The notices it replaced
- Reissues: RFA-TR-24-023 Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
- 2 steps back: RFA-TR-23-016 Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)
Who won it before
10 competing awards (new and renewals) from FY2014 to FY2025 under this notice and the notices it replaced: $3,798,962 in first-year total cost, median $423,384.
Most frequent study sections
- Special Emphasis Panel[ZTR1 RD-1 (01)]10
Most frequent institutions
- CHILDREN'S HOSP OF PHILADELPHIA1
- CLEMSON UNIVERSITY1
- DUKE UNIVERSITY1
- MYOGENE BIO, LLC1
- NORTHWESTERN UNIVERSITY1
| FY | Project | PIs | Institution | First-year cost | Study section |
|---|---|---|---|---|---|
| 2025 | RepSox and its analog for prophylactic intervention in RUNX1 haploinsufficiency FPDMM1R21TR005192-01A1 · under RFA-TR-24-023 | PONCZ, MORTIMER | CHILDREN'S HOSP OF PHILADELPHIA, PA | $489,500 | ZTR1 |
| 2025 | Development of anti-LTBP4 as a biologic to treat Limb Girdle Muscular Dystrophy 2C1R21TR005472-01 · under RFA-TR-24-023 | DEMONBREUN, ALEXIS R.MCNALLY, ELIZABETH M | NORTHWESTERN UNIVERSITY, IL | $440,000 | ZTR1 |
| 2025 | Src Homology and Collagen (Shc) inhibition for the treatment of transplant-related pulmonary fibrosis.1R21TR005507-01 · under RFA-TR-24-023 | HSU, JOE L | STANFORD UNIVERSITY, CA | $427,392 | ZTR1 |
| 2025 | Optimization and Preclinical Validation of QTE Therapy for Friedreich's Ataxia1R21TR005475-01 · under RFA-TR-24-023 | CHANDRAN, VIJAYENDRAN | UNIVERSITY OF FLORIDA, FL | $419,375 | ZTR1 |
| 2025 | Optimization of delivery of HEX for treatment of primary amebic encephalitis1R21TR005502-01 · under RFA-TR-24-023 | CHOUINARD, CHRISTOPHER DONALDMORRIS, JAMES CULVIN | CLEMSON UNIVERSITY, SC | $419,375 | ZTR1 |
| 2025 | Prevention of heterotopic ossification with localized delivery of arsenic trioxide loaded nanoparticles as a novel treatment for fibrodysplasia ossificans progressiva1R21TR005159-01A1 · under RFA-TR-24-023 | JEREMIC, JELENA GVOZDENOVIC | NOSTOPHARMA, LLC, MD | $407,792 | ZTR1 |
| 2024 | Proof-of-concept for the use of enzyme replacement therapy as a lifesaving treatment for sphingosine phosphate lyase insufficiency syndrome1R21TR005157-01 · under RFA-TR-23-016 | SABA, JULIE D | UNIVERSITY OF CALIFORNIA, SAN FRANCISCO, CA | $463,150 | ZTR1 |
| 2024 | Effective treatment of cystinuria with a novel engineered cyst(e)ine-degrading human enzyme therapy1R21TR005156-01 · under RFA-TR-23-016 | GEORGIOU, GEORGE GEORGIOU | UNIVERSITY OF TEXAS AT AUSTIN, TX | $437,761 | ZTR1 |
| 2024 | Triacetin Treatment for Propionic Acidemia by Rebalancing the Acetyl-CoA/Propionyl-CoA Metabolism1R21TR005163-01 · under RFA-TR-23-016 | CHEN, XIAOXIN LUKEZHANG, GUOFANG | DUKE UNIVERSITY, NC | $184,617 | ZTR1 |
| 2024 | Durability of a gene editing therapy that restores dystrophin in a humanized mouse model of Duchenne muscular dystrophy1R21TR005162-01 · under RFA-TR-23-016 | YOUNG, COURTNEY S | MYOGENE BIO, LLC, CA | $110,000 | ZTR1 |
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.