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SJEFS-S19
- Development stage
- Preclinical
- Lead developer
- St. Jude Children's Research Hospital
- Modality
- Cell Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
SJEFS-S19 is a lentiviral vector-based gene therapy candidate developed by St. Jude Children's Research Hospital for the treatment of Diamond-Blackfan Anemia (DBA). DBA is a congenital ribosomopathy primarily caused by heterozygous loss-of-function mutations in the *RPS19* gene, which encodes a key component of the 40S ribosomal subunit. SJEFS-S19 utilizes a third-generation self-inactivating (SIN) lentiviral vector to deliver a codon-optimized *RPS19* transgene into patient-derived CD34+ hematopoietic stem and progenitor cells (HSPCs). By restoring functional RPS19 expression, the therapy aims to correct pre-rRNA processing defects, alleviate p53-dependent cellular stress, and rescue erythroid progenitor survival. This ex vivo approach is intended to provide a curative treatment for DBA while avoiding the risks of graft-versus-host disease associated with allogeneic bone marrow transplantation.
- Other names
- RPS19 gene therapyRPS-19 gene therapyRPS 19 gene therapy
02
Targets
MIF (Oxidized macrophage migration inhibitory factor)40S (Small ribosomal subunit protein uS10)DNA
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