← Back to contracts

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

US · MD · Bethesda National Institutes of Health (NIH) grant open #PAR-25-327
Response due Oct 08, 2027 · 00:00 UTC

Summary

The National Institutes of Health (NIH) is soliciting applications under Program Announcement PAR-25-327 to support translational research efforts aimed at advancing gene-based therapies for ultra-rare neurological and neuromuscular disorders. The funding mechanism is a U01 cooperative agreement, with clinical trials listed as optional. The opportunity is broadly open to researchers working on gene therapy development pipelines targeting rare neurological and neuromuscular conditions. The solicitation opened in December 2024 and accepts applications through October 2027.

What they want

Applicants are expected to conduct translational research to advance gene-based therapeutic strategies for ultra-rare neurological and neuromuscular disorders. This is a U01 cooperative agreement mechanism, meaning NIH program staff will have substantial involvement in the conduct of the funded activities. Clinical trials are optional under this announcement. Specific research activities would be detailed in individual Funding Opportunity Announcement (FOA) documents linked to PAR-25-327.
Technical requirements
  • Research must focus on gene-based therapeutic approaches
  • Disorders targeted must qualify as ultra-rare neurological or neuromuscular conditions
  • Clinical trial component is optional but permissible under this announcement
  • Cooperative agreement (U01) mechanism — substantial NIH programmatic involvement required

How they evaluate

Submission: Grants.gov federal grants portal (electronic submission)

Risks & flags

  • No dollar value or award ceiling disclosed in the synopsis — budget scope is indeterminate
  • Multi-year open window (Dec 2024 – Oct 2027) with rolling submissions may favor established NIH-funded labs with existing infrastructure
  • Ultra-specific disease focus (ultra-rare neurological and neuromuscular disorders + gene-based therapies) significantly narrows the eligible applicant pool

Market context

inferred from NAICS
Professional, Scientific & Technical Services
NAICS 541711
US market size
$2.0T
Typical award
$25K – $50M
Typical buyers
All federal civilianDoDStates
Commonly required
8(a)WOSBSDVOSBPE/PMP

Sector-level estimate — full code lookup not yet in catalog.

Translational Efforts to Advance Gene-base…
Due Oct 08
Onboard ↗