This $367,059 Project Grant award from the National Institute of General Medical Sciences (NIGMS) Biomedical Research and Research Training Program (CFDA 93.859) aims to develop a better understanding of the genomic, epigenomic, and transcriptomic outcomes of gene therapy. The research will explore key questions around the long-term effects of vector integration, the role of cell/tissue type and promoters, and differential host responses to various gene therapy delivery vehicles. The...
This Project Grant award from the National Institute of General Medical Sciences (NIGMS) Biomedical Research and Research Training program (CFDA 93.859) provides $833,652 to the University of Connecticut to develop novel DNA-inspired biomaterials for effective intracellular delivery of messenger RNA (mRNA) therapeutics and research tools. The research aims to optimize the biointerface properties of Janus base nanoparticles (JBNPs) using computational molecular design and experimental validation,...
This Project Grant award from the National Cancer Institute (CFDA 93.398 Cancer Research Manpower) provides $163,640 to the University of Virginia to develop novel non-viral gene delivery platforms for the treatment of glioblastoma, an aggressive brain cancer. The research aims to improve the therapeutic accumulation and delivery of gene therapies by leveraging brain-penetrating nanoparticles and focused ultrasound technology. Key objectives include investigating the tumor-targeting and...
This Project Grant award of $388,621 from the National Institute of General Medical Sciences (NIGMS) Biomedical Research and Research Training Program (CFDA 93.859) will support the development of novel molecular designs with therapeutic potential. The key objectives are: Developing ligand-heterocycle conjugates as targeted RNA degraders with rapid kinetics and selectivity for cancer-associated G-quadruplexes. Engineering nucleic acid polymers with (thio)phosphoramidate backbones as biostable...
This Project Grant award of $450,000 from the National Science Foundation's Engineering program (CFDA 47.041) to North Carolina State University is for developing bio-censors that can simultaneously quantify capsid and transgene titers in adeno-associated viral (AAV) vectors used for gene therapy. The research aims to: 1) investigate peptide ligands' differential recognition of AAV capsids, 2) engineer extended-gate field effect transistor bio-censors to enhance detection and discrimination, and...
This Project Grant award of $1,640,169 from the National Institute of General Medical Sciences (NIGMS) Biomedical Research and Research Training program (CFDA 93.859) supports Synvivia, Inc.'s development of a platform to identify cyclic peptides that can increase the yield and quality of adeno-associated virus (AAV) vectors for gene therapy manufacturing. The key products and services to be delivered include: Demonstrating that Synvivia's cyclic peptide discovery library contains compounds that...
This Project Grant award, provided by the National Institute of General Medical Sciences (NIGMS) under the Biomedical Research and Research Training program (CFDA 93.859), aims to measure and understand the molecular mechanisms of viral nucleocapsid self-assembly and disassembly. The $370,190 award, effective May 10, 2025 through March 31, 2030, will use a combination of quantitative experimental methods, including interferometric scattering microscopy and cryo-electron microscopy, to study...
The National Institute of Biomedical Imaging and Bioengineering (NIBIB) awarded a $1,158,262 Project Grant (CFDA 93.286) to the University of Massachusetts Medical School to develop a synthetic RNA switch technology that can precisely control the timing and dosage of gene therapies delivered via adeno-associated viral (AAV) vectors. The project aims to optimize a highly efficient, leak-free RNA switch system to regulate transgene expression for therapeutic applications, including dose control of...
This Project Grant award from the National Institutes of Health (NIH) Office of the Director under the Trans-NIH Research Support program (CFDA 93.310) provides $487,499 to the Whitehead Institute for Biomedical Research to develop new strategies for highly efficient production of cell-derived bioparticles as delivery vehicles for macromolecular therapeutics. The research aims to identify and manipulate genes in bioparticle producer cells to enhance production efficiency, and establish a...
This Project Grant award from the National Institute of Allergy and Infectious Diseases (NIAID), under the Allergy and Infectious Diseases Research program (CFDA 93.855), provides $769,737 to Yale University to develop capsid-engineered adeno-associated viral (AAV) vectors that can specifically target and deliver gene therapies to human T cells and myeloid cells. The goal is to use these AAV vectors to deliver a designer recombinase called BREC1, which can eliminate HIV provirus integrated in...