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GPH201
- Development stage
- Discontinued
- Lead developer
- MARAbio Systems
- Modality
- Gene Silencing → Gene Therapies, Bone Marrow HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, Peripheral Blood HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
GPH201 is an ex vivo hematopoietic stem cell gene therapy being developed by Graphite Bio for X‑linked severe combined immunodeficiency (XSCID) caused by IL2RG deficiency. It uses Graphite’s targeted gene integration platform based on homology‑directed repair and CRISPR-Cas9 to replace the entire defective IL2RG gene in patient-derived hematopoietic stem cells, with the goal of restoring production of functional common γ-chain and reconstituting adaptive immune function. The program is in IND‑enabling/preclinical development as a potential one‑time curative alternative to existing transplant- or vector-based approaches for XSCID.[2][5][7][11]
02
Targets
IL2RG (Interleukin-2 receptor gamma subunit)
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