Sangamo Therapeutics, Inc, a genomic medicine company, and Voyager Therapeutics, Inc, announced the parties have entered into a definitive license agreement for a potential 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.
“Prion is a devastating, rapidly progressing and deadly disease, for which no treatment options currently exist,” said Sandy Macrae, Chief Executive Officer 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. Our agreement with Voyager highlights the importance of collaboration to address difficult indications with significant unmet medical need. I’m excited to combine Sangamo’s cutting-edge epigenetic regulation capabilities with the delivery abilities of Voyager’s capsid to potentially create the first ever meaningful therapy for prion disease.”
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 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.”
SOURCE: Businesswire