Blueprint Neurotherapeutics Network (BPN): Biologic-based Drug Discovery and Development for Disorders of the Nervous System (UG3/UH3 Clinical Trial Optional)

The NIH Blueprint Neurotherapeutics Network (BPN) for Biologics offers funding for projects focused on biologics-based drug discovery and development targeting nervous and neuromuscular disorders. This opportunity (PAR-24-293) supports a wide range of biotherapeutic modalities, including antibodies, gene therapies, cell therapies, peptides, proteins, and oligonucleotides, with a primary goal of advancing these therapies from lead optimization to early clinical trials. Through a UG3/UH3 cooperative agreement, this phased funding model includes both discovery and development stages and mandates regular collaboration with NIH program staff and NIH-contracted consultants. Selected projects can also utilize BPN-contracted CROs (contract research organizations) specializing in industry-standard manufacturing and regulatory activities to aid in nonclinical and clinical phases. This program is open to projects that align with the missions of participating NIH Institutes and Centers, including NINDS, NEI, NIA, NIAAA, NIDCR, NIDA, NIEHS, NIMH, and NCCIH. Eligible projects must be focused on a specific nervous or neuromuscular disorder and meet either discovery or development stage criteria. Discovery-stage projects should have identified one or more lead agents and shown preliminary efficacy in disease-relevant models, while development-stage projects should have a single clinical candidate ready for IND-enabling studies. All projects are expected to have evidence supporting the biological mechanism’s relevance to the target disease and documented preliminary pharmacokinetics and pharmacodynamics for development-stage candidates. During the UG3 phase, funded activities include lead optimization, safety profiling, and preliminary bioanalytical assay development. Projects entering the development stage should use this phase to complete remaining IND-enabling studies in preparation for the UH3 phase, which funds GLP-compliant toxicology studies, cGMP manufacturing, and a first-in-human clinical trial if feasible. The transition from UG3 to UH3 is contingent upon NIH review of milestone progress, regulatory approvals, and program priorities. This funding opportunity mandates that applications include specific documents, such as a Gantt chart outlining project timelines and an Intellectual Property (IP) strategy. The IP plan should address potential patent issues, list any pending or granted patents related to the project, and outline plans for future IP filings. Applications must also include a Target Product Profile (TPP), summarizing minimal and ideal attributes of the proposed therapeutic, including intended patient population, delivery mode, and clinical efficacy targets. Additionally, all applicants are encouraged to consult with NIH scientific staff prior to submission to ensure alignment with program goals and proper use of available BPN resources. Eligibility extends to U.S. and non-U.S. higher education institutions, nonprofit organizations, for-profit entities, and government agencies. Applicants must complete all required NIH registrations, including SAM and eRA Commons, and ensure that proposals meet NIH guidelines on rigor and reproducibility. Key dates include an earliest submission date of December 27, 2024, with multiple application deadlines available semi-annually through July 2027. Projects will be reviewed based on scientific merit, significance, innovation, rigor, and feasibility. Evaluation will include assessments of the project’s biological rationale, potential clinical impact, feasibility of conducting planned research within the given timeframes, and the potential to advance a safe and effective therapeutic candidate. Applications with budgets exceeding $500,000 per year must contact NIH staff at least eight weeks prior to submission. Recipients will have substantial reporting responsibilities, including annual progress reports and compliance with data management and sharing policies.