RARE Daily

FDA Grants Fast Track Designation to ASC Therapeutics’ Gene Therapy for Hemophilia A

March 30, 2022

Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation for ASC Therapeutics’ 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.

ASC618 is an AAV8-based gene therapy for the treatment of hemophilia A, affecting approximately 1 of every 5000 live-born males. ASC618 incorporates a novel liver-specific promoter and a bioengineered, codon-optimized B domain-deleted FVIII variant. In preclinical studies, ASC618 exhibits at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with native human FVIII bioengineered gene constructs. The company says ASC618 has the potential to increase durability of clotting factor biosynthesis and secretion by minimizing cellular stress and induction of the unfolded protein response, which may lead to diminished FVIII production from liver cells.

“Preclinical 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,” said Oscar Segurado, chief medical officer at ASC Therapeutics.

ASC will conduct a phase 1/2 clinical trial to evaluate the safety, tolerability, and preliminary efficacy of ASC618. The program received IND clearance from the U.S. Food and Drug Administration in 2021.

“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,” said Ruhong Jiang, CEO of ASC Therapeutics.

Fast track is a process designed by U.S. FDA to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier.

Photo: Ruhong Jiang, CEO of ASC Therapeutics

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