ASC Therapeutics, a privately-owned biopharmaceutical company specializing in the development of in-vivo gene replacement, gene editing, and allogeneic cell therapies for hematologic and rare disorders, announced a significant milestone on its 1-year birthday. The company has successfully dosed the first patient in the Phase I/IIa clinical trial of their leading candidate, ASC618, at the Arkansas Children’s Hospital.

ASC618 represents a second-generation gene therapy designed for individuals with severe and moderately severe hemophilia A. The Adeno-Associated Virus (AAV) construct of ASC618 includes a proprietary B-domain deleted codon-optimized bioengineered chimeric Factor VIII (FVIII) gene and a minimal-length liver-specific promoter. In pre-clinical studies, ASC618 has demonstrated the ability to produce therapeutic levels of FVIII protein at significantly lower doses compared to other constructs, showing promise for increased effectiveness and cost-efficiency.

ASC Therapeutics, expressed gratitude for the successful dosing of the first patient, highlighting the potential impact of ASC618 as a more durable and affordable second-generation gene therapy for hemophilia A patients. CEO Dr. Ruhong Jiang emphasized the achievement as a testament to the company's dedication to the hemophilia A community and their team's proficiency in advancing genetic therapies.

Dr. Shelley Crary, Principal Investigator of the ASC618 phase I/IIa clinical trial and Pediatric Hematologist/Oncologist at the Arkansas Children’s Hospital, underscored the significance of assessing this novel one-and-done gene therapy in a clinical setting. The aim is to potentially replace the lifelong, burdensome, and expensive treatments currently used to manage hemophilia A.

Hemophilia A, affecting approximately 1 in every 5000 live-born males, poses challenges with existing treatment regimens, such as the short half-life of therapeutics and the need for frequent and lifelong interventions. ASC618, with its unique attributes, presents a promising alternative by focusing on gene replacement therapies.

ASC618, an AAV8-based gene therapy product, incorporates a liver-specific promoter and a bioengineered, codon-optimized B domain-deleted FVIII variant (ET3). The therapy has demonstrated a significant increase in the biosynthesis and secretion of FVIII compared to other constructs in preclinical studies. The potential to enhance durability and reduce cellular stress makes ASC618 a compelling candidate.

The phase I/IIa clinical trial for ASC618 aims to evaluate safety, tolerability, and preliminary efficacy. The program has received FDA Fast Track and Orphan Drug Designations, along with Orphan Medicinal Product Designation by the European Commission.

 

Source: businesswire.com

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