March 15, 2023

The second patient recruited in the Recomb gene therapy trial

Significant progress has recently been made in the Recomb research project, which is focused on developing gene therapy for RAG1-SCID patients. The trial protocol has recently been approved in Barcelona, which is the second clinical site to gain approval. Moreover, the protocol is in an advanced stage of obtaining approval in Wroclaw (Poland) and London (UK) and under review in Ulm (Germany) and Rome (Italy). In Tel-Hashomer (Israel) the submission is in preparation.

Two patients treated with gene therapy

The trial has recently also successfully recruited the second patient. The first patient was diagnosed with RAG1-SCID immediately after birth because of positive family history and, in parallel, “confirmed” through the neonatal screening program in the Netherlands. Following successful stem collection, the product was infused in August 2021. To date, the patient has remained in good clinical condition, has not developed any therapy-related serious adverse reactions, and shows all signs of successful immune reconstitution with normal numbers of T and B lymphocytes for his age as well as successful vaccination responses.

The second patient was diagnosed with RAG1-SCID via newborn screening and recruited in December 2022. To date, two months after the treatment, the patient is in good condition while awaiting immune recovery.

Building on the positive experience so far, and with additional international clinical sites opening, Recomb hopes to recruit additional patients in the coming months.

As the leader of the work package for dissemination, IPOPI is very pleased to participate in this project to make a difference for patients with RAG1-SCID.

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