EB-101 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

EB-101 is an autologous, engineered cell therapy for RDEB, a rare connective tissue disorder without an approved treatment in which patients suffer with severe epidermal wounds that impact the length and quality of their lives. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce Type VII collagen that helps anchor the dermal and epidermal layers of the skin.

Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into an RDEB patient’s own skin cells (keratinocytes) and transplanting them back to the patient to enable normal Type VII collagen expression and skin function. 

EB-101 has the potential to be the first approved therapy for RDEB and the only durable treatment to address large chronic wounds, which are the most painful and debilitating. In a Phase 1/2a clinical trial, EB-101 was well-tolerated and showed evidence of multi-year wound healing and pain reduction after treatment with a mean follow-up of 5.9 years and a maximum of eight years. Continuous Type VII collagen expression was observed more than two years after treatment, and no drug-related serious adverse events have been observed to date.

The pivotal Phase 3 VIITAL™ study evaluated the efficacy, safety and tolerability of EB-101 in RDEB patients. The VIITAL™ study met its two co-primary efficacy endpoints demonstrating statistically significant, clinically meaningful improvements in wound healing and pain reduction in large chronic RDEB wounds. EB-101 was shown to be well-tolerated with no serious treatment-related adverse events observed, consistent with past clinical experience. Learn more.

Abeona holds several regulatory designations in the U.S. and EU for EB-101 that allow increased interactions and guidance from the FDA and EMA. In the U.S., these include Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and Rare Pediatric Disease designations and Orphan Drug designation in both the U.S. and EU.

ABO-50X for Retinal Diseases

Abeona is exploring the potential of ABO-50X for the treatment of monogenic eye disorders. ABO-50X is a group of AAV-based investigational products that were developed by Abeona researchers that leverage the next-generation AIM^TM vector platform along with unique transgenes to tackle rare ocular diseases with high unmet need. Non-human primate data suggest that next-generation AIM^TM AAV vectors can be used to efficiently target the retina, optic nerve, retinal pigmented epithelium and macula after direct ocular injection, creating the potential for new pipeline candidates that can address multiple eye disorders.