ASC Therapeutics receives key regulatory designations in the United States and Europe to advance its second-generation gene therapy for hemophilia A

MILPITAS, Calif.–(BUSINESS WIRE)–ASC Therapeutics, a private biopharmaceutical company pioneering the development of transformative in vivo gene replacements, gene editing and allogeneic cell therapies for hematological, metabolic and other rare diseases , has received from the United States Food and Drug Administration (FDA) granted Fast Track designation to ASC618, a second-generation gene therapy for hemophilia A. In addition, the committee of Orphan Drugs (COMP) from the European Medicines Agency (EMA) has issued a positive opinion for an orphan medical product designation of ASC618.

Together with the IND clearance from the US FDA in 2021 and the US FDA Orphan Drug Designation in 2020 for ASC618, these regulatory achievements will significantly facilitate the scientific and clinical development of replacement therapies for ASC 618 genes for haemophilia A, reflecting:

Ruhong Jiang, PhD, CEO of ASC Therapeutics, said, “We are very pleased with the FDA and EMA regulatory decisions regarding ASC618. This brings us one step closer to a truly novel therapeutic approach for hemophilia A, potentially providing a functional cure for patients who currently require lifelong care.

Oscar Segurado, MD, PhD, Chief Medical Officer at ASC Therapeutics, added, “These regulatory milestones cap years of hard work, dedication and collaborative efforts by our functional teams. Preclinical studies have shown that ASC618 has the potential to reduce therapeutic doses and increase the durability of hemophilia A gene therapy with a novel bioengineered construct that may improve biosynthesis, refolding proteins and the secretion of factor VIII.

About the ASC618

ASC618 is an AAV8-based gene therapy for the treatment of haemophilia A, affecting approximately 1 in 5,000 live-born men1. ASC618 incorporates a novel liver-specific promoter and bioengineered B-domain deleted FVIII variant2; in preclinical studies, ASC618 exhibits at least a 10-fold increase in FVIII biosynthesis and secretion compared to bioengineered native human FVIII constructs. ASC618 has the potential to increase the durability of clotting factor biosynthesis and secretion by minimizing cellular stress and induction of the unfolded protein response, which may result in decreased FVIII production from cells liverworts.

ASC Therapeutics will conduct a Phase 1/2 clinical trial to assess the safety, tolerability and preliminary efficacy of ASC618. The program has received IND clearance from the United States Food and Drug Administration. The study design is available at https://www.clinicaltrials.gov/ct2/show/NCT04676048

About ASC Therapeutics

ASC Therapeutics is a pioneering biopharmaceutical company in the development of gene replacement therapies, in vivo gene editing and allogeneic cell therapies for hematological, metabolic and other rare diseases. Led by a management team comprised of industry veterans with significant global experience in gene and cell therapy, ASC Therapeutics is developing multiple therapeutic programs based on four technology platforms: 1) In vivo gene therapy for inherited blood clotting disorders, initially focusing on ASC618 for hemophilia A, for which US FDA IND clearance has been received; 2) In vivo gene therapy in metabolic disorders, initially focused on maple syrup urinary disease, in collaboration with the universities of Massachusetts and Pennsylvania; 3) In vivo gene editing, initially focusing on ASC518 for haemophilia A; and 4) allogeneic cell therapy, with the first indication with deciduous stromal cell therapy for acute steroid-refractory graft-versus-host disease. To learn more, go to https://www.asctherapeutics.com/.

The references

1 Steven W. Pipe, Gil Gonen-Yaacovi and Oscar G. Segurado. Gene therapy for hemophilia A: current and next generation approaches, expert opinion on biological therapy. 2022; DOI: 10.1080/14712598.2022.2002842

2Brown HC, Wright JF, Zhou S, et al. Bioengineered clotting factor VIII provides long-term correction of murine hemophilia A after administration of a liver-directed adeno-associated viral vector. Methods Mol Ther Clin Dev. 2014;1:14036

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