Therapeutic Gene-Editing in Alzheimer’s disease

Our overall goal is to take a CRISPR based gene therapy for Alzheimer’s disease to the clinic.

Our overall goal is to take a CRISPR based gene therapy for Alzheimer’s disease to the clinic. Broadly speaking, our approach restores the physiologic balance of the amyloid pathway – decreasing neurotoxic protein fragments, while promoting neuroprotective protein fragments. There are over a 100 ongoing clinical trials worldwide using CRISPRs, and the results so far with non-neurologic disorders have shown unprecedented (almost 100%) efficacy. Our broad vision is to apply CRISPRs for devastating neurologic illnesses like Alzheimer’s disease.

If successful, this will be the first CRISPR based therapeutic for any neurologic disease.

Upon completion, we will be closer to a novel “one and done” CRISPR-based gene therapy for Alzheimer’s disease.