Research IND
Divalent siRNA therapeutic for prion disease
Broad Institute of MIT and Harvard · Eric Minikel, sponsor representative · Symptomatic prion disease · Divalent siRNA (small interfering RNA)
1 submission · 92 documents · Cleared 14 Mar 2025 · with Pre-IND correspondence
About this submission
Research IND for a divalent siRNA targeting prion disease in symptomatic patients. Cleared by FDA in March 2025. The 92-document submission includes the full IND plus Pre-IND Type B and Type D meeting correspondence — a rare public look at the dialogue between sponsor and FDA before clearance.
Related reading
Reading Between the Lines: What the FDA Says vs. What It MeansA field guide to FDA correspondence, drawn from complete INDs in the archive.Browse the complete eCTD package — 92 documents across Modules 1–5 — cover letter, administrative forms, summaries, CMC, nonclinical, and clinical study reports. Free account required.
Create free account →Contributed by Eric Minikel · source
Companion paper: Divalent siRNA for prion disease · Nucleic Acids Research · doi:10.1093/nar/gkag287
Released under CC-BY-4.0