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Cambridge, Mass.-based Voyager Therapeutics entered a deal with Pfizer, allowing the latter to exercise options to license novel capsids for gene therapies. Specifically, the deal enables Pfizer to license novel capsids created by Voyager’s RNA-driven TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) screening technology.

Pfizer will pay $30 million upfront and $20 million in exercise fees for a pair of options the company exercises within 12 months. Also, there’s a biobucks component, with Voyager eligible for up to $580 million in development, regulatory and commercial milestones tied to licenses for two undisclosed Pfizer transgenes with a Voyager licensed capsid. Voyager is also up for mid- to high-single-digit tiered royalties.

Adeno-associated virus (AAV) capsids from Voyager’s TRACER technology platform have shown superior blood-brain-barrier penetration, improved cardiac muscle tropism (turning all or part of an organism in a particular direction), and increased transgene expression in target tissues. In other words, the capsids appear to target cells and tissues more accurately and at lower doses. There also appear to be fewer off-target problems than with conventional AAVs.

Pfizer is in a position where it needs to fine-tune its gene therapy approaches. In late September, it announced that it narrowed the trial protocols after three serious adverse events in an ongoing trial of its gene therapy for Duchenne muscular dystrophy (DMD). Pfizer reported three cases of muscle weakness, two involving myocarditis, or inflammation of the heart, and the External Data Monitoring Committee determined they were associated with the therapy, fordadistrogene movaparvovec, as well as specific mutations in the dystrophin gene.

“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 chief executive officer 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.”

For its part, the deal is good news for Voyager. In August 2020, AbbVie severed an agreement it had with Voyager for Alzheimer’s disease. They had inked a tau and alpha-synuclein vectorized antibody collaboration in August 2018. The focus was on Alzheimer’s patients and Parkinson’s disease, as well as another disease called synucleinopathies that is characterized by the abnormal accumulation of misfolded alpha-synuclein protein. The deal had been valued at more than $1.2 billion.

Seng Cheng, senior vice president and chief scientific officer of Pfizer’s Rare Disease Research Unit, stated, “Our collaboration with Voyager will provide Pfizer with access to additional AAV capsids that may help further advance our industry-leading gene therapy portfolio. 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.”

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