Translational Efforts to Advance Gene-based Therapies for Ultra-Rare Neurological and Neuromuscular Disorders

Published by NIH

Research Project Cooperative Agreements

The Ultra-Rare Gene-Based Therapy (URGenT) network supports Investigational New Drug (IND)-enabling studies and planning activities for First-in-Human (FIH) clinical testing of gene-based or transcript-directed therapeutics, such as oligonucleotides and viral-based gene therapies, for ultra-rare neurological or neuromuscular disorders. The goal of this funding opportunity announcement (FOA) is to accelerate the development of a promising clinical candidate with robust biological rationale and demonstrated proof of concept (POC) data for the intended approach in a model system relevant to a specified patient population towards an IND filing and the initiation of a clinical trial.

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Limited Competition: Small Grant Program for the NCATS Clinical and Translational Science Award

Published by NIH

Small Grant Program

The purpose of this NOFO is to enhance the capability of NCATS CTSA Program KL2/K12 scholars and recipients of diversity and re-entry supplements (Research Supplements to Promote Diversity in Health-Related Research (Admin Supp – Clinical Trial Not Allowed) (PA-23-189 and PA-21-071)) and Research Supplements to Promote Re-Entry, Re-integration into, and Re-training in Health-Related Research Careers (Admin Supp – Clinical Trial Not Allowed) (NOT-OD-23-170 and NOT-OD-21-134)) supported by the CTSA Program to conduct translational science research as they complete the transition to fully independent academic translational scientists.

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Cellular and Molecular Biology of Complex Brain Disorders

Published by NIH

Exploratory/Developmental Research Grant

This Notice of Funding Opportunity (NOFO) encourages research on the biology of high confidence risk factors associated with complex brain disorders, with a focus on the intracellular, transcellular and circuit substrates of neural function. For the purposes of this NOFO , the term “complex” can refer to a multifactorial contribution to risk (e.g., polygenic and/or environmental) and/or highly distributed functional features of the brain disorders

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NINDS Exploratory Clinical Trials for Small Business (R43/R44 Clinical Trial Required)

Published by NIH

Phase IISmall Business Innovation Research (SBIR) Grant - Phase Iand Fast-Track

The purpose of this notice of funding opportunity (NOFO) is to provide a vehicle for Small Business Concerns (SBCs) submitting Small Business Innovation Research (SBIR) grant applications for investigator-initiated exploratory clinical trials to the National Institute of Neurological Disorders and Stroke (NINDS). The projects must focus on products related to the mission and goals of the NINDS and may evaluate drugs, biologics, devices, or diagnostics, as well as surgical, behavioral or rehabilitation therapies.

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Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01) Clinical Trials Required

Published by NIH

Clinical TrialRare diseasesUnmet Needs

The purpose of this funding opportunity announcement (FOA) is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions. Additionally, through the funding of collaborative, efficient, and/or innovative clinical trials, FDA expects to increase the number of approved treatments for rare diseases and exert a broad and positive impact on rare disease drug development.

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NeuroNEXT Small Business Innovation in Clinical Trials (U44 Clinical Trial Optional)

Published by NIH

Rare diseasesInvestigational Medicinal ProductClinical Trial

This Funding Opportunity Announcement (FOA) encourages small business applications for exploratory clinical trials of investigational agents (drugs, biologics, surgical therapies or devices) that may contribute to the justification for and provide the data required for designing clinical studies. Diseases chosen for study should be based on the NINDS strategic plan and clinical research interests (www.ninds.nih.gov/funding/areas/index.htm).

Successful applicants will be given access to the NeuroNEXT infrastructure. Following peer review, NINDS will prioritize and order trials that are given access to the NeuroNEXT infrastructure. The NeuroNEXT Clinical Coordinating Center (CCC) will work with the successful applicant to efficiently implement the proposed study. The NeuroNEXT Data Coordinating Center (DCC) will provide statistical and data management support. The NeuroNEXT clinical sites will provide recruitment/retention support as well as on-site implementation of the clinical protocol.

Applicants do not need to be part of the existing NeuroNEXT infrastructure.

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NINDS Exploratory Clinical Trials for Small Business (R41/R42 Clinical Trial Required)

Published by NIH

Rare diseasesSmall Business Innovation ResearchClinical Trials

The purpose of this funding opportunity announcement (FOA) is to provide a vehicle for Small Business Concerns (SBCs) submitting Small Business Innovation Research (SBIR) grant applications for investigator-initiated exploratory clinical trials to the National Institute of Neurological Disorders and Stroke (NINDS). The projects must focus on products related to the mission and goals of the NINDS and may evaluate drugs, biologics, devices, or diagnostics, as well as surgical, behavioral or rehabilitation therapies.

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Pilot Projects Investigating Understudied Proteins Associated with Rare Diseases (R03 Clinical Trial Not Allowed)

Published by NIH

Rare diseasesUnderstudied Proteins

The purpose of this funding opportunity announcement (FOA) is to solicit applications for pilot projects to elucidate a role for understudied proteins associated with rare diseases. Awards will support generation of preliminary data and/or tools around eligible understudied protein(s). A list of eligible proteins is provided and are members of druggable protein families that have a known association with a rare disease. This FOA is intended to jumpstart research on understudied proteins that are associated with rare diseases and provide applicants with sufficient funding to perform basic biochemical and/or biological work to further the characterization of understudied proteins associated with rare disease.

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