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.

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.

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)