Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders
Funds IND-enabling studies and planning that take a gene-based therapy for an ultra-rare neurological or neuromuscular disorder toward its first human trial.
An ultra-rare neurological or neuromuscular disease rarely has the patient numbers to attract a company's own development budget on its own. This network exists to carry a promising candidate to human testing anyway.
What is funded is Investigational New Drug-enabling studies and planning activities for First-in-Human clinical testing of gene-based or transcript-directed therapeutics — oligonucle…
Investigational New Drug-enabling studies and First-in-Human planning for gene-based or transcript-directed therapeutics — including oligonucleotides and viral-based gene therapies — for ultra-rare neurological or neuromuscular disorders, requiring a robust biological rationale and demonstrated proof-of-concept data in a relevant model system. Clinical trials are optional.
Sign up free to see the funding breakdown
Sign up free to see the industries in scope
Sign up free to see the full eligibility
Sign up free to see how to apply
Sign up free to see what you submit
Sign up free to see how they score you
Sign up free to see the timeline
Sign up free to see where teams trip up
Guides for this application
All guides →NIH Biosketch Guide: Format, SciENcv Steps, and Examples
Learn what an NIH biosketch is, how the format works, what to prepare for SciENcv, and how startup teams should avoid common biosketch mistakes.
Write proposalHow to Write a Specific Aims Page
Learn how to write a specific aims page for NIH, SBIR-adjacent, and translational R&D grants, with structure, reviewer logic, examples, and common mistakes.
Where to applyGrants for Biotech Startups: NIH, NSF, SBIR/STTR, and Non-Dilutive Funding
A practical guide to grants for biotech startups, including NIH SBIR/STTR, NSF, translational funding, evidence, regulatory readiness, and budgets.
Write proposalHow to Write a Grant Proposal Reviewers Can Score
Learn how to write a grant proposal around reviewer criteria, evidence, work packages, budget logic, and commercialization for deeptech and R&D funding.
More from NIH
All grants from this funder →Nutrition Obesity Research Centers (NORCs)
🇺🇸NIH · United States
Funds shared research cores, pilot funding and a catalyst programme where a substantial nutrition or obesity research effort already exists.
Optimizing Behavioral Sleep Interventions for Adolescents and Young Adults
🇺🇸NIH · United States
Funds a pilot trial of a behavioural sleep intervention for adolescents and young adults who have a mental health disorder or are at risk of one.
GREGoRi Data Coordination and Outreach Center
🇺🇸NIH · United States
Funds the one centre that coordinates a rare-disease genomics programme and gets its protocols, data and tools out to everybody else.
GREGoRi Innovation Projects
NIH · United States
Funds a genuinely new experimental or computational approach to finding the cause of a rare genetic disorder, where the current state of the art falls short.
GREGoRi Technology Integration Center
🇺🇸NIH · United States
Funds the centre that turns new molecular methods for rare-disease diagnosis into standards, best practices and a benchmarking dataset.
Clinical Observational (CO) Studies in Musculoskeletal, Rheumatic, and Skin Diseases
🇺🇸NIH · United States
Funds studies that watch patients rather than treat them, in bone, joint, rheumatic and skin disease. No intervention allowed.
Similar grants
Browse the directory →
Emerging Infections Network - Research for Preventing, Detecting, and Managing Travel-associated Infectious Diseases
🇺🇸CDC · United States
Funds surveillance and research networks that detect, diagnose and track infectious diseases among international travelers.
DOW Combat Readiness - Medical Research Program Translational Research Award
🇺🇸MRDC / CDMRP · United States
Funds translational trauma-care research moving a mature idea toward better survival and care right after injury.
Brain Behavior Quantification and Synchronization — Next Gen Sensor Technology Development
🇺🇸BRAIN Initiative · United States
Supports neural behavior-sensing technology development through translational cooperative projects.
DOD Defense Health Agency (DHA) Research & Development FY23-FY27 BROAD AGENCY ANNOUNCEMENT for Extramural Medical Research
🇺🇸MRDC / CDMRP · United States
Funds an unsolicited medical research idea across USAMRDC's whole portfolio, but only after a pre-proposal through eBRAP earns an invitation to submit in full.
ASPIRE Reduction-to-Practice (Seed)
🇺🇸NCATS · United States
Supports translational chemistry and automation platform development through university-linked partnerships.
Animal and Veterinary Innovation Centers
🇺🇸FDA · United States
Funds academic institutions to become long-term FDA partners researching bird flu, animal genomics and unmet veterinary needs.