Supported by our partnership with University College London (UCL) and leading contract development and manufacturing organisations (CDMOs), our approach leverages well-characterised AAV capsids, optimised vector constructs and novel routes of administration to create highly differentiated programs.
We are tackling neurological and metabolic diseases, each with clear and translatable biology and high unmet medical needs. All of our programs have already demonstrated compelling preclinical efficacy and we are now working relentlessly to bring them to patients.
Our academic founders combine unique and world-leading expertise spanning discovery, translation and clinical insights. In addition, we are building a team of industry leaders and subject matter experts with extensive experience in developing and registering gene therapies and rare disease treatments.
