July 7 , 2022

Recomb’s annual meeting confirms promising results for gene therapy for RAG1-SCID

IPOPI’s Johan Prevot and Julia Nordin recently had the pleasure of attending the annual Recomb meeting in Leiden, The Netherlands, on June 14-15. Recomb is a European research consortium developing promising gene therapy for recombination-deficient severe combined immunodeficiency (RAG1-SCID). This meeting is an annual opportunity for all Recomb partners, including IPOPI, to present progress from the past year and to discuss the next steps for the consortium.

The first patient enrolled in the clinical trial
This year’s annual meeting was special, as Professor Frank Staal and Professor Arjan Lankester, Recomb co-ordinators from Leiden University Medical Center (LUMC), announced the successful treatment of the first patient with this new therapy. The patient was diagnosed with RAG1-SCID immediately after birth through positive family history and confirmed through the national SCID neonatal screening programme. The patient was treated in August 2021 in LUMC, experienced an uneventful clinical course and now shows clear signs of T and B cell reconstitution. The news was celebrated by the other centres, several of which are waiting for their own regulatory approval to recruit patients for this trial.

As leaders of the work package for dissemination and exploitation, IPOPI presented the work that the team has been focusing on in recent months. As an example, this included the publication in Health Europa Quarterly and recent efforts to promote Recomb during the IPIC congress in April.

This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement no. 755170 (RECOMB).