Research & Development
Our Development Pipeline
Advancing investigational gene therapies for diseases with significant unmet medical needs. Explore our programs and their current position along the development pathway.
Programs in development
A structured overview of publicly shared research and development programs.
2 programs shown
Program / indication
SpinEx
Spinal muscular atrophy (SMA)
- Therapeutic area
- Neuromuscular
- Approach
- AAV9-mediated SMN1 gene delivery
Current Development Status:
ResearchNonclinicalPhase 1Phase 2Phase 3Post-market
NeofovEx
Age-related macular degeneration (AMD)
- Therapeutic area
- Ophthalmology
- Approach
- Gene-based Anti-VEGF expression
Current Development Status:
ResearchNonclinicalPhase 1Phase 2Phase 3Post-market
AngioEx
Solid Cancers
- Therapeutic area
- Oncology
- Approach
- Gene-based Anti-VEGF expression
Current Development Status:
ResearchNonclinicalPhase 1Phase 2Phase 3Post-market
No programs match these filters.