CureCN: ICGEB European Commission Research & Innovation project funded under Horizon 2020

CureCN Project funded under Horizon 2020

Andrés Muro, Group Leader, Mouse Molecular Genetics, ICGEB Trieste has been awarded funding together with 12 partners, comprising clinics, patient associations and management groups across Europe and the USA, to develop phase I/II gene therapy clinical trials to find new therapies for a rare genetic disease.

Crigler-Najjar (CN) Syndrome is an ultra-rare disease, affecting about 1 in 1,000,000 individuals at birth. Caused by the deficiency of a liver-specific uridine diphosphate (UDP) glucuronosyltransferase 1A1 (UGT1A1) resulting in the toxic, life-threatening accumulation of unconjugated bilirubin in all body tissues, especially in the brain.  Without effective treatment it causes irreversible neurological damage and eventually death by kernicterus (bilirubin-induced brain dysfunction). Although the molecular bases of CN syndrome are well known, there is currently no curative treatment for the disease, apart from liver transplantation, with all the limitations and risks this approach entails.

The 5-year CureCN project, which will commence on 1 January 2018, is coordinated by Federico Mingozzi, of the Liver and metabolic diseases programme, Genethon France.

The objective of the project is to develop a curative gene therapy for CN syndrome based on liver gene transfer with adeno-associated virus (AAV) vector expressing the UGT1A1 transgene. AAV vector-mediated gene therapy has shown promising results in preclinical animal models and, more recently, in humans. These results, together with the recent market approval of a gene therapy drug based on an AAV vector, established a novel treatment paradigm for monogenic disorders, which will now be applied to liver gene therapy for Crigler-Najjar Syndrome.

Andrés Muro welcomes the exciting prospect to work with clinical centres in France, Germany, Holland and Italy (Bergamo and Naples) and research groups in France, The Netherlands, the UK and the USA. ICGEB's involvement in the project consititutes a fine example of a model for developing gene therapy; establishing gene therapy clinical trials to create innovative, alternative therapies for liver disease treatment.


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