The viral vectors in our programs have been designed with a deep understanding of the biology of our target diseases. We have chosen well-characterised adeno-associated virus (AAV) capsids that have demonstrated safety and efficacy in clinical and/or commercial settings to accelerate clinical translation, optimise manufacturability and maximise regulatory success. We have combined them with innovative cassettes constructed for optimal therapeutic effect.
For our neurological programs – BGT-DTDS, BGT-NPC and BGT-INAD – we are leveraging direct administration to the CNS delivery approaches, enabling low treatment doses, improved safety, targeted efficacy and reduced impact of pre-existing immunity to the capsids, when present.
UCL (University College London) is a world-leading academic centre for the discovery and clinical development of gene therapies, with a network of affiliated hospitals in London providing some of the best clinical care in the world to patients with rare genetic diseases. As a spin-out of UCL, we have established a strong partnership with the institution, which spans the completion of our non-clinical research activities to the implementation of our clinical trials. UCL’s unparalleled capabilities and access to patients, through its affiliated hospitals, will help us accelerate the development of our programs.
To minimise requirements for costly and lengthy Chemistry, Manufacturing, and Control (CMC) comparability studies that have plagued the gene therapy field to date, we have decided to partner from the outset with carefully chosen contract development and manufacturing organisations (CDMOs) with a track record in production of commercial or near-commercial-grade AAV gene therapies. Combined with our meticulous capsid selection, we are deploying a manufacturing strategy enabling a fast progression to applications for regulatory approvals.
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