CIRM awarded $4,048,253 to Dr. Joseph Anderson and his UC Davis team to develop a blood stem cell gene therapy that could help people seeking treatment for Tay‑Sachs disease.
Tay-Sachs disease is a rare genetic disorder caused by a deficiency in the Hex A enzyme. Without Hex A, certain fats accumulate in brain and nerve cells, causing progressive neurological decline.
The disease appears in infant, juvenile, and adult forms. More than 100 mutations in the Hex A gene can disrupt enzyme function. No effective treatments or cures currently exist for Tay‑Sachs.
The UC Davis team will genetically modify the patient’s blood stem cells to restore the missing Hex A enzyme.
Their goal is to complete safety studies and apply to the FDA for an Investigational New Drug, the authorization needed to begin a clinical trial.
“The successful development of this therapy will not only help patients with Tay‑Sachs but will demonstrate the use case of this approach for other monogenic neurodegenerative diseases,” the team said.
This work continues a previous CIRM grant.
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nice post.