Project Grant R43TR006346
- The National Center for Advancing Translational Sciences awarded Goeppert LLC, a Philadelphia-based small disadvantaged business, $350,000 on July 1, 2026, under CFDA 93.350 (National Center for Advancing Translational Sciences) to develop NANO-PAVE, a nanopore-based platform for assessing adeno-associated virus (AAV) capsid quality in gene therapy manufacturing. NANO-PAVE addresses a critical bottleneck in AAV clinical translation: current quality-control assays (ELISA, ddPCR, analytical...
- The National Institute of General Medical Sciences awarded the University of Massachusetts Medical School $1,675,000 on May 1, 2026, under the Biomedical Research and Research Training program (CFDA 93.859) to interrogate the roles of truncated and mutated inverted terminal repeat (ITR) sequences in adeno-associated virus (AAV) vectors and their effects on transgene stability through episome formation and host cell genome integration. The project, designated R01GM155648, addresses a critical gap...
- The National Institute of General Medical Sciences awarded Avagene Therapeutics Inc. $220,958 on September 15, 2026, under the Biomedical Research and Research Training program (CFDA 93.859) to advance an automated manufacturing platform for adeno-associated virus (AAV) gene therapy production. Avagene will develop and demonstrate technical feasibility of Bodhi, a compact, closed incubator platform that integrates AI-powered brightfield and fluorescence microscopy, programmable...
- The National Heart, Lung, and Blood Institute awarded Emmune Inc. $299,664 on September 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop a non-viral gene therapy vector for treating and preventing atherosclerotic cardiovascular disease. Emmune will leverage lipid nanoparticle (LNP) technology to achieve non-immunogenic gene delivery of a therapeutic protein capable of treating and preventing atherosclerosis. The company has discovered a novel approach to...
- The National Institute of Neurological Disorders and Stroke awarded The Broad Institute, Inc. $404,282 on May 1, 2026, under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) to engineer transferrin receptor 1 (TFR1) binding adeno-associated virus (AAV) capsids that evade pre-existing neutralizing antibodies while maintaining central nervous system delivery capability. The funded research addresses a critical barrier to AAV gene therapy for...
- The National Center for Advancing Translational Sciences at the National Institutes of Health awarded $931,463 to Acousticabio, Inc. on August 1, 2026, under SBIR Phase II funding (CFDA 93.350) to develop microgel-based formulations enabling subcutaneous delivery of monoclonal antibodies at ultra-high concentrations. Monoclonal antibodies currently require intravenous administration because their structural complexity and poor stability make high-concentration formulation difficult. IV...
- The National Institute on Aging awarded Massachusetts Institute of Technology $466,340 on February 1, 2026, under the Aging Research program (CFDA 93.866, assistance type: Project Grant) to pilot a new non-viral vector for therapeutic nucleic acid delivery to the central nervous system. MIT will develop nucleic acid nanoparticle formulations designed to distribute to specific CNS regions and cell types following intrathecal administration. The project addresses the delivery challenge facing...
- The National Institutes of Health National Center for Advancing Translational Sciences awarded Javelin Biotech Inc. $699,354 on July 1, 2026, under the National Center for Advancing Translational Sciences program (CFDA 93.350). Javelin will use the Javelin Liver Tissue Chip Plus platform, which integrates human liver and kidney microphysiological systems, to investigate the distribution, metabolism, excretion, and toxicity profiles of four FDA-approved antisense oligonucleotide drugs. The work...
- The National Center for Advancing Translational Sciences awarded Aigene Inc. $349,890 on July 1, 2026, under the NCATS program (CFDA 93.350) to develop a novel home and laboratory system for improved cell-free DNA screening and monitoring using transrenal DNA. The project addresses limitations in blood cell-free DNA analysis for diagnostic and monitoring applications including perinatal screening, transplant monitoring, cancer therapy selection, and multiple-cancer early detection. The system...
- The National Institute of Arthritis and Musculoskeletal and Skin Diseases awarded Vecgen, Inc. $333,689 on September 1, 2026, under the Arthritis, Musculoskeletal and Skin Diseases Research program (CFDA 93.846) to develop and validate a diagnostic tool predicting immune responses to adeno-associated virus (AAV) gene therapies. Vecgen will clone and produce a comprehensive set of AAV vectors carrying FDA-approved gene therapy sequences and establish standardized immunogenicity benchmarks using...
The National Institutes of Health National Center for Advancing Translational Sciences awarded $306,872 to Endless Vectors Bioscience on September 3, 2026, under the NCATS Program (CFDA 93.350) to develop and validate a cell cycle-dependent replicating adeno-associated virus platform for treating neonatal inborn errors of metabolism. Endless Vectors will assess tissue-specific replication, episomal persistence, and long-term safety and efficacy of the CCRAAV platform in neonatal mouse liver models. The technology addresses mitotic dilution limitations of conventional recombinant AAV vectors in proliferating tissues by using scaffold/matrix attachment regions for nuclear tethering and replication initiation regions to sustain episomal replication alongside host cell division, maintaining long-term gene expression in growing organs. Preliminary studies demonstrate retention of 1–2.5 genome copies per cell and greater than 10 percent reporter-positive cells after approximately 60 cell doublings, compared to near-complete loss with standard AAV. This Phase I SBIR project aims to establish proof-of-concept for a low-dose, durable, and safe gene therapy platform applicable to metabolic disorders including methylmalonic acidemia and hereditary tyrosinemia type 1. Performance occurs in Cambridge, Massachusetts, with a period of performance from September 3, 2026, through August 31, 2027.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $306.9k | 9/3/26 |