Le Lézard
Classified in: Health, Science and technology
Subjects: Contract/Agreement, Product/Service

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


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:

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."

Oscar 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 https://www.clinicaltrials.gov/ct2/show/NCT04676048

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

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 https://fnih.org.


These press releases may also interest you

at 07:07
Tenacore is pleased to announce that they have named industry veteran Riley Van Hofwegen as Chief Commercial Officer. In his new role, Van Hofwegen will lead the company's full commercial team and execute the company's sales and marketing plan as...

at 07:05
Precision BioSciences, Inc. (the "Company"), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, today announced the closing of an underwritten offering of 35,971,224 shares of...

at 07:05
Nuvation Bio Inc. , a biopharmaceutical company tackling some of the greatest unmet needs in oncology by developing differentiated and novel therapeutic candidates, today announced the Food and Drug Administration (FDA) has placed a partial clinical...

at 07:05
A new market study published by Global Industry Analysts Inc., (GIA) the premier market research company, today released its report titled "Nanorobotics - Global Market Trajectory & Analytics". The report presents fresh perspectives on opportunities...

at 07:04
Harbour BioMed ("HBM", HKEX: 02142) announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for the B7H4x4-1BB bispecific antibody (HBM7008), which has successfully completed the dosing of...

at 07:00
Medtronic plc today announced that its Board of Directors appointed Lidia Fonseca, Executive Vice President, Chief Digital and Technology Officer at Pfizer, to the Board as an independent director, effective June 27, 2022. Ms. Fonseca will serve on...



News published on 24 may 2022 at 18:05 and distributed by: