Project Grant R43AI142839

This Project Grant award from the National Center for Advancing Translational Sciences (NCATS) under CFDA 93.350 will provide $463,150 to The Regents of the University of California, San Francisco (UCSF) to establish proof-of-concept for enzyme replacement therapy (ERT) as a lifesaving treatment for sphingosine phosphate lyase insufficiency syndrome (SPLIS), an ultra-rare and often lethal metabolic disorder. The project aims to demonstrate the efficacy of ERT in preserving kidney function and...

This Project Grant award from the National Institute of Diabetes and Digestive and Kidney Diseases (CFDA 93.847) provides $299,648 to Cometa Therapeutics Inc. to conduct enabling studies for CMT-101, a novel gene therapy for Glycogen Storage Disease 1B (GSD-1B). GSD-1B is a rare metabolic disorder caused by mutations in the G6PT1 gene, resulting in impaired glucose regulation and serious health complications. The funding will support preclinical development activities, including dose-finding and...

The National Center for Advancing Translational Sciences (NCATS), under the CFDA Program 93.350, awarded a $295,923 Project Grant to Kinetiq Therapeutics LLC to conduct preclinical efficacy studies of a subcutaneous enzyme therapy for Fabry disease. The proposed therapy, named BRYSQ, aims to transform the current standard of care by providing a convenient, self-administered treatment that could improve patient quality of life, reduce hospital use, and lower treatment costs compared to the...

The National Institutes of Health National Institute of Diabetes and Digestive and Kidney Diseases awarded $3.6 million under the Diabetes, Digestive, and Kidney Diseases Extramural Research program (CFDA 93.847) to Anagram Therapeutics Inc. to develop a novel pancreatic enzyme replacement therapy. Specifically, Anagram will engineer lipase enzymes for improved stability against low pH and proteolytic degradation. This will allow for effective digestion of fats in both the stomach and small...

This Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS), under the Extramural Research Programs in the Neurosciences and Neurological Disorders (CFDA 93.853) program, provides $461,701 to Tega Therapeutics Inc. to develop and validate an intracerebroventricular (ICV) enzyme replacement therapy for the treatment of GM1 gangliosidosis, a rare lysosomal storage disease affecting the central nervous system. The key objectives are to engineer cell lines to...

The National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH), awarded a $306,870 Project Grant (CFDA 93.837 - Cardiovascular Diseases Research) to Kinetiq Therapeutics LLC, a life science startup in Texas. The funding will support proof-of-concept studies to evaluate the feasibility of an enzyme replacement therapy (ERT) targeting aldehyde toxicity as a potential treatment for Fanconi anemia, a rare inherited disorder characterized by bone marrow failure...

This federal Project Grant award from the National Institute of Allergy and Infectious Diseases (CFDA 93.855 - Allergy and Infectious Diseases Research) provides $2,216,525.00 to Cedars-Sinai Medical Center to conduct a randomized controlled trial investigating the effects of pre-implantation, intrarenal artery injection of C1 esterase inhibitor (C1INH) on kidney transplant outcomes. The primary objective is to determine if this complement system inhibition therapy can improve allograft function...

This Project Grant award from the National Institute of Allergy and Infectious Diseases (NIAID), under the Allergy and Infectious Diseases Research program (CFDA 93.855), will provide $231,345.00 to the University of California, San Diego (UCSD) to conduct research on the role of heparan sulfate in regulating the complement system. The project aims to (i) define the binding affinities of complement proteins Factor H and Factor H-related proteins for heparan sulfate, and quantify complement...

The federal Project Grant award of $2,368,153 from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) supports research to evaluate the safety and efficacy of using adeno-associated viral (AAV) vectors for cystic fibrosis (CF) gene therapy. The key objectives are to: Assess single-dose administration of an AAV1 vector containing a truncated CFTR gene (AAV1-D27-264CFTR) in rhesus macaques. Determine the therapeutic effects of...

The National Institute of Allergy and Infectious Diseases (NIAID) awarded a $149,008 Cooperative Agreement (CFDA 93.310 Trans-NIH Research Support) to Cartesian Therapeutics Inc. to conduct a Phase 2 clinical study evaluating the safety and efficacy of their Descartes-08 therapy for treating systemic lupus erythematosus (SLE). Descartes-08 is an RNA-engineered chimeric antigen receptor (CAR) T cell therapy that targets B cell maturation antigen (BCMA) expressed on plasma cells responsible for...