The National Science Foundation (NSF) awarded a $274,438 SBIR Phase I grant to Script Biosciences Inc. under the NSF Technology, Innovation, and Partnerships (CFDA 47.084) program. The project, titled "CASPLUS: A Safer, More Efficient, and Targeted Method of Deploying CRISPR In Vivo," aims to develop a platform technology that significantly reduces the potential inaccuracy of current gene editing techniques. The innovation targets the desired gene, increasing the safety and efficacy of...
This $325,000 project grant awarded by the National Institute of General Medical Sciences (NIGMS) under the Biomedical Research and Research Training program (CFDA 93.859) will support the development and testing of a new class of programmable, non-viral gene therapy carriers and cargo known as virus-inspired DNA origami (VIDO) vectors and repair templates. The project aims to demonstrate that VIDO vectors can effectively encapsulate and co-deliver CRISPR-Cas9 gene editing agents and repair...
Through a $1,000,000 Cooperative Agreement award from the National Science Foundation (NSF) Technology, Innovation, and Partnerships (CFDA 47.084) program, Optimeos Life Sciences, Inc., a for-profit biotechnology company, is developing a non-viral platform for delivering mRNA-based gene therapies. The project aims to validate a polymer-lipid hybrid formulation that can safely and repeatedly deliver genetic instructions to patients with rare urea cycle disorders, overcoming limitations of current...
This Project Grant award from the National Institute of General Medical Sciences (NIGMS) under the Biomedical Research and Research Training Program (CFDA 93.859) provides $364,167 to Villanova University to develop novel non-viral gene delivery methods for cell therapies. The key objectives are: The Bracaglia Lab will develop new polymeric gene delivery vehicles to improve the delivery of siRNAs and other payloads to T cells and hematopoietic stem cells (HSCs). Drs. Elmer and Huang will...
This Project Grant award from the National Institute of General Medical Sciences (NIGMS) Biomedical Research and Research Training program (CFDA 93.859) supports research to improve the efficiency of CRISPR gene editing in human pluripotent stem cells. The goal is to develop a novel carbon nanotube array technology that can efficiently transfer nucleic acids and proteins into these cells to enhance CRISPR-based gene editing and homology-directed recombination. The $275,045 award to Advanced Gene...
This Project Grant award from the National Science Foundation's (NSF) Technology, Innovation, and Partnerships program (CFDA 47.084) provides $275,000 to Targeting Systems to develop a novel intramuscular gene delivery platform capable of sustained expression and endogenous secretion of bispecific natural killer cell engager (BIKE) therapeutics to treat solid tumors like hepatocellular carcinoma. The platform aims to deliver BIKEs in a less invasive manner than current immunotherapies,...
Acrigen Biosciences, Inc. was awarded a $1,000,000 cooperative agreement from the National Science Foundation under the Engineering program (CFDA 47.041) to develop a curative gene therapy for spinal muscular atrophy. Over the two year period from March 2022 to February 2024, Acrigen will engineer an anti-CRISPR protein to increase the precision and safety of CRISPR-Cas gene editing. They will pair this with a novel CRISPR nuclease and validate the system for editing and disease correction in...
This $1,716,429.77 Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research Program (CFDA 93.837) aims to develop a novel nonviral delivery approach for introducing CRISPR-Cas9 gene editing reagents into hepatocytes to treat familial hypercholesterolemia (FH). The project will directly compare the efficiency and specificity of gene editing using different nonviral delivery methods, including lipid nanoparticles (LNPs),...
This Project Grant award from the National Science Foundation (NSF) under the Technology, Innovation, and Partnerships (CFDA 47.084) program provides funding of $304,951 to Neucore Bio, Inc. to develop an innovative gene therapy delivery system using engineered extracellular vesicles (EVs). The project aims to address key technical challenges in the scalable manufacturing of EV-based gene therapies, focusing on optimizing methods for EV isolation, purification, and cargo loading to enhance...
This Project Grant from the National Science Foundation's Technology, Innovation, and Partnerships program totaling $100,000 will support research to develop next-generation clustered regularly interspaced short palindromic repeat (CRISPR) systems for curing inherited diseases. Specifically, the awardee, the University of California, Berkeley, will work to circumvent in vivo delivery challenges with CRISPR-Cas9 by developing miniature RNA-guided genome editors smaller than Cas9 that can be...