This federal Project Grant award, valued at $253,167.00 and issued on August 23, 2024 by the National Institute of Child Health and Human Development (CFDA 93.865 - Child Health and Human Development Extramural Research), aims to develop a non-viral gene therapy approach to prevent fertility impairments in women affected by classic galactosemia, a rare genetic disorder. The key objectives of this research project are to: 1) Develop a nanoparticle-based gene therapy that can deliver episomal GALT...
This Project Grant award from the National Institute of General Medical Sciences (NIGMS) under the Biomedical Research and Research Training Program (CFDA 93.859) provides $1,095,274 to develop complementary biotherapeutic delivery platforms for enabling gene re-delivery. The award, effective August 1, 2024 through May 31, 2029, aims to pioneer innovative delivery strategies that overcome biological barriers and modulate immune responses to improve the effectiveness of adeno-associated virus...
This $195,625 Project Grant award from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) under the Diabetes, Digestive, and Kidney Diseases Extramural Research program (CFDA 93.847) will fund a 2-year study to test whether AAV9-based GALT gene therapy can prevent long-term complications in a rat model of classic galactosemia. The goal is to evaluate the long-term biochemical and phenotypic efficacy of a single dose of scAAV9-hGALT administered intravenously to neonatal...
The National Institute of Child Health and Human Development (NICHD) awarded a $295,923 Project Grant to Biostrategies LC under the Child Health and Human Development Extramural Research program (CFDA 93.865). The purpose of the award is to develop a "delivery-enhanced" gene therapy drug comprising an RTB:GALNS fusion protein for treating the rare metabolic disorder Mucopolysaccharidosis Type IVA (Morquio A Syndrome). The key objectives are to: 1) optimize the RTB:GALNS construct for...
This $367,059 federal Project Grant award from the National Institute of General Medical Sciences (NIGMS) under the Biomedical Research and Research Training program (CFDA 93.859) supports research to develop a better understanding of the genomic, epigenomic, and transcriptomic outcomes of gene therapy. The research aims to explore the long-term effects of vector integration, the impact of cell/tissue type and promoters on vector behavior, and the host response to different gene therapy delivery...
This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), provides $2,654,903 to The Children's Hospital Corporation (doing business as Boston Children's Hospital) to develop innovative gene therapy technologies for the in vivo and ex vivo delivery of genetic payloads to hematopoietic stem cells. The objectives are to: (1) develop integrating vectors to deliver a curative transgene for X-linked...
This federal Project Grant award of $364,167, provided by the National Institute of General Medical Sciences (NIGMS) under the Biomedical Research and Research Training program (CFDA 93.859), aims to develop novel non-viral gene delivery methods for cell therapies. The key goals of this collaborative multi-PI proposal are to: 1) develop polymeric gene delivery vehicles to improve siRNA (or other cargo) delivery to T cells and hematopoietic stem cells (Aim 1); 2) discover and test novel drugs...
This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research (CFDA 93.837) program, provides $2,368,153.00 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-D27-264CFTR) in rhesus macaques and ferret models, as well as conduct a Phase I...
This federal Project Grant award, provided by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837), aims to develop a novel hydrogel-based system for delivering gene therapy to the heart. The primary awardee, Emory University, will engineer a hydrolysis-cleavable polyethylene glycol (PEG) hydrogel to target the epicardium and optimize cardiac transduction of gene therapy vectors. The total funding amount is $635,300.00, with a...
This Project Grant award under the National Institute of General Medical Sciences (NIGMS) Biomedical Research and Research Training Program (CFDA 93.859) supports the development of prototype mouse models to investigate the SEND (Selective Endogenous Encapsidation for Cellular Delivery) system for mRNA delivery and gene therapy applications. The $330,025 award, provided by the Department of Health and Human Services (HHS), will fund the creation of three genetically engineered mouse models to...