Gene Therapy Developer

Pharmaceutical R&D Developer
On October 6, 2021, Voyager Therapeutics announced a research and development collaboration agreement with Pfizer. Leveraging Voyager's novel adeno-associated virus (AAV) capsid development platform, the collaboration will help Pfizer develop two gene therapies for the treatment of neurological and cardiovascular diseases.
Voyager Therapeutics' TRACER technology platform is an RNA-based functional screening platform that enables rapid iterative evolution of AAV capsids, generating AAV capsids capable of efficiently transducing specific cells or tissues across multiple species, including non-human primates. Initial data demonstrate that one novel AAV capsid effectively crosses the blood-brain barrier. When administered intravenously in non-human primates, it increases transgene expression by over 1,000-fold across broad brain regions compared to the conventional AAV9 vector. Another AAV capsid efficiently transduces cardiac tissue while reducing transgene delivery to the dorsal root ganglia, thereby mitigating potential toxic side effects.
Image source: Voyager Therapeutics official website
Under the agreement, Pfizer will have the right to evaluate novel capsids from Voyager’s TRACER platform for delivering gene therapies to the central nervous system and heart, and to select these capsids for use in AAV gene therapies delivering two undisclosed transgenes. Voyager will receive a $30 million upfront payment. Additionally, Voyager will be eligible for up to $580 million in development, regulatory, and commercial milestone payments, as well as tiered royalties on net sales of the related products.
References:
[1] Voyager Therapeutics Announces License Option Agreement With Pfizer For Next-Generation TRACERᵀᴹ AAV Capsids To Enable Neurologic And Cardiovascular Gene Therapy Programs. Retrieved October 6, 2021, from https://ir.voyagertherapeutics.com/news-releases/news-release-details/voyager-therapeutics-announces-license-option-agreement-pfizer
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