ASC-618 for Patients With Hemophilia A: Ruhong Jiang, PhD

"We are really eager to see how the first dosing of [a] patient [goes] because everything comes from patient data." Ruhong Jiang, PhD, cofounder, president, and chief executive officer of ASC Therapeutics

Fresh off its FDA Fast Track Designation and a positive opinion by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products for its AAV8-based gene therapy for patients with hemophilia A, ASC Therapeutics joined CGTL to discuss the status of ASC-618, as well as other agents in the pipeline.

After receiving Investigational New Drug clearance from the FDA in mid 2021, ASC Therapeutics launched a phase 1/2 trial (NCT04676048) to assess the safety, tolerability, and preliminary efficacy of ASC-618 in patients with hemophilia A.

“These regulatory milestones culminate years of hard work, dedication, and collaborative efforts by our functional teams," Oscar Segurado, MD, PhD, chief medical officer at ASC Therapeutics, said in a statement. "Preclinical studies have shown that ASC618 has the potential to reduce therapeutic dosing and increase durability of hemophilia A gene therapy with a novel bioengineered construct that can improve biosynthesis, protein folding, and secretion of factor VIII.”

In a conversation with CGTL, Ruhong Jiang, PhD, cofounder, president, and chief executive officer of ASC Therapeutics, shares updates from the ongoing study of ASC-618, as well as ASC's progress on ASC-930, a stem cell therapy for the treatment of graft-versus-host disease.