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ASC Therapeutics Begins Phase I/IIa Trial of Hemophilia A Gene Therapy

NEW YORK – ASC Therapeutics on Wednesday said that the first child with hemophilia A has received its investigational gene therapy ASC618 within a Phase I/IIa trial.

In the Phase I/IIa clinical trial, Milpitas, California-based ASC Therapeutics is evaluating ASC618's safety, tolerability, and preliminary efficacy. "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," ASC Therapeutics CEO Ruhong Jiang said in a statement.

Hemophilia A is a hereditary hemorrhagic disorder due to a deficit of factor VIII, which causes excessive bleeding. ASC618 uses an adeno-associated virus to deliver a bioengineered, B-domain deleted, codon-optimized chimeric factor VII gene and a minimal-length liver-specific promoter to patients.

ASC618 was developed originally at Emory University based on researchers' work on a next-generation hemophilia A gene therapy. ASC Therapeutics obtained exclusive global rights to develop ASC618 from Expression Therapeutics in 2019 and has since conducted investigational new drug-enabling studies showing that this treatment can bolster factor VIII secretion at lower therapeutic doses compared to other B domain-deleted human factor VIII constructs. In preclinical studies, biosynthesis and secretion of factor VIII increased by tenfold with ASC618.

The US Food and Drug Administration has granted ASC618 fast-track and orphan drug designations, and the European Commission has also deemed the gene therapy an orphan medicinal product.