Company News

Expanded relationship will build upon established processes initiated in 2019 to meet ASC Therapeutics clinical program WILMINGTON, Mass.–(BUSINESS WIRE)–Charles River Laboratories International, Inc. (NYSE: CRL) and ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in vivo gene replacement, gene editing and allogeneic cell therapies, today announced they have agreed to manufacture ASC618, a … Read more

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 … Read more

First known bovine model used to demonstrate safety and efficacy in gene replacement therapy MSUD is a severe genetic disease with liver transplantation or dietary restriction as the only treatments currently available CORRECTION…by ASC Therapeutics MILPITAS, Calif.–(BUSINESS WIRE)–Please replace the release dated May 16, 2022 with the following corrected version due to multiple revisions. “The … Read more

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 … Read more

The U.S. Food and Drug Administration (FDA) has given fast track designation to ASC618, an investigational one-time gene therapy for hemophilia A in the pipeline of ASC Therapeutics. Fast track status works to speed the development and regulatory review of treatments for serious disorders that show a potential to fill an unmet need. The Committee for Orphan Medicinal Products … Read more

We are very pleased with the FDA’s and EMA’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.

Experienced in manufacturing of next-generation hemophilia gene therapies at uniQure Experienced in transformational cell culture manufacturing Consolidates global leadership team of gene and cell therapy industry veterans September 27, 2021 08:00 PM Eastern Daylight Time MILPITAS, Calif.–(BUSINESS WIRE)–ASC Therapeutics, a privately held biopharmaceutical company pioneering the development of transformative in-vivo gene replacement, gene editing and … Read more

Second-generation gene therapies can increase protein synthesis and secretion Minimizing cellular stress can increase durability of liver-targeted gene therapies A peer-reviewed, open-access, comprehensive seminal review 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 for hematologic, metabolic and other rare diseases … Read more

UMass Medical School researchers Guangping Gao, PhD, and Dan Wang, PhD, are working with ASC Therapeutics, a privately held biopharmaceutical company developing in-vivo gene replacement, gene editing and allogeneic cell therapies, to bring a gene therapy for maple syrup urine disease (MSUD) to the clinic. “Our significant progress developing an AAV-based therapy for MSUD will … Read more