This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), provides $2,368,153 to The Johns Hopkins University to conduct research on cystic fibrosis gene therapy using adeno-associated viral (AAV) vectors. The research aims to evaluate the safety and efficacy of an AAV1 vector containing a truncated CFTR gene (AAV1-CBD27-264) in preclinical studies using rhesus macaques and F508-del cystic fibrosis...
This federal Project Grant award, valued at $540,524 and effective from March 1, 2025 to February 28, 2029, was provided by the National Institute of Biomedical Imaging and Bioengineering (NIBIB) under the "Discovery and Applied Research for Technological Innovations to Improve Human Health" (CFDA 93.286) program. The primary objective of this award is to develop an enhanced RNA switch technology based on the T3H38 ribozyme, which can enable precise dose control and temporal regulation...
The National Institute of Biomedical Imaging and Bioengineering (NIBIB) awarded Cornell University a $435,412 Project Grant under the Discovery and Applied Research for Technological Innovations to Improve Human Health program (CFDA 93.286). The goal of this 2-year project is to develop a biomaterial-driven, immunosuppressive platform that enables the re-administration of engineered adeno-associated virus (AAV) gene delivery vectors while maintaining their transduction efficiency....
The National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH), awarded a $1,845,871 Project Grant under the Cardiovascular Diseases Research program (CFDA 93.837) to the University of Kansas Medical Center Research Institute, Inc. (KUMCRI) for the "Development of Novel Approaches for Gene Editing Therapies of Cystic Fibrosis". The project aims to develop a novel gene editing method called Homology-Independent Targeted Integration (HITI) to...
This Project Grant from the National Science Foundation's Technology, Innovation, and Partnerships program totaling $100,000 will support research to develop next-generation clustered regularly interspaced short palindromic repeat (CRISPR) systems for curing inherited diseases. Specifically, the awardee, the University of California, Berkeley, will work to circumvent in vivo delivery challenges with CRISPR-Cas9 by developing miniature RNA-guided genome editors smaller than Cas9 that can be...
The National Heart, Lung, and Blood Institute (NHLBI) awarded a $299,995 Project Grant under the Cardiovascular Diseases Research (CFDA 93.837) program to Nanite Inc., a small disadvantaged business in Massachusetts. The grant will fund the development of computationally-designed polymer-based delivery vehicles for mRNA payloads to treat cystic fibrosis (CF), a debilitating genetic disorder affecting the lungs. The project aims to leverage computational modeling and high-throughput polymer...
The National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), awarded a $783,000 Project Grant to the University of Toronto to develop a novel, non-viral base editing platform for treating cystic fibrosis (CF) caused by nonsense mutations. The project aims to refine adenine base editors (ABEs), formulate lipid nanoparticles (LNPs) for targeted pulmonary delivery, and validate the therapeutic efficacy and safety in a mouse model of CF. By...
The National Heart, Lung, and Blood Institute (NHLBI) awarded a $320,251 Project Grant (CFDA 93.837 Cardiovascular Diseases Research) to Ogb5, Inc. for the development of a non-viral nanoparticle gene therapy delivery platform to treat cystic fibrosis and other chronic lung diseases. The grant aims to further advance Ogb5's proprietary technology, which utilizes DNA and RNA nanoparticles combined with uptake enhancers to improve gene transfer efficiency compared to current viral-based delivery...
This National Science Foundation (NSF) Technology, Innovation, and Partnerships (CFDA 47.084) Project Grant award for $275,000 will support the development of a plasmonic nanopore sensor device for quality control of adeno-associated viruses (AAVs) used in gene therapy. The proposed device will enable accurate characterization of AAV payload (single-stranded DNA, double-stranded DNA, or empty) at low, pre-scale-up concentrations to optimize formulations. The project aims to demonstrate the...
This National Science Foundation Project Grant award of $299,998 provides funding from July 1, 2022 through June 30, 2024 to develop magnetothermal control of cell fates and function. The award is made under the NSF Engineering program (CFDA 47.041) to Michigan State University. Specifically, the funding supports research to engineer prokaryotic cells as programmable pseudo-organelles that can be controlled using alternating magnetic fields to activate mammalian transcription factors and...