ASC Therapeutics Announces Podium and Poster Presentations of IND-Enabling Studies for ASC618, a Second-Generation Gene Therapy for Hemophilia A, at the 25th ASGCT Meeting, May 16-19, 2022 in Washington, D.C.

• Podium presentation highlights significant transduction and transgene expression in pharmacokinetic studies in mice, non-human primates and a humanized liver model
• Poster presentation focuses on pharmacology, toxicology and safety studies
• Poster presentation focuses on the development of a novel transduction assay to evaluate in-vitro relative Infectivity

MILPITAS, Calif.--(BUSINESS WIRE)-- ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and allogeneic cell therapies, will provide a comprehensive review of IND-enabling studies of ASC618, a second-generation gene therapy for hemophilia A, at the 25th Annual Meeting of The American Society of Gene and Cell Therapy (ASGCT) held May 16-19, 2022 in Washington, D.C. These studies were included in a filing to the U.S. Food and Drug Administration (FDA) leading to IND clearance of ASC618.

A podium presentation entitled “ASC618, a Second-Generation FVIII Gene Therapy for Hemophilia A, Exhibits Major Transduction and Transgene Expression in the Target Liver Tissues: Results of IND-Enabling Pharmacokinetics Studies in Mice and Non-Human Primates” will be given by Dr. Chengtao Yang, ASC618 Group Leader and Principal Scientist, on Thursday, May 19 (Room 102 A/B, 11:30 AM - 11:45 AM ET, abstract number: 1262).

A comprehensive summary of ASC618 preclinical development and dose translation approach will be presented in a poster: “Pharmacology, Toxicology and Safety Studies of ASC618, a Second-Generation Factor VIII Gene Therapy for Hemophilia A Development” on Tuesday, May 17 (Hall D, Poster Board Number: Tu-288, 5:30 PM - 6:30 PM ET, abstract number: 783).

In addition, a poster: “New Transduction Assay to Evaluate In-Vitro Relative Infectivity of ASC618” will describe the development of a more accurate and precise assay as an alternative to TCID50 to support ASC618 product release and characterization, will also be presented on Tuesday, May 17 (Hall D, Poster Board Number: Tu-287, 5:30 PM - 6:30 PM ET, abstract number: 782).

Ruhong Jiang, PhD, founder and CEO of ASC Therapeutics, stated: “We are very proud of the achievements and rapid progress our therapeutics team has made thus far and expect to see great clinical data very soon.”

Zoya Gluzman-Poltorak, PhD, VP Therapeutic Development at ASC Therapeutics, added: “I am honored to lead a team of extremely passionate and talented scientists who completed the ASC618 IND-enabling studies in one year culminating in a flawless IND clearance. We are thrilled to bring a second-generation gene therapy treatment to patients with Hemophilia A.”

The Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) is the world’s largest gathering of gene and cell therapy professionals and provides an international forum where the latest scientific discoveries are presented and discussed. Thousands of cell and gene therapy developers, including basic and clinical investigators represent the academic, regulatory, and biotechnology sectors.

About ASC618

ASC618 is a second-generation gene therapy for the treatment of hemophilia A. ASC618 incorporates a novel liver-specific promoter and a bioengineered, codon-optimized B domain-deleted FVIII variant¹; 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. 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 cells².

ASC Therapeutics will conduct a phase 1/2 clinical trial to evaluate the safety, tolerability, and preliminary efficacy of ASC618. The program received IND clearance, as well as Fast-Track and Orphan Drug Designations from the U.S. Food and Drug Administration. The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has also granted an Orphan Medical Product Designation. The study design will be presented at the International Society of Thrombosis and Hemostasis (ISTH) Congress in London, U.K in July 2022 and is available at https://www.clinicaltrials.gov/ct2/show/NCT04676048.

About ASC Therapeutics

ASC Therapeutics is a biopharmaceutical company pioneering 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 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 of inherited blood clotting disorders, initially focusing on ASC618, a second generation gene replacement treatment for hemophilia A; 2) In-vivo gene therapy in metabolic disorders, initially focusing on Maple Syrup Urine Disease; 3) In-vivo gene editing, initially focusing on ASC518 for hemophilia A; and 4) Allogeneic cell therapy, initially focusing on a Decidua Stromal Cell-based therapy for steroid-refractory acute Graft-versus-Host Disease.

To learn more please visit https://www.asctherapeutics.com/.

References

1. Brown HC, Wright JF, Zhou S, et al. Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery. Mol Ther Methods Clin Dev. 2014;1:14036
2. Steven W. Pipe, Gil Gonen-Yaacovi & Oscar G. Segurado. Hemophilia A gene therapy: current and next-generation approaches, Expert Opinion on Biological Therapy 2022 Jan 6;1-17, DOI: 10.1080/14712598.2022.2002842

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