Voyager Therapeutics Licenses Next-Generation AAV Capsid to Pfizer for Rare Neurologic Disease Target

Voyager Therapeutics said that Pfizer has exercised its option to license a novel capsid generated from Voyager’s TRACER capsid discovery platform to help enable a potential gene therapy program against an undisclosed rare neurologic disease target.

Photo: Alfred Sandrock, CEO of Voyager

Voyager’s TRACER capsid discovery platform is an RNA-based screening platform that enables rapid discovery of AAV capsids with robust penetration of the blood brain barrier and enhanced central nervous system tropism in multiple species, including non-human primates. TRACER generated capsids have demonstrated superior and widespread gene expression in the CNS compared to conventional AAV capsids as well as cell- and tissue-specific transduction, including to areas of the brain that have been traditionally difficult to reach.

Under the terms of the license option agreement, originally announced in October 2021, Voyager previously received a $30 million upfront payment and is entitled to receive a $10 million option exercise payment. Voyager is eligible to receive potential future development, regulatory, and commercialization milestone payments of up to $115 million, sales milestones of up to $175 million, and mid- to high-single-digit tiered royalties in connection with Pfizer’s use of its capsid in development and commercialization of a gene therapy for a rare neurologic target.

The target under agreement with Pfizer is distinct from those utilized in Voyager’s internal pipeline programs. Pfizer has elected not to exercise its option to license a capsid for the cardiac target under the original agreement, and all capsid rights for that target are returned to Voyager. Voyager retains global rights to all licensed capsids for use with other transgenes and to all other applications of its TRACER technology.

“Pfizer’s decision to exercise this option reflects the significant research progress our scientific teams have made and further validates the potential of our TRACER platform to help enable gene therapy for neurological diseases,” said Alfred Sandrock, CEO of Voyager. “Collaborations, such as this Pfizer partnership, are a pillar of our two-pronged strategy to leverage our next-generation capsids both to help enable our partners’ gene therapy programs and to advance Voyager’s internal pipeline of highly differentiated candidates for GBA1 Parkinson’s disease, SOD1 ALS, and other neurological diseases.”

Author: Rare Daily Staff