On October 6, 2021, Voyager Therapeutics and Pfizer entered into a license option agreement, which allows Pfizer to exercise options to license Voyager’s adeno-associated virus (AAV) capsids for gene therapies.

Voyager is a Massachusetts-based company developing AAV-based gene therapies.  The AAV capsids to be licensed under the agreement are generated from Voyager’s proprietary screening platform, TRACER^TM (Tropism Redirection of AAV by Cell-type-specific Expression of RNA), with which Voyager has identified capsids that can target cells and tissues with higher accuracy and at lower doses.  Voyager has shown that the AAV capsids have advantages in blood-brain-barrier penetration, cardiac muscle tropism, and transgene expression levels in target tissues compared to conventional AAV capsids.

Pfizer will use the licensed AAV capsids to develop, manufacture, and commercialize its own gene therapy pipelines to treat certain neurologic and cardiovascular diseases.  Recently, in its ongoing Duchenne muscular dystrophy gene therapy trial, Pfizer had to change its protocol to address a series of safety issues.  The deal with Voyager appears to be a part of Pfizer’s efforts to fine-tune its gene therapy approaches to improve the safety profiles.

“Our collaboration with Voyager will provide Pfizer with access to additional AAV capsids that may help further advance our industry-leading gene therapy portfolio,” said Dr. Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizer’s Rare Disease Research Unit. “We are impressed with Voyager’s results to date and are enthusiastic about the potential to utilize these novel capsids to help accelerate the development of new therapeutic options for patients living with certain neurologic and cardiovascular diseases.”

Under the agreement, Pfizer will be able to evaluate Voyager’s capsids selected for central nervous system and cardiac tropisms, and exercise options to license capsids for exclusive use in development of gene therapies with two undisclosed transgenes, which are different from those used/planned for Voyager’s pipelines.  Voyager will retain the rights to all licensed capsids for use with other transgenes and to all other applications of its TRACER^TM platform.

Pfizer will pay $30 million upfront and up to $20 million in exercise fees for two options exercisable within 12 months.  Voyager will be eligible to receive up to $580 million in development, regulatory, and commercial milestones, as well as mid- to high-single-digit tiered royalties based on net sales of the relevant Pfizer products.

Here are some reports relating to this deal for your reference: Voyager press release