The federal Project Grant award R01HD117940, totaling $696,246.00, was provided by the National Institute of Child Health and Human Development (NICHD) under the Child Health and Human Development Extramural Research program (CFDA 93.865). The grant will fund a 5-year study titled "Evaluating Maternal and Neonatal Pharmacokinetics of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators - The ELECTRA Pregnancy Study". The study aims to 1) determine the...
This $2,389,542 federal Project Grant award was provided by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research (CFDA 93.837) program. The grant funds research to investigate the mechanisms by which CFTR correctors, a type of cystic fibrosis treatment, modulate the activity of calcium-activated potassium (KCa) channels. The research aims to define how CFTR correctors inhibit the intermediate conductance KCa3.1 channel and potentiate the large...
This $2,368,153 Project Grant awarded by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) will support research to evaluate the safety and efficacy of using recombinant adeno-associated viral (AAV) vectors to deliver a truncated version of the cystic fibrosis transmembrane conductance regulator (CFTR) gene as a gene therapy for cystic fibrosis. The key objectives are to: 1) assess single-dose administration of the...
The federal Project Grant award of $320,251 from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), supports the development of a non-viral nanoparticle gene therapy delivery platform to treat cystic fibrosis and other chronic lung diseases. The project aims to demonstrate that the technology can effectively deliver therapeutic genes, such as the cystic fibrosis transmembrane conductance regulator (CFTR), to human bronchial...
The National Heart, Lung, and Blood Institute (NHLBI), a division of the National Institutes of Health (NIH), awarded a $750,672 Project Grant (R01HL172845) under the Cardiovascular Diseases Research (CFDA 93.837) program to The Leland Stanford Junior University. The grant, with a performance period from December 10, 2024 to November 30, 2028, will support research to identify the cellular protein quality control (PQC) machinery that governs the stability and trafficking of the cystic fibrosis...
This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research Program (CFDA 93.837) provides $629,319 to Emory University for a project titled "Tuning Translation Efficiency to Overcome Refractory Defects in CFTR." The project aims to investigate how modulating ribosomal protein expression can rescue the synthesis and assembly of cystic fibrosis transmembrane conductance regulator (CFTR) variants with premature...
This federal Project Grant award, provided by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research Program (CFDA 93.837), will fund research focused on understanding the role of neutrophil extracellular traps (NETs) in lung inflammation and infection in mucoobstructive lung diseases like cystic fibrosis. The total award amount is $756,875.00 over a 5-year period from January 1, 2025 to December 31, 2029. The primary awardee is the University of...
This federal Project Grant award, valued at $2,000,987.00 and provided by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837), supports research to examine how the innate antiviral immune response and associated metabolic changes in the respiratory epithelium facilitate viral-bacterial co-infections by Pseudomonas aeruginosa in cystic fibrosis. The primary awardee, Trustees of Dartmouth College, will use an improved model of P....
This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) provides $730,494 to Emory University to conduct research on the mechanisms linking the cystic fibrosis transmembrane conductance regulator (CFTR) protein to dysregulated barrier function, insulin receptor function, and glucose transport in the cystic fibrosis (CF) lung. The research aims to identify the underlying mechanisms for the...
This 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) provides $613,558 to Genetobe Inc. to develop novel, selective sodium-dependent glucose cotransporter 1 (SGLT1) inhibitor compounds as an adjunctive therapy for cystic fibrosis (CF) associated metabolic disorders. The research aims to mitigate CF-related liver disease and CF-related diabetes,...
This federal Project Grant award of $2,519,726.00, provided by the National Heart Lung and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research), aims to analyze the pharmacokinetics, pharmacogenetics, and clinical utility of the triple combination therapy (TC) elexacaftor/tezacaftor/ivacaftor for cystic fibrosis (CF) patients. The study, titled "ENSURING ACCESS TO OPTIMAL THERAPY IN CF: ENACT STUDY," will:
Analyze the population pharmacokinetics of the TC compounds in plasma to determine pharmacokinetic parameters and correlate drug exposure with drug response.
Identify novel genetic variants associated with TC concentration or response, including pulmonary measures.
Conduct a prospective study to assess the relationship between TC concentration and neuropsychological side effects, and evaluate the utility of dose titration to balance side effects with optimal clinical responses.
The project will be led by the Arkansas Children's Research Institute, with a sub-award to the University of Washington to support participant recruitment, neuropsychological assessments, and sample processing over the 5-year project period starting July 2024.