Drug intelligence / Profile preview

ASC518

Development stage
Preclinical
Lead developer
ASC Therapeutics
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration
Intravenous
01

Overview

ASC518 is an **in vivo gene editing therapy** being developed by **ASC Therapeutics** for **hemophilia A**. It is a preclinical investigational program designed to address the underlying genetic defect responsible for factor VIII deficiency, distinguishing it from conventional factor replacement approaches and from ASC Therapeutics' separate hemophilia A gene replacement candidate. Based on the available information, ASC518 uses a gene transfer and gene editing strategy intended to correct or functionally repair the defective **F8** gene in patients with congenital hemophilia A.

02

Targets

F8 (Coagulation Factor VIIIa)

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