This segment focuses on the research, development, and clinical application of gene and cell therapies for rare genetic diseases. Abeona utilizes adeno-associated virus (AAV) vectors and autologous, gene-corrected cell therapies to target the underlying genetic causes of these conditions. Key research activities include vector engineering, cell modification, and preclinical studies to optimize therapeutic efficacy and safety. The segment addresses therapeutic areas such as recessive dystrophic epidermolysis bullosa (RDEB) and Sanfilippo syndrome. The goal is to provide potentially curative treatments that improve patient outcomes and quality of life. This segment leverages the AIM vector platform and other proprietary technologies to enhance gene delivery and expression. Regulatory and clinical aspects are central, with ongoing clinical trials and efforts to secure FDA approval for lead product candidates. Partnerships and collaborations with research institutions and patient advocacy groups support the advancement of these therapies.

This segment is dedicated to developing and commercializing therapies for rare genetic diseases with high unmet medical needs. Abeona's approach involves identifying and validating novel therapeutic targets, developing innovative gene and cell therapy products, and conducting rigorous clinical trials to demonstrate safety and efficacy. The segment focuses on diseases such as Sanfilippo syndrome types A and B, CLN3 disease, and cystic fibrosis. Research and development efforts include optimizing gene delivery methods, enhancing gene expression, and developing biomarkers to monitor treatment response. The segment aims to provide transformative therapies that address the underlying genetic defects and improve the lives of patients and their families. Market positioning involves establishing strong relationships with key opinion leaders, patient advocacy groups, and regulatory agencies. Future opportunities include expanding the pipeline to address additional rare genetic diseases and leveraging advanced technologies to develop more effective and personalized therapies.