Abeona Therapeutics
Abeona Therapeutics specializes in developing cell and gene therapies for severe diseases, with a focus on its Phase 3 EB-101 therapy for recessive dystrophic epidermolysis bullosa and AAV-based therapies for ophthalmic conditions.
Company Overview
Abeona Therapeutics develops cell and gene therapies aimed at treating serious diseases. The company's lead clinical program, EB-101, is an investigational autologous, gene-corrected cell therapy currently in Phase 3 development for treating recessive dystrophic epidermolysis bullosa. Abeona's portfolio also includes AAV-based gene therapies targeting unmet medical needs in ophthalmic diseases.
Lead Clinical Program EB-101
EB-101 is Abeona Therapeutics' lead clinical program, focusing on treating recessive dystrophic epidermolysis bullosa. This investigational autologous, gene-corrected cell therapy is currently in Phase 3 development. EB-101 aims to address a critical need in managing this severe skin disorder.
AAV-Based Gene Therapies
Abeona Therapeutics develops AAV-based gene therapies designed to treat ophthalmic diseases with high unmet medical needs. These therapies use AAV vectors to deliver genetic payloads to specific tissues, potentially addressing conditions that currently have limited treatment options.
Manufacturing Capabilities
Abeona Therapeutics possesses significant manufacturing capabilities for both retrovirus and AAV-based gene therapies. The company can produce commercial-grade retrovirus manufacturing and scale AAV-based gene therapies and vectors, supporting its extensive development pipeline.
Regulatory Designations
Abeona Therapeutics holds several regulatory designations in the U.S. and EU for pz-cel (prademagene zamikeracel). These designations facilitate increased interactions and guidance from regulatory bodies such as the FDA and EMA, potentially accelerating development and approval processes.
AIM™ Vector Technology
The AIM™ Vector technology developed by Abeona Therapeutics utilizes AAV biology to deliver genetic payloads selectively to various tissues. This platform aims to evade immune responses typically encountered with naturally occurring AAV vectors, potentially increasing the efficacy and safety of gene therapies.