October 13, 2017

IPOPI to contribute to development of gene therapy for recombinant SCID

IPOPI has joined an international consortium led by Prof Frank Staal, from Leiden University Medical Center, that aims at developing and testing a gene therapy for children affected by a very specific type of severe combined immunodeficiency (SCID): Recombinase Activating Genes (RAG) deficient SCID.

The project has the very ambitious goal of developing a major treatment option and possibly the first treatment of choice for over 70% of children suffering from SCID. The project, also known as RECOMB, has been recently approved by the European Commission and will receive funding from the European research budget to develop this ground-breaking therapy.

IPOPI will be mainly contributing to the project by sharing the activities to a wide range of stakeholders and by facilitating the use of the results from RECOMB.

The studies are undertaken by world renown clinical and basic scientists in the field of gene therapy and stem cell transplantation with a proven track record from many European countries (such as the Netherlands, France, the UK, Italy, Germany, Poland or Spain) and a research infrastructure from Israel.