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ASC618
- Development stage
- Phase 2
- Lead developer
- ASC Therapeutics
- Modality
- Recombinant Proteins and Enzymes, Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
ASC618 is an investigational, second-generation gene therapy for the treatment of hemophilia A. It utilizes an adeno-associated virus serotype 8 (AAV8) vector to deliver a bioengineered, codon-optimized B domain-deleted variant of the factor VIII (FVIII) gene under control of a novel liver-specific promoter. This design enables significantly increased biosynthesis and secretion of FVIII—over 10-fold higher than previous constructs—at lower therapeutic doses. The therapy aims to provide durable expression of FVIII by minimizing cellular stress and reducing induction of the unfolded protein response in hepatocytes, potentially offering a one-time treatment alternative to lifelong prophylactic or on-demand FVIII replacement therapies. ASC618 is being developed by ASC Therapeutics, which has obtained exclusive global rights from Expression Therapeutics[2][5][6][7][8].
- Other names
- ASC-618ASC618ASC 618
02
Targets
Coagulation Factor IXaF10 (Factor Xa)BB-031 (Von Willebrand factor)F8 (Coagulation Factor VIIIa)
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