Gene Therapy

Sarepta Therapeutics Presented Positive Preliminary Results from the First Three Children Dosed in the Phase 1/2a Gene Therapy Micro-dystrophin Trial to Treat Patients with Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, said that at the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from its Phase 1/2a gene therapy clinical trial assessing AAVrh74.MHCK7.micro-Dystrophin in individuals with Duchenne muscular […]

Cell Therapy

Researchers Encouraged by Continued Signs of Tolerability and Disease Modification in Gene Therapy Trial for Sanfilippo Syndrome

Researchers from Abeona Therapeutics Inc, a clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases, provided, an update on clinical results through 30 days post-injection for the completed low-dose cohort (n=3) in the ongoing Phase 1/2 trial for ABO-102 (AAV-SGSH) at the Orphan Drugs & Rare Disease Conference (London, UK). The first-in-man […]

Cell Therapy Clinical Trials

Understanding Natural History Studies in Drug Development for Rare Diseases

Q &A with Michelle Berg, Vice President, Patient Advocacy, Abeona Therapeutics Inc. Developing a new drug for patients with a disease, particularly when it’s a disease that affects a relatively small number of patients,  poses unique challenges that do not apply to medical therapies for more prevalent diseases. However, an increasingly common, and particularly important […]