Our Translational Pipeline
Our development roadmap is built around inherited disorders and high-need rare disease programs where precise genomic intervention can make a meaningful difference.
Priority Therapeutic Areas
- Sickle Cell Disease
- Cystic Fibrosis
- Duchenne Muscular Dystrophy
- Inherited Retinal Disorders
- Transthyretin Amyloidosis
- Select oncology-directed cell engineering programs
Program Stages
Discovery: Target selection, sequence analysis, and mechanistic validation.
Optimization: Editing architecture refinement, delivery screening, and specificity analysis.
Translational Readiness: Preclinical efficacy, toxicology strategy, manufacturing readiness, and trial design support.
Clinical Activation: Site preparation, patient identification, and trial execution in collaboration with clinical partners.