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Graphite Bio non-genotoxic conditioning program
- Development stage
- Preclinical
- Lead developer
- MARAbio Systems
- Modality
- Monoclonal Antibodies → Antibody-Based Therapeutics
- Administration
- Intravenous
01
Overview
Graphite Bio's non-genotoxic conditioning (NGTC) program is a discovery-stage platform designed to replace traditional, DNA-damaging myeloablative conditioning (such as busulfan or radiation) used in hematopoietic stem cell (HSC) transplantation. The technology utilizes a "best-in-class" antibody-based approach to selectively target and clear a niche in the bone marrow for the engraftment of gene-edited autologous or allogeneic hematopoietic stem and progenitor cells (HSPCs). By avoiding genotoxic agents, this regimen aims to reduce treatment-related toxicity, eliminate the need for prolonged hospitalization, and broaden patient eligibility for gene therapies, potentially enabling outpatient administration. The specific molecular targets of the program remain undisclosed, but the technology is intended to support Graphite Bio's broader pipeline of CRISPR-based gene therapies for genetic diseases.
- Other names
- Non-genotoxic conditioningHSC-targeted conditioning approaches
02
Targets
KIT (c-KIT proto-oncogene receptor tyrosine kinase)PTPRC (Receptor-type tyrosine-protein phosphatase C)
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