RARE Daily

Sangamo and Voyager Enter License Agreement for Epigenetic Regulation Treatment of Prion Disease

June 29, 2023

Rare Daily Staff

Sangamo Therapeutics and Voyager Therapeutics have entered into a definitive license agreement for a potential treatment of prion disease.

Prion disease is a rapidly progressing, fatal neurodegenerative disease caused by the misfolding of the cellular prion protein, PrPC, 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 United States. 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.

Using its proprietary epigenetic regulation platform, Sangamo has developed zinc finger transcriptional regulators (ZF-TRs) which it believes can specifically and potently block expression of the prion protein, the pathogenic driver of prion disease. Sangamo’s ZF-TRs have been shown in animal models to significantly reduce expression of the prion protein in the brain, extend life span and limit formation of toxic prion aggregates.

Under the agreement, Voyager has provided Sangamo with access to a capsid derived from Voyager’s proprietary TRACER platform. This capsid has been shown to effectively cross the blood-brain barrier when delivered intravenously in animal models.

“Prion is a devastating, rapidly progressing and deadly disease, for which no treatment options currently exist,” said Sandy Macrae, CEO at Sangamo. “While evaluating delivery mechanisms for our zinc finger transcriptional regulators, we were pleased to identify Voyager’s capsid as being well suited to potentially achieve the specific central nervous system coverage required for this indication today.”

Under the terms of the agreement, Sangamo has received a non-exclusive license to combine a Voyager TRACER capsid with Sangamo’s ZF-TRs designed to treat prion disease. Sangamo is solely responsible for the research, development, manufacture, and commercialization of any product candidates using the Voyager capsid. Voyager is eligible to earn certain license fees, royalties on potential commercial sales of any products using Voyager’s capsid, and, in the event the prion program is out licensed by Sangamo, a portion of all licensing revenues received with respect to this program. Sangamo expects to submit a potential IND for a product candidate treating prion disease in 2025.

“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 Sandrock, CEO 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.”

Photo: Sandy Macrae, CEO at Sangamo


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