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Abeona Therapeutics Preparing to Submit BLA for Rare Skin Condition Cell Therapy

NEW YORK – Abeona Therapeutics on Wednesday said it's preparing a biologics license application to submit to the US Food and Drug Administration for its investigational cell therapy EB-101 early this fall, following a positive pre-BLA meeting with the agency.

The autologous engineered cell therapy is being developed as a treatment to heal wounds on patients with recessive dystrophic epidermolysis bullosa (RDEB), a recessively inherited form of the rare skin condition. RDEB is caused by a lack of type 7 collagen protein that results in fragile skin with patients easily experiencing blisters, sores, and tears.

The cell therapy, consisting of keratinocytes derived from skin biopsies and converted with a recombinant retrovirus to contain a full-length COL7A1 gene that expresses C7, is administered through a one-time surgical application to a patient's wound sites.

Cleveland-based Abeona said it "reached alignment with the FDA" in a recent pre-BLA meeting that clinical efficacy and safety data for the drug will be adequate to support a BLA submission. The FDA requested additional data on topics like EB-101 potency and identity assays, chemistry, and manufacturing.

In June, the company had pushed back its pre-BLA meeting date for EB-101, the firm's lead clinical program, after receiving feedback from the FDA that it needed additional data to establish comparability for retroviral vectors manufactured at Indiana University and in-house at Abeona, both of which had been used in clinical trials.

"We are pleased with the outcome of the pre-BLA meeting for EB-101 and believe that we have aligned with the FDA on what is needed for our upcoming BLA submission," Abeona CEO Vish Seshadri said in a statement. "We are focused on gathering and packaging the existing data over the coming weeks to meet the agency's expectations."

The FDA previously granted regenerative medicine advanced therapy, breakthrough therapy, orphan drug, and rare pediatric disease designations to EB-101.