Therapeutic Innovation in Rare and Inherited CVD
Our goal is to create a human and technical resource that enables therapeutic innovation in rare and inherited cardiovascular conditions.
Vision
Approximately 1 in 17 people (3.5 million in the UK) will be affected by a rare disease during their lives and data suggest that the health burden of rare and inherited cardiovascular conditions (RICC) is substantial and underestimated.
UCL is a recognised leader in the development of therapies, devices, and diagnostics. Furthermore, UCL is a leader in cell and gene therapy, running 46% of all academic and 39% of all industry-sponsored UK clinical trials.
This theme will combine these resources with our unique RICC cohorts to accelerate therapeutic innovation, by creating a human and technical resource that enables therapeutic innovation in rare and inherited cardiovascular conditions (RICC).
UCL core clinical partners represent the largest clinical service in Europe, providing comprehensive evaluation and management for the full range of RICC. Patients with >350 individual rare diseases and half of the 60 nationally commissioned specialist services for rare diseases in England are based at UCL partner hospitals.
Clinical services for RICC include many national referral services and are managed in a network that includes St. Bartholomew’s Hospital & GOSH (among the largest clinical cohorts in the world for Cardiomyopathies, Channelopathies, Aortic Syndromes, CHD, Metabolic and Neurological Diseases, and Cardio-oncology), The Royal Free Hospital (national services for Lysosomal Storage Diseases, Pulmonary Hypertension, Vasculopathies, Amyloidosis, Connective Tissue Disorders) and UCLH (National Hospital for Neurological Disease, Charles Dent Metabolic Unit & The Haemostasis Research Unit).
This clinical resource is coupled with RICC research across UCL Faculties and Departments and has had major international impact in the last decade.
The theme will leverage UCL stratified patient cohorts and stored biomaterials to enable rapid recruitment into experimental and proof of principle studies of potential therapies in the preclinical phase and will generate validated outcome measures that can be used in early and late phase studies.