Sangamo Therapeutics and Voyager Therapeutics Enter License Agreement for Epigenetic Regulation Treatment of Prion Disease - Seite 2

“We believe the novel, brain-penetrant capsids emerging from our TRACER capsid discovery platform have the potential to enable gene therapies for a wide variety of diseases of the central nervous system – far more than we could prosecute internally,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager. “We continue to explore a variety of capsid license structures, such as in this transaction with Sangamo, to leverage our technology for more programs, and ultimately, more patients.”

About Prion Disease

Prion disease is a rapidly progressing, fatal neurodegenerative disease caused by the misfolding of the cellular prion protein, PrP^C, encoded by the PRNP gene. Misfolded prion protein is acutely toxic to neurons, which can lead to the rapid development of dementia, difficulty walking and changes in gait, hallucinations, muscle stiffness, confusion, fatigue, difficulty speaking, and ultimately death. Most cases are sporadic or caused by inherited dominant mutations in PRNP, with an estimated 500 patients diagnosed per year in the US. The most common form of prion disease that affects humans is Creutzfeldt-Jakob disease. There are currently no approved or clinical-stage disease-modifying therapies for the prevention or treatment of prion disease.

About Sangamo Zinc Finger Transcriptional Regulators

Sangamo’s zinc finger transcriptional regulators, or ZF-TRs, recognize and bind to a specific DNA sequence within or near a particular gene, allowing expression of that target gene to be potentially regulated. ZF activators, or ZF-As, are created by attaching a zinc finger array to an activation domain with the aim of increasing the expression of a target gene relative to an untreated cell. ZF repressors, or ZF-Rs, are created by attaching a zinc finger array to a repression domain in order to down regulate or completely turn off a gene. ZF-Rs can also be designed to selectively repress expression of a mutant allele while allowing for the expression of the healthy allele. Sangamo is currently evaluating ZF-TRs in a variety of pre-clinical programs, including Nav1.7 for the potential treatment of chronic neuropathic pain and for prion disease.