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AAT targeted gene insertion program
- Development stage
- Preclinical
- Lead developer
- MARAbio Systems
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Stem Cell Therapies → Cell Therapies, Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
Graphite Bio's AAT targeted gene insertion program is an experimental ex vivo gene-edited cell therapy designed for the treatment of Alpha-1 antitrypsin (AAT) deficiency. The therapy utilizes CRISPR-based precision gene editing to perform targeted insertion of a functional SERPINA1 (AAT) coding sequence into autologous hematopoietic stem and progenitor cells (HSPCs). Once these edited cells are transplanted back into the patient following a conditioning regimen, they are intended to engraft in the bone marrow and differentiate into blood lineages that serve as a permanent, systemic 'bio-factory' for the AAT protein. This approach aims to provide a one-time curative treatment by restoring protective levels of circulating AAT to address both lung and liver manifestations of the disease.
- Other names
- Therapeutic protein production program (alpha-1 antitrypsin)SERPINA1 gene editing programSERPINA-1 gene editing programSERPINA 1 gene editing programAAT targeted gene insertion program
02
Targets
SERPINA1 (Alpha-1-antitrypsin)
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