Voyager Therapeutics’ shift in strategy to gene therapy technology leads to alliance with Pfizer

With its major gene therapy programs blocked by a series of setbacks, Voyager Therapeutics has been talking for months about refocusing its business on in-house technology that finds the capsids, the protein envelopes that envelop gene therapy and deliver it into a target cell. This strategy is starting to bear fruit, as Pfizer has signed an agreement to exploit Voyager technology.

Pfizer is paying $ 30 million upfront for the right to evaluate new capsids selected by Voyager’s TRACER technology platform. Voyager argues that this technology finds capsids that reach tissues that current approaches cannot reach, providing genetic cargo in a more secure manner. Pfizer is interested in the discovery of capsids that could transport two undisclosed experimental gene therapies in the central nervous system and heart tissue. Depending on what Voyager is able to find for its new partner, biotech could earn up to $ 580 million in milestone payments, plus royalties on sales if Pfizer gene therapies hit the market.

The administration method of choice for many gene therapies is adeno-associated virus. These modified viruses serve as the vehicle that delivers therapy to a target cell. The capsid is the part of the virus that protects gene therapy throughout this journey, and it also delivers the genetic cargo into the cell when it reaches the destination. One of the challenges of gene therapy is that some capsids are not well suited for delivery to certain types of tissue. Safety is another issue, as these viruses can trigger a dangerous immune response.

Voyager’s TRACER technology is an RNA-based screening platform. It is designed to quickly identify AAV capsids with desired properties, such as the ability to reach specific cells and transfer its genetic makeup to them. Delivery to central nervous system tissue has been a challenge for these therapies.

In May, at the American Society of Gene and Cell Therapy annual meeting, Voyager presented promising data from tests on monkeys. The results showed that an AAV capsid discovered by TRACER, administered by intravenous injection, led to the expression of the transferred gene in the brain at levels more than 1000 times higher than those of conventional AAV gene therapy. The week after the gene and cell therapy conference, Voyager announced its change of strategy to focus on TRACER, as well as a reshuffle of the C suite that led to the departure of CEO Andre Turenne and physician Omar Khwaja. -Head and responsible for research and development. Interim CEO Michael Higgins said in a prepared statement that the Pfizer deal highlights the potential of Voyager technology.

“We believe that our TRACER platform has the ability to produce not only improved blood-brain barrier penetrating capsids, but also new capsids with improved tropisms in a variety of tissues and cell types, offering the promise to unleash the full potential of gene therapies for a wide range of diseases with unmet medical needs, ”he said.

The outlook for Voyager’s gene therapy has been difficult over the past year. In August 2020, AbbVie ended a collaboration that began in 2018 focused on the development of gene therapies for two neurodegenerative diseases, Alzheimer’s disease and Parkinson’s disease. The partnership ended without gene therapy for either disease being tested in humans. Last December, a gene therapy candidate for Parkinson’s disease Voyager was placed on clinical hold due to safety concerns. Neurocrine Biosciences, announced in February that it would end the alliance on this gene therapy. The Voyager pipeline includes a Huntington’s disease gene therapy candidate who was placed on clinical hold last October; the FDA lifted this wait in April.

When Voyager announced his new focus on TRACER, he said the technology offered the potential for licensing agreements and collaborations in neuroscience as well as other areas. Although the Pfizer alliance validates the technology, the capsid discovery space is crowded. Capsida Therapeutics emerged from stealth in May with $ 50 million plus $ 90 in funding from AbbVie, which is looking at the potential of the Thousand Oaks, Calif., Based company to find capsids that can reach the brain.

The distribution of central nervous system tissue is also a goal of Affinia Therapeutics, which followed Capsida’s announcement with the unveiling of a $ 110 million Series B funding. Affinia, based in Waltham, Mass., Has a partnership with Vertex Pharmaceuticals, which is interested in delivering gene therapy to muscle tissue. Meanwhile, Dyno Therapeutics, which closed a $ 100 million Series A round in May, is not pursuing its own gene therapies. The Cambridge-based company is instead using its artificial intelligence technology to design capsids for other companies. Its disclosed partners include Roche, Sarepta Therapeutics and Novartis.

Photo: iLexx, Getty Images

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