RAG1-SCID
Recombinase 1-Activating Gene 1 Severe Combined Immunodeficiency
ClinicalActive
Key Facts
Indication
Recombinase 1-Activating Gene 1 Severe Combined Immunodeficiency
Phase
Clinical
Status
Active
Company
About Genewity
Genewity is a private, clinical-stage biotech focused on pioneering stem cell-based gene therapies for rare pediatric diseases. Its lead program targets RAG1-SCID, with early clinical results showing restored immune function. The company leverages a patient-centric, autologous ex vivo approach where a patient's own bone marrow stem cells are genetically modified and reinfused, aiming for durable, one-time cures. Based in the Leiden Bio Science Park, Genewity is building a pipeline of therapies for severe genetic disorders with high unmet need.
View full company profile