The California Institute for Regenerative Medicine (CIRM) has awarded $4,048,253 to Dr. Joseph Anderson and his team at UC Davis to develop a blood stem cell gene therapy for the treatment of Tay-Sachs disease.
Tay-Sachs disease is a rare genetic disorder where a deficiency in the Hex A gene results in excessive accumulation of certain fats in the brain and nerve cells and causes progressive dysfunction.
There are several forms of Tay-Sachs disease, including an infant, juvenile, and adult forms. Over a hundred mutations in the disease-causing Hex A gene have been identified that result in enzyme disfunction. There are currently no effective therapies or cures for Tay-Sachs.
The UC Davis team will genetically modify the patient’s own blood stem cells to restore the Hex A enzyme that is missing in the disease.
The goal is to complete safety studies and to apply to the US Food and Drug Administration for an Investigational New Drug (IND), the authorization needed to begin a clinical trial in people.
“The successful development of this therapy will not only help patients with Tay-Sachs but will demonstrate the use case of this therapeutic approach for other monogenic neurodegenerative diseases,” the UC Davis team said.
This work is a continuation of a CIRM grant that the team received.