ABEO

Abeona Therapeutics Inc. operates as a clinical-stage biopharmaceutical company focused on developing gene and cell therapies for life-threatening rare genetic diseases. Headquartered in New York City, the company concentrates on adeno-associated virus (AAV)-based gene therapies and plasma-based products. Abeona's pipeline includes ABO-102, an investigational gene therapy for MPS IIIA (Sanfilippo syndrome type A), and ABO-101 for MPS IIIB (Sanfilippo syndrome type B), both utilizing AAV9 technology. The company also develops ABO-202 for infantile Batten disease (CLN1) and ABO-201 for juvenile Batten disease (CLN3). In 2022, Abeona faced a significant setback when the FDA placed clinical holds on its MPS IIIA and IIIB programs following safety concerns, prompting the company to restructure operations and reduce headcount. The company maintains its plasma-derived product line through its subsidiary, including Recorlev (levoketoconazole), though this asset has faced its own regulatory challenges. Abeona's strategy centers on advancing its gene therapy candidates through clinical development while managing cash resources carefully. The company operates with a lean organizational structure following recent cost-cutting measures and continues to evaluate strategic alternatives for maximizing shareholder value. As of recent reports, Abeona employs approximately 20-30 full-time employees and maintains research collaborations with academic institutions focused on rare disease therapeutics.