ASC Therapeutics Joins U.S. Consortium of Gene Therapy Partners Including the NIH and FDA to Accelerate Treatments for Rare Diseases

Public, private and non-profit organizations have joined forces to create a standardized approach that will reduce up-front costs and lower barriers to developing novel gene therapies for rare and ultra-rare diseases

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 has joined as a full partner the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC) including the National Institutes of Health (NIH) and the Food and Drug Administration (FDA), managed by the Foundation for the National Institutes of Health (FNIH).

The AMP® BGTC brings together partners from the public, private, and non-profit sectors to foster development of gene therapies intended to treat rare genetic diseases, which affect populations too small for viable commercial development. BGTC has defined the following objectives:

  • Basic research: to better understand the basic biology of adeno-associated viruses (AAV) in order to optimize bioproduction technologies and increase their therapeutic efficacy.
  • Clinical research: to create a development model to shorten the time between preclinical studies and human trials.
  • Bioproduction: to design a standard set of analytical tests for the manufacture of viral vectors broadly applicable to different manufacturing methods.
  • Regulatory: to streamline regulatory requirements and processes and expedite approvals from the FDA.

Ruhong Jiang, PhD, Chief Executive Officer at ASC Therapeutics, stated, “We are truly honored to be joining this public-private partnership with 22 private sector partners that will facilitate scientific, clinical and regulatory advances that will ultimately benefit the entire field. We are proud to collaborate with NIH, FDA, industry and non-profit organizations focused on developing transformative therapies for patients in need.”

Accolade Segurado, MD, PhD, Chief Medical Officer at ASC Therapeutics, added, “This Consortium fits perfectly with our mission and vision to provide functional cures for patients with hemophilia and maple syrup urinary disease who currently require life-long care. Joining AMP BGTC will significantly optimize our gene therapy technologies, operations and processes while we will be contributing whole-heartedly to the ultimate goal of helping patients with rare diseases.”

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 substantial 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.

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 is available at

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About The AMP Bespoke Gene Therapy Consortium (BGTC)

The Accelerating Medicines Partnership (AMP®) program is a public-private partnership between the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), and multiple public and private organizations. The AMP BGTC aims to develop platforms and standards that will speed the development and delivery of customized or ‘bespoke’ gene therapies that could treat the millions of people affected by rare diseases.

About The Foundation for the National Institutes of Health

The FNIH creates and manages alliances with public and private institutions in support of the mission of the NIH. The FNIH works with its partners to accelerate biomedical research and strategies against diseases and health concerns in the United States and across the globe. Established by Congress in 1990, the FNIH is a not-for-profit 501(c)(3) charitable organization. For additional information about the FNIH, please visit


Accolade Segurado

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