Project Grant R41HL180243
- This $888,836 Project Grant award from the National Heart, Lung, and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research) supports research by The Children's Hospital of Philadelphia (CHOP) to develop gene therapy approaches for treating hemophilia A. The key objectives are to: 1) Target factor VIII expression to liver sinusoidal endothelial cells (LSECs) rather than hepatocytes using genome editing and AAV gene addition methods, and 2) Compare the biological effects of expressing...
- This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI) under CFDA 93.837 - Cardiovascular Diseases Research provides $704,635 to the University of Florida to develop optimized adeno-associated virus (AAV) vectors for gene therapy of hemophilia. Specifically, the award aims to engineer both capsid- and genome-modified AAV3 vectors that can transduce human hepatocytes more efficiently than current AAV5 vectors, potentially reducing the required vector dose and...
- This Project Grant award of $636,982.00 was provided by the National Heart, Lung, and Blood Institute under the Cardiovascular Diseases Research federal grant program (CFDA 93.837). The grant supports research to comprehensively investigate the proteolytic mechanisms that govern factor VIII regulation before and after activation, which is critical for maintaining normal blood clotting and preventing bleeding disorders like hemophilia A. The project aims to understand how proteolysis of factor...
- This Project Grant award from the National Heart Lung and Blood Institute under the Cardiovascular Diseases Research program (CFDA 93.837) provides $814,209 to the Fred Hutchinson Cancer Center to support research on the use of targeted nanoparticles for in vivo hematopoietic stem cell gene therapy. The project aims to refine the target for hematopoietic stem cell gene therapy and develop a method to genetically modify these cells directly in the patient, which would improve the feasibility...
- 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 of $234,750.00 from the National Heart Lung and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research) aims to examine the immunological mechanisms that initiate and orchestrate the development of anti-factor VIII alloantibodies, known as inhibitors, in patients with severe hemophilia A. The research is being conducted by Emory University and seeks to identify key immune pathways and risk factors that predict the likelihood of inhibitor development, with the goal...
- This Project Grant award of $470,553 from the National Institute of Allergy and Infectious Diseases (CFDA 93.855 - Allergy and Infectious Diseases Research) supports research at Emory University to investigate the genetic regulation of factor VIII inhibitor development in hemophilia A patients. The research aims to elucidate the mechanisms driving immunity to factor VIII replacement therapy, which can lead to the development of neutralizing antibodies that render the treatment ineffective. Using...
- This Project Grant award of $395,314 from the National Heart, Lung, and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research) to The Leland Stanford Junior University aims to define the key cellular components and early immunologic events that mediate factor VIII immunity in persons with hemophilia A. The research will investigate the role of type 2 conventional dendritic cells (CDC2) in the development of anti-factor VIII antibodies, with the long-term goal of developing targeted...
- This Project Grant award of $786,188, provided by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837), aims to develop a novel gene therapy platform to prevent the failure of prosthetic bypass grafts used to treat arterial blockages in the lower extremities. The research, conducted by Beth Israel Deaconess Medical Center, Inc. (BIDMC), focuses on optimizing a biodegradable hydrogel system that can deliver small interfering RNA...
- This Project Grant, awarded by the National Heart, Lung, and Blood Institute (CFDA Program 93.837 Cardiovascular Diseases Research), provides $787,021 to The Leland Stanford Junior University (Stanford University) from September 20, 2025 through June 30, 2030. The grant supports research aimed at understanding the mechanisms of Factor VIII inhibitor formation in persons with hemophilia A, with the long-term goal of developing targeted therapies to prevent inhibitor development during early...
This Project Grant award for $314,363, provided by the National Heart, Lung, and Blood Institute under the Cardiovascular Diseases Research program (CFDA 93.837), aims to develop a novel gene therapy technology called STITCHR to enable full gene replacement for Hemophilia A. The proposed research will engineer robust adeno-associated virus (AAV) constructs to deliver and express the full-length Factor VIII gene, addressing the limitations of current truncated versions. The project will evaluate different tissue-specific promoters to optimize Factor VIII expression and durability, and assess the therapeutic and unintended host responses using a Hemophilia A mouse model. This project seeks to validate the STITCHR approach for large gene replacement therapies and establish a foundation for future research in higher mammals, with the goal of providing an advanced treatment option for Hemophilia A.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $314.4k | 9/12/25 |