First Patient With Hemophilia A Dosed in New Gene Therapy Trial


ASC618 is designed to minimize cellular stress and may increase durability of gene therapy for hemophilia.

ASC Therapeutics has dosed the first patient with hemophilia A with ASC618 gene therapy in a phase 1/2 clinical trial (NCT04676048).1

“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. 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,” Oscar Segurado, MD, PhD, chief medical officer, ASC Therapeutics, said in a statement.1

The first patient was dosed at the Arkansas Children’s Hospital. The trial is primarily evaluating the safety, tolerability, and preliminary efficacy of ASC618 as measured by the incidence and severity of adverse events. Secondary outcome measures include changes in FIII activity from baseline, annualized FIII consumption and annualized bleeding rate.

ASC618 is an investigational second-generation adeno-associated virus (AAV8) gene therapy that contains a proprietary B-domain deleted codon-optimized bioengineered chimeric Factor VIII (FVIII) gene variant (ET3) and a minimal-length liver-specific promoter. The therapy was granted Fast Track and Orphan Drug designations by the FDA and Orphan Medicinal Product designation by the European Commission.

READ MORE: Hemophilia Gene Therapy Shared Decision Making Tool Needed, Providers Say

“The successful dosing of the first patient is a testament of both our commitment to the hemophilia A community and our team's capability in advancing novel genetic therapies into the clinic,” Ruhong Jiang, PhD, chief executive officer, ASC Therapeutics, added.1

ASC618 has been shown to produce therapeutic levels of FVIII protein at comparatively low doses. It yielded at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with a B domain-deleted human FVIII construct and may increase durability with hemophilia gene therapies. ASC618 was developed at Emory University and ASC Therapeutics obtained exclusive global rights from Expression Therapeutics to develop the therapy.

“Treating our first patient with ASC618 reinforces our focus on providing cutting edge therapeutic modalities to our patients with hemophilia A. 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,” principal investigator Shelley Crary, MD, Pediatric Hematologist/Oncologist, Arkansas Children’s Hospital, added.1

The first gene therapy for treating hemophilia A, valoctocogene roxaparvovec (BioMarin) was approved in June 2023 under the name Roctavian. The approval was based on data from the phase 3 GENEr8-1 clinical trial (NCT03370913).

"Adults with severe hemophilia A face a lifelong burden, with frequent infusions and a high risk of health complications, including uncontrolled bleeding and irreversible joint damage," Steven Pipe, MD, an investigator on GENEr8-1 and the director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital, said in a statement."The approval of Roctavian, as the first gene therapy for severe hemophilia A, has the potential to transform the way we treat adults based on years of bleed control following a single, 1-time infusion."

1. ASC Therapeutics doses first patient with ASC618 second-generation gene therapy for Hemophilia A. News release. ASC Therapeutics. November 29, 2023.
2. U.S. Food and Drug Administration Approves BioMarin's ROCTAVIAN™ (valoctocogene roxaparvovec-rvox), the First and Only Gene Therapy for Adults with Severe Hemophilia A. News release. BioMarin Pharmaceutical Inc. June 29, 2023.,-the-First-and-Only-Gene-Therapy-for-Adults-with-Severe-Hemophilia-A
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