Building Natural History Data for Rare Disorders to Improve Existing Strategies for Gene Therapy Development
24 Jun 2025
Gene Therapy Development
Establishing robust natural history datasets to better understand disease variability, progression, and key biomarkers for gene therapy targeting. – Utilizing real-world patient data to refine trial eligibility criteria, outcome measures, and regulatory submissions. – Strengthening evidence for efficacy and long-term benefits to support approvals, reimbursement, and patient access. (TBC)
Industry Expert