PBGENE-RAG1

PBGENE-RAG1 is an investigational *ex vivo* gene editing therapy developed by Precision BioSciences for the treatment of RAG1-deficient Severe Combined Immunodeficiency (SCID). The therapy utilizes the proprietary ARCUS platform, which features an engineered meganuclease derived from the I-CreI homing endonuclease. PBGENE-RAG1 is designed to target a specific DNA sequence within the RAG1 gene in patient-derived hematopoietic stem cells (HSCs). By creating a precise double-strand break, the meganuclease facilitates the site-specific insertion of a functional RAG1 cDNA, thereby restoring the production of the RAG1 protein. RAG1 is essential for V(D)J recombination, a process required for the development of functional T and B lymphocytes. This approach aims to provide a durable cure for patients with SCID who lack a matched bone marrow donor by reconstituting a functional immune system.

Other names

meganuclease targeting RAG1RAG1 ARCUS nucleaseRAG-1 ARCUS nucleaseRAG 1 ARCUS nucleaseARCUS meganuclease targeting RAG1