Precision Medicines for Genetic Diseases

Who we are

Systematic Approach to Monogenic Disease
Utilizing a mapping of the 7,000+ genetic disease landscape to identify opportunities for therapeutic intervention
Disease-modifying therapeutics
Targeting well-defined genetic diseases at their source
Experienced leadership team
Leveraging our strong R&D track-record and lean operational experience
Focusing on individual products, not technology platforms, to rapidly bring therapies to patients
Assets housed in multiple discrete subsidiaries

Our Team

Seven programs to date – two in the clinic – and actively seeking new opportunities


Our Investors

Get In Touch

165 University Ave

Suite 5

Palo Alto, CA 94301

TEL: (650) 521-0894