The invention is an in vitro method that allows the recovery of F5 gene expression by the use of CRISPR/Cas9, a genetic modification tool of high precision.
The access to exploit this methodology will benefit the licensee the possibility to perform cellular and genetic therapy to treat and reverse the disease.
Factor 5 deficiency due to homozygous or compound heterozygous mutation (also known asOwren's disease orparahemophilia) has an estimated prevalence of one in one million people.It is a rare disease and may be genetic or acquired due to an inhibitor. There is no solution in the market for its treatment.
Current development status: It has been validated in cellular models and are now working in the validation of animal models with focus to reach clinical trials.
The Sant Pau Institute of Biomedical Research (IIB Sant Pau) is a research centre that holds 9 different entities grouped under one name for strengthening collaborative translational research and for bridging the gap between basic research and clinical practice so as to ultimately improve patient care.
The different entities are based on the Sant Pau Biomedical Campus, where other entities, such as the Barcelona Health Hub (BHH), are working together in the health sector. The most tangible benefits of this collaborative environment are the application of technology to health, and the scientific dissemination of these discoveries.
Members of the IIB Sant Pau:
- Fundació Institut de Recerca de l’Hospital de la Santa Creu i Sant Pau
- Fundació de Gestió Sanitària de l’Hospital de la Sant Creu i Sant Pau
- Fundació Privada de l’Hospital de la Santa Creu i Sant Pau
- Universitat Autònoma de Barcelona
- Fundació Puigvert
- Banc de Sang i Teixits
- Centre Cochrane Iberoamericà
- Equip d’Atenció Primària Sardenya
- Agència de Salut Pública de Barcelona
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