Divalent siRNA for prion disease

This repository holds regulatory documents, raw data, source code, and figures for our divalent siRNA drug candidate for prion disease.

IND

We have made public 92 PDF documents comprising our Investigational New Drug (IND) application as cleared by FDA on March 14, 2025, as well as our Pre-IND Type B and Type D interactions with FDA. We hope this will be useful to the rare disease community. As a disclaimer, note that FDA evaluates every drug and every indication individually. What was considered acceptable for our Research IND for this drug candidate in symptomatic prion disease patients may not be considered acceptable in other programs.

Pre-print

A pre-print describing this work has been posted to bioRxiv:

Gentile JE, Corridon TL, Serack FE, Echeverria D, Kennedy ZE, Gallant-Behm CL, Hassler MR, Kinberger G, Kamath NG, Lian Y, Gross KY, Miller R, DeSouza-Lenz K, Howard M, Guzman K, Chan N, Curtis DT, Fettes K, Lemaitre M, Cappon G, Jackson AL, Yamada K, Alterman JF, Coffey AA, Minikel EV, Khvorova A, Vallabh SM. Divalent siRNA for prion disease. bioRxiv. 2024 Dec 5;2024.12.05.627039.

This work is licensed under a Creative Commons Attribution 4.0 International License.