Our current project focuses on curing familial hypercholesterolemia (FH) to reduce patient's risk of cardiovascular disease through an effective gene-edited vaccine
Get in TouchThere are current medications and treatments that patients can use to limit the effects of FH, however, there are no current cures available.
It is a drug that lowers LDL-cholesterol by 30% by blocking the liver's ability to produce cholesterol.
It is a medication that lowers LDL-cholesterol levels by 13% to 20% by preventing cholesterol absorption from the intestines.
Keeping a balanced lifestyle can lower the risk of diseases associated with FH.
We are developing a vaccine that will use CRISPR technology to
edit one of the three mutated genes that cause FH in patients. The
Cas9 will be embedded in lipid nanoparticles and targeted to the
liver cells through a guide RNA. We hypothesize that by targeting
the expressed mutated genes in the liver, we can eradicate FH in patients.
Before getting the vaccine treatment, the patient will need to get a genetic testing through whole-genome sequencing to find the FH-causing mutated gene.
Before we begin testing our vaccine on humans, preclinical trials will be conducted on animals to evaluate safety, efficacy, and dosing. We estimate that the vaccine will be tested on 60 rodents over the course of 2 years before being approved.
Clinical trials will test the vaccines on humans to test for efficiency. This process may last 5+ years before it can be FDA approved. During this period, gene-editing methods and efficiency will improve as technology advances.
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