission from sickle cell disease following treatment with Lyfgenia, a gene therapy developed by Bluebird Bio, at Northwell Health/Cohen Children’s Medical Center. Administered on December 17, 2024, the...
AstraZeneca to Acquire EsoBiotec for Cell Therapy Advancement
AstraZeneca (LSE/STO/Nasdaq: AZN) has announced the acquisition of EsoBiotec SA, a Belgian biotechnology company pioneering in vivo cell therapies. EsoBiotec's Engineered NanoBody Lentiviral (ENaBL) platform, which has demonstrated promising early clinical activity,...
Dyne’s DYNE-251: 18-Month Data Shows Unprecedented DMD Improvement
WALTHAM, Mass., March 16, 2025 -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company developing life-transforming therapies for genetically driven neuromuscular diseases, today announced positive long-term clinical data from its ongoing Phase 1/2 DELIVER...
Popular Services
AAV Packaging Service
AAV Analytical Service
Vector Design