Advancing Site-Specific Genome Integration Delivering Curative Hemophilia A Therapies Through IND-Enabling Translational Development
- Demonstrating durable factor VIII expression enabling potentially curative one-time hemophilia a treatment strategies
- Establishing precise genome editing minimizing off-target activity supporting confident regulatory advancement toward clinical translation
- Leveraging proprietary metagenomics platforms expanding scalable therapeutic applications across secreted protein deficiencies beyond hemophilia