On Friday May 21, Alastair Kent OBE, Chair of Congenica’s Patient Advocacy and Engagement Advisory Board and Dr Charles Steward, Congenica’s Patient Advocacy and Engagement Lead, attended the virtual Cambridge Rare Disease Network (CRDN) Companies Forum Patient Group and Industry Partnering event.

In this blog, Dr Steward talks about the event and how collaboration is driving progress for patient groups.


Cambridge Rare Disease Network logo-1The event brought industry and patient groups together to discuss possible areas of mutual collaboration. This was an invitation only event for CRDN Companies Forum Members, which included representation from Illumina, Abcam, Healx, Pfizer and Takeda amongst others.


Of the thousands of rare diseases currently identified, around 95% have no treatment, and most of the remaining 5% have limited treatment options. Development of treatments for rare diseases is often hindered by small market size, inadequate sources of information, and low awareness of the conditions. CRDN Companies Forum aims to tackle these issues and contribute to the development of new treatments through creation of the right networks.


The programme featured five-minute pitches by 10 rare disease patient groups that discussed what they had achieved so far and where they need support to help them with their journey to developing new therapies for their disorders. Following this, we had a series of speed-dating style partnering sessions, where representatives from each patient group and each company met to discuss potential common interests and areas for collaboration. We finished the day by having a networking event of informal conversation.


It was great to meet some very inspirational patient advocates, many of whom are world-leading authorities in their disease area and learn more about them and the challenges faced by those affected as well as some of the research that is being carried out. Congenica is already working with some of the se advocacy groups – Allison Watson from Ring20 research and support, Nuala Summerfield from Schinzel-Giedion Syndrome Foundation and Sue Routledge from Pitt Hopkins UK. Other groups we spoke with included Dravet Syndrome UK and Timothy Syndrome Alliance, both of whom we will be promoting by hosting articles by them for our PAE work.


It is incredible to see how much progress the patient groups have all made to understand their disease and drive research and we look forward to working with a few of them closely.


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