Abeona Therapeutics (Nasdaq: ABEO) is a fully-integrated gene and cell therapy company at the forefront of the rapidly-advancing field of genetic medicine. The Company's multi-platform expertise across the manufacture, delivery, development, and discovery of novel gene and cell therapies has it uniquely positioned for success. Underpinning the Company's robust pipeline is its fully-operational manufacturing facility producing therapies and vectors for preclinical and clinical studies. Abeona is also developing the AIM™ Vector Platform: 100+ next-generation AAV capsids for delivering gene therapies targeting a wide range of organs and multiple routes of delivery. A robust and diverse pipeline is led by a novel gene-corrected cell therapy poised to enter Phase 3 in mid-2019 and complemented by one-time gene therapy candidates across four lysosomal storage diseases. Several preclinical discoveries are led by an emerging program in cystic fibrosis that uses the AIM vector platform and a capsid that has shown potential across inherited and acquired retinal diseases.

Gene Therapy Human-derived plasma proteins Biotechnology Pharma Oncology supportive care Ovarian Cancer Alpha-1 deficiency IVIG Ultra orphan proteins Oral Mucositis Rare Disease Sanfiippo Syndrome Type A Sanfiippo Syndrome Type B MPS IIIA juvenile Batten Disease Fanconi anemia Infantile Batten Disease Epidermolysis Bullosa Lysosomal Storage Disease Cell Therapy Pediatric Rare Diseases manufacturing B2B Biotech SaaS Commercial Physical Research