NOTE 12 – SUBSEQUENT EVENTS

Biologic License Application

On October 28, 2024, the Company resubmitted its Biologics License Application (“BLA”) to the U.S. Food and Drug Administration (“FDA”) for pz-cel, the Company’s investigational autologous cell-based gene therapy, as a potential new treatment for patients with RDEB. The FDA notified the Company on November 8, 2024 that the BLA was accepted for review, with an assigned Prescription Drug User Fee Act (“PDUFA”) target action date of April 29, 2025.

The BLA resubmission follows the Company’s Type A meeting in August 2024, where the Company aligned with the FDA on the content of the resubmission, including additional information to satisfy all Chemistry Manufacturing and Controls (“CMC”) requirements outlined in the Complete Response Letter (CRL) received in April 2024. In the CRL, the FDA required that certain additional information needed to satisfy CMC requirements be provided before the application could be approved. The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support the approval of pz-cel. The BLA resubmission is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study (NCT04227106) and a Phase 1/2a study (NCT01263379).

Lease Agreement

On October 18, 2024, the Company signed a lease for 16,566 square feet of office space at 6700 Euclid Avenue, Cleveland, Ohio. The lease commences on January 1, 2025 and the lease term matches the term for the Company’s existing 6555 Carnegie Avenue facility. The additional space at the 6700 Euclid Avenue facility will allow the Company to convert office space at the 6555 Carnegie Avenue facility into additional manufacturing space to increase pz-cel manufacturing capacity.