October 20, 2025
RECOMB Project Final Report Highlights Advances in Gene Therapy for RAG1-SCID
The RECOMB project, a European research consortium funded by the Horizon 2020 programme of which IPOPI is a leading partner, has concluded after seven years of work to develop a gene therapy for RAG1-SCID, the most common form of severe combined immunodeficiency (SCID).
The project focused on correcting patients’ own blood-forming stem cells, which are then transplanted back, reducing the need for families to travel abroad for treatment. Before starting the clinical trial to treat patients, the safety, efficacy, and stability of the technology were demonstrated in preclinical models. Once confirmed, the trial opened in the Netherlands, Spain, Poland, the United Kingdom and Italy. In addition to these Recomb clinical centres, two non-Recomb collaborating centres were added from Turkey and Australia to empower recruitment of study patients. To date, five patients have been treated and remain clinically stable, with preliminary results showing the therapy to be both safe and effective.
The RECOMB 2025 Final Report summarises the project outcomes and outlines future perspectives for gene therapy in RAG1-SCID, offering new hope to affected families worldwide.
Although the RECOMB project has officially ended, the consortium remains dedicated to ongoing patient follow-ups and recruiting new patients as the therapy moves through the complex process of market approval.
