AbbView has announced it has bought the rights for development and commercialisation of RGX-314. This gene therapy product has been developed by Regenxbio, a US based company for the treatment of age related macular degeneration (AMD).
AMD is a severe retinopathic disease causing vision loss. RGX-314 aims to treat this in patients by using an AAV8 vector to deliver a monoclonal antibody fragment to interrupt the VEGF pathway and restore blood vessel formation in the eye. Patient numbers suffering from AMD are estimated to reach 288 million globally by 2040.
The results of clinical trials are expected in 2023. The huge investment in gene therapy shows the field moving towards treating the cause of the disease rather than just the symptoms. The need to understand the safety profile of these vectors is needed now more than ever to safely treat patients for these debilitating genetic diseases.