In The Media STAT News
FDA Unveils Rules for Bespoke Gene Therapies, Predicting Flood of Rare Disease Applications
Interview
Search Results
Venture Philanthropy Transformed Cystic Fibrosis. Can It Deliver for Other Diseases?
CF Foundation’s Landmark Drug Approvals With Vertex Inspired Patient Groups to Act Like Biotech Investors. But Only a Few Groups Have Made Big Bets, LDI Fellow Finds
In The Media STAT News
FDA’s New ‘Plausible Mechanism Pathway’ for Personalized Gene Editing Raises Concerns
Extended Interview
Impact: LDI Fellows Shaped Public Discussion and Public Policy in 2025
Highlighting 10 Ways LDI Fellows Put Their Research Into Action
In The Media STAT News
FDA Review Staff was Excluded from Voting on Whether to Approve First Priority Voucher Drug
Interview
These Patients Have Nothing Else. Should That Be Enough for FDA Drug Approval?
Despite Streamlined Processes, FDA’s Role in Rare-Disease Drug Development Draws Continued Scrutiny
In The Media KFF Health News
Under Trump, FDA Seeks To Abandon Expert Reviews of New Drugs
Interview
Policy Recommendations for Strengthening the Accelerated Approval Program for Oncology Drugs
Memo: Delivered to the U.S. Food and Drug Administration (FDA)