- Designed preclinical and nonclinical gene therapy studies for Alzheimer’s disease targeting APP, PSEN1 and PSEN2 genes
- Optimized FIH trial designs for Frontotemporal Dementia (FTD) gene therapies targeting MAPT, GRN and C9orf72 genes including findings from preclinical, non-clinical, and natural history studies
- Constructed regulatory dossiers to assure alignment on pathological foundations of disease progression in dementias including natural history controls and registrational endpoint design.
- Restructured clinical program to include progranulin mutations including GRN-related FTD and C9orf72 mutations
Early Stage Indication MoA Foundation
Design preclinical and nonclinical gene therapy studies for Alzheimer’s disease targeting APP, PSEN1 and PSEN2 genes
Optimize Gene Therapy Target Population
Optimize trial designs for frontotemporal dementia gene therapies targeting MAPT, GRN and C9orf72 genes including findings from preclinical, nonclinical, and natural history studies
Build and Deliver on Regulatory Strategy
Construct regulatory dossiers to assure alignment on pathological foundations of disease progression in dementias including natural history controls and registrational endpoint design.
Expertise in AAV Gene Therapy
While the Gaucher program utilized a lentiviral vector, my broad expertise extends to AAV (adeno-associated virus) gene therapy, which could also be instrumental for Gaucher disease or other genetic disorders. My experience allows me to:
- Assess Feasibility: Evaluate whether AAV or other vectors are the most appropriate approach for different patient populations or disease types.
- Develop Strategies: Design effective and efficient clinical and regulatory strategies for AAV-based programs, considering specific manufacturing, dosing, and safety considerations.
- Address Key Considerations: Anticipate and address the critical regulatory, clinical, and logistical issues unique to AAV gene therapy, such as potential immunogenicity and dose optimization.