CRISPR Medicine

ASC Therapeutics

Company Type: Therapeutic development

Main focus: Developing curative treatments for various diseases through a proprietary gene-editing platform, TARGATT™, and other gene-editing technologies

Company stage: Clinical

Diseases: β-Thalassemia, haemophilia, and graft-versus-host disease

Genome-editing tool: CRISPR-Cas9 and TARGATT™

Funding stage: Private

Location: Milpitas, California, USA

Website: https://www.asctherapeutics.com/

Pipeline: https://www.asctherapeutics.com/our-pipeline/

Partners: University of Massachusetts, University of Pennsylvania, Stanford University, Emory University, Shantou University, Peking University Shenzhen Hospital


ASC Therapeutics is a biotechnology company focused on developing gene therapies for rare genetic disorders. The company uses advanced CRISPR gene editing and gene replacement technologies to create treatments for diseases such as haemophilia A and B, β-thalassemia, and other monogenic conditions. ASC Therapeutics has so far disclosed three pre-clinical-stage gene-editing programmes.

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