The first patient has been dosed in ASC Therapeutics clinical trial of ASC618, the company’s investigational AAV8 vector-based  gene therapy for patients with severe and moderately severe hemophilia A.

Investigators of the phase I/II trial are evaluating safety, tolerability, and preliminary efficacy of ASC618, which was granted Fast Track Designation by U.S. Food and Drug Administration (FDA) back in April 2022. This designation is meant to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The therapy has also received Orphan Drug status from the FDA.

“This is a special day for ASC Therapeutics and for patients with hemophilia A hoping to access a more durable and affordable second-generation gene therapy,” said ASC Therapeutics’ Chief Medical Officer Oscar Segurado, MD, PhD. “I am grateful to our team members and the clinical research team at Arkansas Children’s Hospital involved in the successful dosing of our first patient.”

The patient was dosed at the Arkansas Children’s Hospital, where Shelley Crary, MD, Pediatric Hematologist/Oncologist, is principal investigator. “Treating our first patient with ASC618 reinforces our focus on providing cutting edge therapeutic modalities to our patients with hemophilia A, said Crary. “We are now assessing in a clinical setting the relevance of a novel one-and-done gene therapy that may replace lifelong, burdensome, and expensive treatments to manage hemophilia A.”

Learn more about the study.

Source: BioSpace, November 28, 2023