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NIH funding notice PAR-22-030

Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders (U01 - Clinical Trial Optional)

Closed on October 11, 2024.

Posted
November 9, 2021
Close date
October 11, 2024
Activity codes
U01
Institutes taking part
NINDS

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 PAR-25-327.

Who won it before

5 competing awards (new and renewals) from FY2014 to FY2025 under this notice and the notices it replaced: $8,508,908 in first-year total cost, median $800,485.

Most frequent study sections

  1. Special Emphasis Panel[ZNS1 SRB-M (12)]3
  2. Special Emphasis Panel[ZNS1 SRB-M (15)]1
  3. Special Emphasis Panel[ZNS1 SRB-M (18)]1

Most frequent institutions

  1. BROAD INSTITUTE, INC.1
  2. COLUMBIA UNIVERSITY HEALTH SCIENCES1
  3. RESEARCH INST NATIONWIDE CHILDREN'S HOSP1
  4. UNIVERSITY OF PENNSYLVANIA1
  5. UT SOUTHWESTERN MEDICAL CENTER1
Competing awards under PAR-22-030 and the notices it replaced
FYProjectPIsInstitutionFirst-year costStudy section
2024Silence ALS: A Platform for the Discovery and Development of Antisense Therapeutics for Patients with Ultra-Rare Forms of ALS1U01NS134684-01SHNEIDER, NEIL ALANCOLUMBIA UNIVERSITY HEALTH SCIENCES, NY$5,159,965ZNS1
2024IND-enabling studies of non-immunogenic gene therapy for ultra-rare DMD patients excluded from dystrophin trials1U01NS134672-01A1STEDMAN, HANSELL HUNIVERSITY OF PENNSYLVANIA, PA$1,541,019ZNS1
2023Advancement of Prion Protein-Lowering Divalent siRNA Therapy for Prion Disease1U01NS132994-01MINIKEL, ERIC VALLABHBROAD INSTITUTE, INC., MA$800,485ZNS1
2023IND-enabling studies for Aspartylglucosaminuria (AGU) to support the initiation of an AAV9/AGA gene transfer clinical trial1U01NS129339-01A1GRAY, STEVEN JIANNACCONE, SUSAN TUT SOUTHWESTERN MEDICAL CENTER, TX$631,697ZNS1
2023Viral Gene Therapy for Menkes Disease1U01NS129343-01A1KALER, STEPHEN GERARDRESEARCH INST NATIONWIDE CHILDREN'S HOSP, OH$375,742ZNS1

Data: Grants.gov's daily extract and NIH's notice pages and award files, in ScienceGraph release conductgrants-2026-11-09-0.1.0. 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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