Our therapies use a patient’s own bone marrow stem cells that are genetically modified outside the body to by inserting a functional copy of the defect gene. Once returned and infused into the patient, these cells repopulate the bone marrow and restore the function of the corrected gene, addressing the root cause of the disease.

The process leverages the well-established logistics of stem cell transplantation. Patients’ bone marrow cells are collected locally, shipped to specialized facilities for the gene insertion, and returned for infusion in a hospital near home. Building on this infrastructure, children can receive this therapy in a local hospital. This way we can minimize disruption for families while maintaining the highest standards of quality and care.

The correction of genes in bonw marrow stem cells offers the possibility of a durable, one-time treatment, providing long-lasting benefit and addressing the disease at its root.

Key advantages of our approach
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Targeted: Focused on the underlying genetic defect.
Durable: One-time therapy with potential for life-long benefit.
Patient-centered: Cells travel, not the patient.
Versatile technology: Applicable to multiple severe children’s diseases.

Our clinical-stage programis already demonstrating the power of its platform technology. Early results inpatients with Recombinase 1-Activating Gene 1 (RAG1)-SCID have been highly encouraging, with children showing restored immune function and thriving development, giving them the opportunity to live healthy, normal lives. Building on this success, Genewity plans to expand the approach to treat other genetic disorders, offering the promise of durable, one-time therapies that address the root cause of disease.