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NIH funding notice RFA-TR-23-016

Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)

Closed on March 25, 2024.

Posted
July 5, 2023
Close date
March 25, 2024
Activity codes
R21
Institutes taking part
NCATS, NIMH

Read the notice on Grants.gov · NIH Guide page

The notices it replaced

Its page names no earlier notice: it is new, or it does not say.

Replaced by RFA-TR-24-023.

Who won it before

4 competing awards (new and renewals) from FY2014 to FY2025 under this notice and the notices it replaced: $1,195,528 in first-year total cost, median $311,189.

Most frequent study sections

  1. Special Emphasis Panel[ZTR1 RD-1 (01)]4

Most frequent institutions

  1. DUKE UNIVERSITY1
  2. MYOGENE BIO, LLC1
  3. UNIVERSITY OF CALIFORNIA, SAN FRANCISCO1
  4. UNIVERSITY OF TEXAS AT AUSTIN1
Competing awards under RFA-TR-23-016 and the notices it replaced
FYProjectPIsInstitutionFirst-year costStudy section
2024Proof-of-concept for the use of enzyme replacement therapy as a lifesaving treatment for sphingosine phosphate lyase insufficiency syndrome1R21TR005157-01SABA, JULIE DUNIVERSITY OF CALIFORNIA, SAN FRANCISCO, CA$463,150ZTR1
2024Effective treatment of cystinuria with a novel engineered cyst(e)ine-degrading human enzyme therapy1R21TR005156-01GEORGIOU, GEORGE GEORGIOUUNIVERSITY OF TEXAS AT AUSTIN, TX$437,761ZTR1
2024Triacetin Treatment for Propionic Acidemia by Rebalancing the Acetyl-CoA/Propionyl-CoA Metabolism1R21TR005163-01CHEN, XIAOXIN LUKEZHANG, GUOFANGDUKE UNIVERSITY, NC$184,617ZTR1
2024Durability of a gene editing therapy that restores dystrophin in a humanized mouse model of Duchenne muscular dystrophy1R21TR005162-01YOUNG, COURTNEY SMYOGENE BIO, LLC, CA$110,000ZTR1

Data: Grants.gov's daily extract and NIH's notice pages and award files, in ScienceGraph release conductgrants-2026-11-10-0.1.1. An award is listed only where NIH's own record names the notice, and a PI only by NIH's profile ID. Grants.gov and NIH do not endorse ScienceDex.

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RFA-TR-23-016: Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed) | ScienceDex | ConductScience