Voyager Therapeutics’ Novel AAV Capsid Tech for Gene Therapy Attracts Pfizer’s Investment

On October 6th, Cambridge, MA-based Voyager Therapeutics announced an agreement through which Pfizer can exercise options to license its novel, next-generation TRACER AAV capsids to enable neurologic and cardiovascular therapy programs.

Voyager’s RNA-driven TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) screening technology was invented to advance gene therapies for life-changing treatments.

As per agreement terms, Voyager receives $30 million upfront with the potential to exercise fees and milestone payments that add up to $600 million besides product sales-based royalties. Pfizer will have the right to evaluate novel capsids selected for the central nervous system and cardiac tropisms from Voyager’s TRACER platform and to integrate the AAV gene therapies within its two undisclosed transgenes.

“This transaction highlights the potential of our TRACER platform to identify novel AAV capsids that target desired cells and tissues with greater specificity at lower doses and with fewer off-target risks than conventional AAV serotypes,” said Michael Higgins, Interim CEO of Voyager.

“We believe that our TRACER platform has the ability to produce not only enhanced blood-brain-barrier penetrant capsids but also novel capsids with enhanced tropisms across a diversity of tissues and cell types, offering promise to unlock the fullest potential of gene therapies for a wide array of diseases with unmet medical need.”

“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 Seng Cheng, Ph.D., 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.”

TRACER Platform

AAV capsids from Voyager’s TRACER platform showed promising blood-brain-barrier penetration, cardiac muscle tropism, and transgene expression in target tissues compared to other AAV capsids in non-human primates (NHPs).

Compared to conventional AAV capsids, one Voyager AAV capsid candidate demonstrated more than 1,000-fold increased transgene expression in a wide array of brain regions when it was administered intravenously in NHPs. Results presented significantly fortifying cardiac muscle transduction and dorsal root ganglia de-targeting. Furthermore, a handful of proprietary AAV capsids are currently in screening with its TRACER platform to target various diseases in multiple tissues and cell types for gene therapy treatments.

Author

Judy Ya-Hsuan Lin

Success is the ability to go from one failure to another with no loss of enthusiasm.