ProQR Usher clinical development programme

Posted on: Wednesday, April 18th, 2018



ProQR is a young biotech firm based in the Netherlands that is in the early stages of planning a number of clinical trials targeting vision loss in Usher 2A patients (due to mutations in Exon 13 of Ush2A gene).

Their approach uses RNA oligonucleotide therapy to make changes to the RNA of these patients, with the aim of restoring some of the vision that has been lost. This type of therapy has some advantages over gene therapy, as it involves a less invasive procedure and is reversible, therefore does not preclude the patient from gene therapy in future. If this treatment is demonstrated to be safe and effective in trials for Usher 2A, the learnings could be used in the development of treatments targeting other mutations which lead to RP.

ProQR held a meeting to gather patient views on their plans for the trial, and will use the insights gathered to help inform trial design and also tailor their communication with participants and the wider RP/Usher community. ProQR will provide further information to the RP/Usher community (including information about participating sites for the trial) when more detail is known.

ProQR recently hosted an international group of Usher syndrome patient representatives from different organisations in the USA, Canada, UK, Ireland and the Netherlands. The objectives of the advisory meeting were to gather patient insights on ProQR’s QR-421a clinical development programme, offer a platform for networking, exchange experience and share learnings between participants. Several groups presented selected initiatives highlighting the impressive patient-led work being done internationally to help further understanding and support of the Usher community. During the lively and interactive discussions, participants had the opportunity to ask questions and provide feedback on the QR-421a development programme for patients with RP due to mutations in Exon 13 of the USH2A gene that is expected to start before the end of year. The importance of good communication between study staff and study patients as well as the order of study tests were discussed. Some patient educational and informational needs were identified and the group brainstormed on ways these could be addressed. The information gathered from the first QR-421a study will be applied to future studies and other potential development programs. ProQR reiterated its ongoing commitment to keep the community informed of its progress and to publish results of future studies regardless of the outcome. It was acknowledged that it is still early days and there is a lot to learn.

There remains a high unmet need for all types of Usher Syndrome but the group felt that as research continues to advance there is reason for hope.