Welcome to the CGTA Research Group
Our name stands for “CNS Gene therapy Translation Acceleration.”
Our goal is to bring gene therapies for rare Central Nervous System diseases into clinical trials and use as quickly and efficiently as possible. Our first project is focused on developing a gene therapy for Huntington’s disease.
We have initiated our main study and now have seven animals in the “in-life” stage of our study plan. Interim results will be presented at upcoming conferences in the spring of 2023.
After unavoidable delays due to the COVID-19 pandemic, we have initiated our study of the safety of long-term huntingtin-lowering in the brain with the delivery of a huntingtin-lowering agent to the thalamus of a pilot animal. The purpose of the pilot is to confirm the activity and the dosage of the agent to be used in the main study that will start later this year.
We have received word from the National Institute of Neurological Disorders and Stroke as follows: “[your] application was reviewed by the National Advisory Neurological Disorders and Stroke Council at its recent meeting. We are pleased to inform you that we anticipate funding this application. Once all pre-award requirements have been met, a notice of grant award will be issued…”
We will provide more details here once this multi-year research project is underway!
Our Huntington’s disease-related grant application to the National Institute of Neurological Disorders and Stroke (NINDS) was reviewed, discussed, and favorably scored by the scientific study section in October, 2019. Here’s hoping the NIH budget is sufficient this year for our score to be within the pay line.
Our grant application to the National Institute of Neurological Disorders and Stroke (NINDS) was received in good order in June, 2019, and will be reviewed by the Chronic Dysfunction and Integrative Neurodegeneration Study Section on October 15, 2019.
We are in the process of preparing a grant application for submission to NINDS, regarding huntingtin-lowering. This application will be responsive to the reviews of our latest application (which received positive reviews, but not a sufficiently good priority score to make the funding cut), and also recent findings about the effects of huntingtin-lowering in animals. See the literature review by Kaemmerer and Grondin, just published this month.