Past clinical trials have suggested a favourable safety profile for AAV-based gene therapies. While early signs of efficacy have been very encouraging, it has become apparent that improved and human adapted AAV vectors are required in order to fully replicate the remarkable levels of efficacy observed in animal models.
Therapeutic AAV vectors consist of two main components: the AAV capsid and the expression cassette. The AAV capsid is responsible for directing the vector into the correct cells and the expression cassette guides tissue-specific synthesis of the therapeutic protein.
Our proprietary AAV vector technology, using human-adapted, novel capsids and highly optimized expression cassettes, is designed to deliver a functional copy of a therapeutic gene into the human liver to offer durable secretion of therapeutic enzymes into the patient’s blood.
We are focused on severe indications with high unmet-need that can be treated using enzymes secreted into the blood by the liver. This approach maximises the potential benefit that can be derived from a gene therapy since improvements in either capsid or expression cassette can synergistically contribute towards enhanced clinical benefit. It also capitalises on the significant experience of treating patients with enzyme replacement therapies.
Treatment of bleeding disorders is particular well suited for our platform. However, several hundreds of systemic and liver associated disorders exists that may be addressable using this technology in the future.