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. […]

Gene Therapy Pharmaceutical Business News

Abeona Receives FDA Regenerative Medicine Advanced Therapy Designation for Gene Therapy in Epidermolysis Bullosa

Gene therapy developer, Abeona Therapeutics, announced that the FDA has granted Regenerative Medicine Advanced Therapy designation to EB-101, a gene-corrected autologous cell therapy product for patients with recessive dystrophic epidermolysis bullosa (RDEB). Abeona`s EB-101 product is an autologous, ex-vivo gene-corrected cell therapy in which the COL7A1 gene is inserted into a patient`s own skin cells (keratinocytes) for […]

Gene Therapy Genetic Disorders

Novel Gene Therapy Provides Significant Wound Healing in Severe Form of Epidermolysis Bullosa

Recent data presented at the Society for Investigative Dermatology (SID) conference demonstrated that EB-101, a gene therapy provided significant wound healing in patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB), a severe form of epidermolysis bullosa (EB). RDEB is a subtype of an inherited genetic skin disorder characterized by chronic skin blistering, open and painful wounds, […]

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 […]

Gene Therapy

Gene Therapy Company Acquires Next Generation AAV Gene Therapy Vector Platform from The University of North Carolina at Chapel Hill

The University of North Carolina at Chapel Hill has licensed the exclusive worldwide rights of a next generation gene therapy AAV capsid portfolio to Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases. The AIM™ vector system is a next generation platform of AAV capsids capable of widespread […]

Gene Therapy rare diseases

New Collaboration focuses on Gene Therapy to Tackle Epidermolysis Bullosa (EB) Treatments

Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company focused on delivering gene and plasma-based therapy for life-threatening rare diseases, EB Research Partnership (EBRP) and EB Research Medical Foundation (EBMRF) announced today a collaboration focusing on gene therapy treatments for epidermolysis bullosa (EB), a group of devastating rare genetic skin disorders impacting children; characterized by skin blisters […]

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 […]