Cell Therapy

Abeona Therapeutics Reports Top-Line Data from Phase 1/2 Gene Therapy Trial in Sanfillippo Syndrome

Abeona Therapeutics Inc., a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, announced updated clinical data from the ongoing Phase 1/2 trial for ABO-102 (AAV-SGSH), the company’s investigational gene therapy for the treatment of Sanfilippo syndrome Type A (MPS IIIA), a rare autosomal-recessive lysosomal storage disease. […]

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 […]