Project Grant U1A41739

This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research federal grant program (CFDA 93.837), provides $306,870 to Kinetiq Therapeutics LLC, a life science startup in Texas, to conduct proof-of-concept studies on an enzyme replacement therapy (ERT) for Fanconi anemia. Fanconi anemia is a rare inherited disease characterized by bone marrow failure and malignancies. The project aims to demonstrate the feasibility of using an...

This $195,625 Project Grant awarded by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) under CFDA Program 93.847 "Diabetes, Digestive, and Kidney Diseases Extramural Research" aims to test whether a single dose of gene therapy using an adeno-associated virus (AAV9) vector delivering the human GALT gene can prevent long-term complications in a rat model of classic galactosemia. The 2-year study will be conducted by Emory University and will assess the...

This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), part of the Cardiovascular Diseases Research program (CFDA 93.837), provides $300,000 to Gigamune, Inc. to develop an in vivo gene therapy for sickle cell disease. The project aims to test a novel lentiviral vector technology for efficiently editing hematopoietic stem cells to knockout the BCL11A enhancer, a key genetic target for sickle cell treatment. Through in vitro assays, the Phase I effort will...

This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) aims to investigate the use of non-genotoxic hematopoietic stem cell transplantation (HSCT) to address the elevated risk of lymphoma, immune deficiencies, and chronic inflammation in an animal model of ataxia telangiectasia (A-T), a genetic disorder affecting DNA repair. The $117,375 award, effective September 1, 2024 through August 31, 2026, will...

This $131,250 Project Grant was awarded by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research federal grant program (CFDA 93.837). The grant will support a research project led by Vitalant Research Institute (VRI) to investigate the potential for transfusion-transmitted cerebral amyloid angiopathy (CAA). The key objectives are to: (A) assess the prevalence of amyloid-beta pathology among blood donors using the PTAU217 protein marker, and (B)...

This Project Grant award from the National Heart, Lung, and Blood Institute (CFDA 93.837 - Cardiovascular Diseases Research) provides $2,654,903.00 to The Children's Hospital Corporation (Boston Children's Hospital) to develop a novel alpha-retroviral vector-based platform for in vivo and ex vivo delivery of gene therapy payloads to hematopoietic stem cells (HSCs). The key objectives are to: 1) Develop integrating vectors to deliver a curative transgene to HSCs in vivo to treat X-linked severe...

This Project Grant award from the National Heart, Lung, and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research program) provides $483,852 to The General Hospital Corporation, doing business as Massachusetts General Hospital (MGH), to develop a high-throughput platform technology for measuring the kinetics and equilibrium concentration of sickle hemoglobin polymerization in single red blood cells. The platform will enable optimization of emerging sickle cell disease treatments, such as...

This Project Grant award of $765,622 from the National Heart, Lung, and Blood Institute (CFDA 93.837 - Cardiovascular Diseases Research) supports the development of orally bioavailable protein degraders to elevate fetal hemoglobin (HBF) levels and treat sickle cell disease (SCD). The primary awardee, Yale University, will collaborate with The Children's Hospital of Philadelphia to test the degraders in primary human erythroid cells and mouse models. This research aims to discover new small...

This Project Grant award from the National Center for Advancing Translational Sciences (NCATS), under CFDA program 93.350, provides $489,500.00 to The Children's Hospital of Philadelphia Research Institute to conduct research on familial platelet disorder with associated myeloid malignancy (FPDMM). The key objectives are to study the effects of the compound Repsox on the RUNX1 transcription factor to correct both the platelet defects and leukemic risk associated with FPDMM, a rare genetic...

This Project Grant award from the National Heart Lung and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research) to the University of North Carolina at Chapel Hill provides $573,821 to conduct research on the role of endothelin-1 (ET-1) signaling in promoting iron overload and kidney disease in sickle cell disease. The key objectives are to: 1) determine how ET-1/ETA receptor signaling increases iron uptake in proximal tubule cells, 2) define how ET-1/ETA signaling reduces iron export,...