SUPPORTING INDIVIDUALS, FAMILIES AND RESEARCHERS
IN THE CYSTINOSIS COMMUNITY

Gene Therapy Trials Update

If you are following the gene therapy trials in the USA you will have seen that the 6th and final patient was dosed at the end of October. AVROBIO, the company that is developing the technology, indicate that they are hoping to start their own trials in 2023 and they have also said that they are looking for sites in Europe and other countries to extend their trials. While the UK hasn’t specifically been mentioned there are some clues in other announcements. Manchester Clinical Research Facility (MCRF), Manchester University NHS Foundation Trust (MFT) and the University of Manchester (UoM) have created a strategic partnership with AVROBIO to trial a first-in-person gene therapy treatment for Hunter Syndrome using lentiviral vector-modified stem cells at the MCRF at Royal Manchester Children’s Hospital (RMCH). THis was approved in September. On October 19 AVROBIO announced that they had been granted an Innovation Passport for their gene therapy for Gaucher disease (another lysosomal storage disease) under the Innovative Licensing and Access Pathway (ILAP). The Innovation Passport is the first step in the ILAP process, triggering the MHRA and its partner agencies, including the National Institute for Health and Care Excellence (NICE), the Scottish Medicines Consortium (SMC) and National Health Service (NHS) England, to chart a roadmap for regulatory and development milestones with the goal of early patient access in the U.K. On November 8 AVROBIO announced they had submitted a meeting request to the MHRA to discuss a company-sponsored clinical trial for AVR-RD-04 (cystinosis gene therapy) and expect a scientific advice meeting with MHRA in the first quarter of 2023. We will of course let you know as soon as we hear any further information.

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