With its lead gene therapy programs stalled by a series of setbacks, Voyager Therapeutics has been talking for months about refocusing its business on internal technology that finds capsids—the protein shells that envelope a gene therapy and deliver it into a target cell. That strategy is starting to pay off, as Pfizer has inked a deal to tap into the Voyager technology.
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Pfizer is paying $30 million up front for the right to evaluate novel capsids selected by Voyager’s TRACER technology platform. Voyager contends this technology finds capsids that reach tissues current approaches can’t, delivering genetic cargo more safely. Pfizer is interested in finding capsids that could carry two undisclosed experimental gene therapies into central nervous system and cardiac tissue. Depending on what Voyager is able to find for its new partner, the biotech could earn up to $580 million in milestone payments, plus royalties from sales if Pfizer gene therapies reach the market.
The delivery method of choice for many gene therapies is adeno-associated virus. These engineered viruses serve as the vehicle that gets the therapy to a target cell. The capsid is the part of the virus that protects the genetic therapy along that journey, and it also delivers the genetic cargo into the cell upon reaching the destination. One of the challenges for gene therapies is that certain capsids are not well-suited for delivery to certain types of tissue. Safety is another problem, as these viruses can trigger a dangerous immune response.
Voyager’s TRACER technology is an RNA-based screening platform. It’s designed to quickly identify AAV capsids with desired properties, such as the ability to reach specific cells and transfer its genetic cargo into them. Delivery to central nervous system tissue has been a challenge for these therapies.
In May, during the annual meeting of the American Society of Gene and Cell Therapy, Voyager presented promising data from tests in monkeys. The results showed a TRACER-discovered AAV capsid, delivered by intravenous injection, led to expression of the transferred gene in the brain at levels more than 1,000 times higher than conventional AAV gene therapy. The week after the gene and cell therapy conference, Voyager announced its strategy shift to focus on TRACER, along with a C-suite shakeup that led to the departures of CEO Andre Turenne and Omar Khwaja, chief medical officer and head of research and development. Interim CEO Michael Higgins said in a prepared statement that the Pfizer deal highlights the potential of the Voyager technology.
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“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,” he said.
Voyager’s gene therapy prospects have been rocky over the past year. In August 2020, AbbVie ended a collaboration started in 2018 focused on developing gene therapies for two neurodegenerative diseases, Alzheimer’s and Parkinson’s. The partnership ended without a gene therapy for either disease reaching human testing. Last December, a Voyager Parkinson’s disease gene therapy candidate was placed under a clinical hold due to safety concerns. Neurocrine Biosciences, gave notice in February that it will end the alliance on that gene therapy. The Voyager pipeline includes a Huntington’s disease gene therapy candidate that was placed under a clinical hold last October; the FDA lifted that hold in April.
When Voyager announced its new focus on the TRACER, it said it the technology offered the potential for licensing deals and collaborations in neuroscience as well as other areas. Though the Pfizer alliance lends some validation to the technology, the capsid discovery space is getting crowded. Capsida Therapeutics emerged from stealth in May with $50 million in financing plus $90 from AbbVie, which is interested in the potential of the Thousand Oaks, California-based company find capsids that can reach the brain.
Central nervous system tissue delivery is also a focus of Affinia Therapeutics, which followed Capsida’s announcement with the unveiling of $110 million in Series B financing. Waltham, Massachusetts-based Affinia has a partnership with Vertex Pharmaceuticals, which is interested in the delivery of gene therapies to muscle tissue. Meanwhile, Dyno Therapeutics, which closed a $100 million Series A round in May, isn’t pursuing its own gene therapies. The Cambridge-based company instead uses its artificial intelligence technology to design capsids for other companies. Its disclosed partners include Roche, Sarepta Therapeutics, and Novartis.
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