pz-cel for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

pz-cel is an autologous, engineered cell therapy for RDEB, a rare connective tissue disorder 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 pz-cel 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. 

pz-cel has the potential to be the only durable treatment to address large chronic wounds, which are the most painful and debilitating. In a Phase 1/2a clinical trial, pz-cel 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 pz-cel 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. pz-cel 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 pz-cel 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.