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# Abeona Refiles BLA for Gene Therapy
- URL: https://www.fdaweb.com/abeona-refiles-bla-for-gene-therapy/
- Published: 2024-10-29T12:00:00.000Z
- Updated: 2026-09-14T14:45:32.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5158075

Abeona Therapeutics has resubmitted a BLA for prademagene zamikeracel (pz-cel), an autologous cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB). The resubmission follows an April complete response letter that requested additional information to satisfy chemistry manufacturing and controls requirements ([see earlier story](https://www.fdaweb.com/complete-response-on-abeonas-pz-cel-bla/)).

“We have incorporated the agency’s feedback and are confident that our resubmission package addresses all the chemistry manufacturing and controls items identified in the complete response letter, including observations from the completed pre-license inspection of our manufacturing facility,” Abeona says.

According to the company, the resubmission is based on data from the pivotal Phase 3 VIITAL study (NCT04227106) and a Phase 1/2a study (NCT01263379). “Both studies revealed that a one-time application of pz-cel on large and chronic wounds delivered sustained wound healing and pain reduction,” it adds.

RDEB is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life, Abeona says. “People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin,” it says. Pz-cel is designed to “incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells and enable long-term gene expression by using a retroviral vector to stably integrate into the dividing target cell genome,” it adds.