Apertura Gene Therapy and Viralgen, a contract development and manufacturing organization based in San Sebastián, Spain, have entered a manufacturing collaboration centered on TfR1 CapX, a next-generation AAV capsid engineered to cross the blood-brain barrier for CNS gene therapy delivery (announcement). New York-based Apertura developed the gene therapy capsid at the Broad Institute of MIT and Harvard. Viralgen, a subsidiary of AskBio within the Bayer AG group, will provide GMP manufacturing services to support Apertura's licensees as they advance programs toward clinical trials.
Financial terms of the collaboration were not disclosed. The arrangement appears structured as a manufacturing services partnership rather than a traditional licensing deal with upfront payments, milestones, or royalties. Viralgen will supply plasmid production, Master Cell Bank creation, process optimization, fill-finish, and quality control testing for TfR1 CapX-based programs. No equity component was described.
Deal context
TfR1 CapX is an intravenously administered AAV capsid that binds human transferrin receptor 1, exploiting receptor-mediated transcytosis to shuttle gene therapy payloads from the bloodstream into the brain and spinal cord. This mechanism addresses a central limitation of conventional AAV vectors such as AAV9, which achieve poor and inconsistent CNS penetration when dosed systemically and often require invasive intrathecal injection. Apertura describes TfR1 CapX as a second-generation capsid with improved CNS delivery compared to the first-generation BI-hTFR1 vector published by the Deverman Lab. Preclinical testing has shown selectivity for CNS tissue over peripheral organs, with transduction of neurons and astrocytes across multiple brain regions. All programs using the capsid remain preclinical. Apertura has stated that multiple partner programs are expected to enter clinical trials in 2026 and 2027, though no IND filings or clinical trial registrations have been disclosed.