Central Nervous System

Lead Indication Approach Program Research Preclinical Phase 1/2 Phase 3
ZFP TF (repress) Shire
Research Phase complete
Preclinical Phase in progress
Phase 1/2 Phase not started
Phase 3 Phase not started

Huntington’s disease is an inherited, progressive neurologic disease for which there is no treatment or cure.  The disease is caused by a mutation in the HTT gene. In collaboration with Shire, Sangamo Therapeutics is developing ZFP Therapeutics that can selectively repress the expression of the mutant disease causing form of HTT while leaving expression levels of the normal gene unchanged. Preclinical studies in animal models of the disease are ongoing, and Shire is responsible for all clinical development activities including filing the IND application.

Research Phase in progress
Preclinical Phase not started
Phase 1/2 Phase not started
Phase 3 Phase not started

Sangamo Therapeutics is using its zinc finger protein transcription factor (ZFP-TF)-mediated gene regulation technology to develop potential one-time treatments for neurodegenerative diseases associated with tau protein aggregation, including Alzheimer’s disease and other tauopathies. Sangamo intends to seek a partner with CNS disease area expertise for the development and commercialization of its therapeutic gene regulation approach to these indications.

About Huntington’s Disease

Huntington’s disease (HD) is an inherited, progressive neurologic disease for which there is no treatment or cure. Symptoms include deterioration of muscle control, cognition and memory. 

The disease is caused by a particular type of mutation in a single gene, the HTT gene. Most patients inherit one normal and one defective or mutant copy of the HTT gene, which causes HD. The mutation is characterized by expansion of a repeated stretch of DNA sequence within the gene called a “CAG repeat.” A normal copy of the HTT gene usually has 10 to 29 of these CAG repeats but a defective copy has many more—generally greater than 39 repeats. While the protein produced by the normal copy of the gene appears to be essential for development (mice lacking the gene do not survive to birth), the product of the mutated gene is damaging to cells.

About Alzheimer's Disease and Tauopathies

Alzhiemer's disease and other tauopathies are a class of neurodegenerative diseases characterized by the aggregation of hyper-phosphorylated tau protein, known as neurofibrillary tangles, in the brain leading to widespread neuronal dysfunction and loss.

Although the cause of Alzheimer's disease is still not fully understood, the histopathological hallmarks of the disease include intracellular tau protein aggregates and extracellular amyloid plaque accumulation.  The reduced expression of the tau gene has been shown to provide neuronal protection and reversal of pathology in disease models of Alzheimer's and other tauopathies.

HD is usually fatal within 10 to 20 years after the onset of symptoms

Current stats according to the Huntington’s Disease Society of America (HDSA)

~30,000

People in the U.S. have HD

According to the Huntington's Disease Society of America (HDSA)

~200,000

People in the U.S. are at risk of developing the disease

According to the Huntington's Disease Society of America (HDSA)