Vesicle Therapeutics Inc aims to develop and commercialize a new therapy for homozygousfamilial hypercholesterolemia (hoFH). A majority of hoFH is caused by mutations in both allelesof the gene encoding the LDL receptor (LDLR). Since the efficacy of both statins and PCSK9 antibody therapies largely depends on functional LDL receptors, patients with hoFH show limitedresponses to these existing therapies. Other than recently approved evinacumab, an antibodytherapy against Angiopoietin-like 3 (ANGPTL3), few options are available to treat this disease.There is still an unmet medical need for developing new targeted therapies to lower both LDL-Cand lipids in hoFH. GRP146 has recently emerged as an exciting and potential new therapeutictarget for hoFH. Our goal is to leverage our proprietary delivery technology to develop a newGPR146 therapy for hoFH. The overall objective of this Phase I SBIR project is to demonstratethat silencing of GPR146 by proprietary gectosome delivery of LwaCas13a/GPR146 crRNA isefficacious in lowering both LDL-C and lipids with acceptable safety profile in mice, enabling anew therapy for hoFH.
Public Health Relevance Statement: Project Narrative The goal of this phase I SBIR project is to demonstrate that silencing of GPR146 by gectosome delivery of LwaCas13a/GPR146 crRNA is efficacious in lowering LDL-C with acceptable safety profile in mice.
Project Terms:
|
