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Modulating gene expression to treat the root cause of genetically defined diseases

We are focused on improving the lives of patients with genetically defined diseases in areas of high unmet medical need.

Therapeutic Focus

We are focused on improving the lives of patients with genetically defined diseases in areas of high unmet medical need.

Learn more about our areas of focus

Pipeline

Our lead program, pociredir, is being investigated in clinical trials to treat sickle cell disease (SCD). Explore other programs and discovery efforts within our pipeline.

Explore our pipeline

Join Our Team

Our core values define our culture. Learn more about opportunities to join us.

See our job openings

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About
Pipeline
Publications & Presentations
Therapeutic Focus
Careers
- Current Openings
Investor Relations
Contact