Gene Therapy Gets Fast Track Status for Hemophilia A

The Food and Drug Administration (FDA) has granted Fast Track designation to ASC618 for the treatment of hemophilia A.

ASC618 is an adeno-associated virus (AAV)-based gene therapy which incorporates a novel liver-specific promoter and a bioengineered, codon-optimized B domain-deleted FVIII variant. The Company believes that ASC618 may potentially increase the 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.

The designation is supported by data from preclinical studies which showed that ASC618 has been shown to have at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with native human FVIII bioengineered constructs. The Company will investigate the safety, tolerability, and preliminary efficacy of ASC618 in a phase 1/2 trial (ClinicalTrials.gov Identifier: NCT04676048).

Ruhong Jiang, PhD, CEO at ASC Therapeutics stated, “We are very pleased with the FDA’s and [European Medicines Agency’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.”

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