ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell therapies for hematologic, metabolic, and other rare diseases has received from the United States (U.S.) Food and Drug Administration (FDA) the Fast Track Designation for ASC618, a second-generation gene therapy of hemophilia A. In addition, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion for an Orphan Medical Product Designation of ASC618.

In conjunction with the U.S. FDA IND clearance in 2021 and U.S. FDA Orphan Drug Designation in 2020 for ASC618, these regulatory achievements will significantly facilitate the scientific and clinical development of ASC 618’s gene replacement therapies for hemophilia A, reflecting:

A novel second-generation approach to achieve a safe, durable, and stable transgene expression to overcome the challenges of factor VIII replacement therapies.

The viral construct’s critical role with decreasing the therapeutic dose and minimizing cellular stress by increasing synthesis and secretion of factor VIII.

Ruhong Jiang, PhD, Chief Executive Officer at ASC Therapeutics, stated, “We are very pleased with the FDA's and EMA’s regulatory decisions regarding ASC618. This brings us one step closer for providing a truly novel therapeutic approach for hemophilia A, providing potentially a functional cure for patients who currently require life-long care.”

Oscar Segurado, MD, PhD, Chief Medical Officer at ASC Therapeutics, added, “These regulatory milestones culminate years of hard work, dedication, and collaborative efforts by our functional teams. Pre-clinical studies have shown that ASC618 has the potential to reduce therapeutic dosing and increase durability of hemophilia A gene therapy with a novel bioengineered construct that can improve biosynthesis, protein folding, and secretion of factor VIII.”