This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), provides $765,622 to support research into developing orally bioavailable protein degraders to treat sickle cell disease (SCD). The award aims to create small molecule degraders of two fetal hemoglobin gene repressors in order to elevate fetal hemoglobin levels and prevent red blood cell sickling, a key pathological mechanism in SCD....
This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), provides $300,000 to Parmedics, Inc., a for-profit, woman-owned biotechnology company, for the development of small molecule protease-activated receptor-2 (PAR2) antagonists as potential oral therapeutics for asthma. The goal is to characterize two novel PAR2 antagonists, C937 and C938, with improved pharmacodynamics and pharmacokinetics to enable...
This National Heart, Lung, and Blood Institute (NHLBI) Project Grant under CFDA 93.837 - Cardiovascular Diseases Research program provides $202,366 in funding to Emory University to investigate the role of heme-induced complement activation in sickle cell disease (SCD) pregnancy. The research aims to characterize the effects of SCD and heme-induced complement activation on pregnancy outcomes in a preclinical mouse model (Aim 1), and determine if heme and/or complement modulation can improve...
This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), is focused on characterizing the role of the alternative complement pathway in the pathogenesis of acute chest syndrome (ACS) in sickle cell disease (SCD). The key objectives are to: 1) define the mechanisms by which complement contributes to the development of ACS using novel preclinical models, microfluidics, and in vitro assays; 2) characterize...
This Project Grant award, provided by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837), is focused on developing a high-throughput platform to measure the equilibrium and kinetics of hemoglobin polymerization in single red blood cells. The $483,852 award will enable the primary grantee, The General Hospital Corporation (dba Massachusetts General Hospital), to create this novel platform technology to optimize the development...
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) provides $1,829,965.51 to support a randomized, double-blind, placebo-controlled Phase 2 clinical trial of intravenous citrulline for vaso-occlusive crisis in sickle cell disease. The goal is to evaluate whether the administration of citrulline, which plays a role in nitric oxide production, can shorten hospital length of stay and decrease opioid...
This federal Project Grant award of $827,753.90 from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) will support research to investigate the role of thiol isomerases, specifically protein disulfide isomerase (PDI), in promoting vaso-occlusive events in sickle cell disease. The awardee, Bloodworks (doing business as Puget Sound Blood Center and Program), will conduct a series of integrated studies to mechanistically examine...
This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research federal grant program (CFDA 93.837), aims to characterize the role of Factor XII (FXII) in the thrombo-inflammatory complications of sickle cell disease (SCD). The $1,449,454 award to the University of North Carolina at Chapel Hill (UNC-CH) will fund research to: 1) identify disease-specific triggers of FXII activation, 2) characterize FXII effects on neutrophil...
This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), provides $608,126 to the Regents of the University of Minnesota to conduct research on developing and validating single-cell biomarkers for predicting clinical outcomes in sickle cell disease. The key objectives are to: 1) define the relationship between common sickle cell disease adverse outcomes and single-cell biochemical and mechanical...