A next-gen capsid for gene therapies designed to treat central nervous system diseases
Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, and Viralgen, a contract development and manufacturing organisation (CDMO) specialising in recombinant adeno-associated virus (rAAV) gene therapies, have formed a strategic manufacturing collaboration to support gene therapy programs using Apertura’s TfR1 CapX, an AAV capsid that targets human Transferrin Receptor 1 (hTfR1) to cross the blood-brain barrier.
Under the collaboration, Apertura licensees can access the TfR1 CapX plasmid via Viralgen’s integrated AAV manufacturing platform by utilising its technical expertise. This collaboration aims to enable the scale-up process and ensure a seamless transition from research to clinical development.
Diego Garzón, chief business officer at Apertura, said, “We expect to see multiple Apertura partners advance programs using TfR1 CapX into the clinic this year and next, and this collaboration with Viralgen is designed to facilitate the advancement of novel therapies leveraging TfR1 CapX.”
TfR1 CapX is an intravenously (IV) dosed AAV capsid that can bind to human TfR1, cross the blood-brain barrier, and broadly transduce tissues in the CNS while administered at low doses. TfR1 CapX has demonstrated selectivity to the CNS compared to other tissues, with robust transduction of neurons and astrocytes across different brain regions.
Jimmy Vanhove, chief executive officer at Viralgen, said, “We believe having a well-characterised production framework in place enables gene therapy programs to progress more efficiently toward clinical development and supports the development of more effective and scalable gene therapies.”
Viralgen has scaled up TfR1 CapX manufacturing and is prepared to support research and clinical development programs. The AAV platform manufacturing process has been deployed across more than 1.500 batches.
Viralgen provides CMC services, including essential starting materials such as Master Cell Bank (MCB) creation and plasmid supply as part of its AAV manufacturing services. These capabilities are currently being used to generate MCBs and plasmids for the TfR1 CapX partnership.
Jorge Santiago-Ortiz, vice president of CMC and Regulatory Affairs at Apertura, said, “Through this collaboration, Apertura licensees can benefit from an efficient and fast supply of TfR1 CapX plasmid to support clinical production of investigational gene therapies using a scalable and established manufacturing process. The collaboration is structured to support clinical manufacturing planning and regulatory engagement as Apertura advances its gene therapy programs.”
