Our therapies use a patient’s own stem cells, genetically modified outside the body to by inserting a functional copy of the target 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’ cells are collected locally, shipped to specialized facilities for modification, and returned for infusion in a hospital near home. Building on this infrastructure, children can receive advanced gene therapy without extended travel, minimizing disruption for families while maintaining the highest standards of quality and care.
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By combining gene delivery with the regenerative potential of stem cells, our approach offers the possibility of a durable, one-time treatment, providing long-lasting benefit and addressing the disease at its root.
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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.
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Changing children’s lives

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 highlyencouraging, with children showing restored immune function and thriving development,giving them the opportunity to live healthy, normal lives. Building on thissuccess, Genewity plans to expand the approach to treat other geneticdisorders, offering the promise of durable, one-time therapies that address theroot cause of disease.
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