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HSC-LNP Gene Writer
- Development stage
- Preclinical
- Lead developer
- Tessera Therapeutics
- Modality
- Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
HSC-LNP Gene Writer is a preclinical gene-editing therapeutic candidate developed by Tessera Therapeutics for the in vivo treatment of sickle cell disease (SCD). The platform utilizes an all-RNA composition consisting of Gene Writer mRNA and a template guide RNA, encapsulated within proprietary hematopoietic stem cell (HSC)-targeting lipid nanoparticles (LNPs). Unlike traditional ex vivo gene therapies that require bone marrow harvesting and transplantation, this approach enables direct genomic rewriting within long-term hematopoietic stem cells (LT-HSCs) in the bone marrow following intravenous administration. The system leverages target-primed reverse transcription (TPRT) to perform precise genetic modifications, such as correcting the E6V mutation in the hemoglobin subunit beta (HBB) gene. Preclinical studies in humanized mice and non-human primates have demonstrated high editing efficiency in LT-HSCs with stable multi-lineage persistence.
- Other names
- HSC-targeted Gene Writer LNPs
02
Targets
HBB (Hemoglobin subunit beta (HBB) gene locus)
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