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Immunostem

Development stage: Unknown
Lead developer: Altheia Science
Modality: Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration: Intravenous

Overview

Immunostem is an autologous hematopoietic stem and progenitor cell (HSPC) therapy genetically modified to express human programmed death-ligand 1 (PD-L1), currently under development by Altheia Science for the treatment of recent-onset type 1 diabetes mellitus (T1D). The therapy involves the ex vivo transduction of a patient's own CD34+ HSPCs with a lentiviral vector encoding PD-L1. Once re-infused into the patient, these engineered cells and their progeny are intended to migrate to the pancreas and lymphoid organs to restore immune tolerance by activating the PD-1 pathway on autoreactive T cells. This approach aims to halt the autoimmune destruction of insulin-producing beta cells and preserve residual pancreatic function in newly diagnosed patients.

Brand names: Immunostem
Other names: LVV-hPD-L1–modified autologous HSPCs

Targets

PDCD1 (Programmed cell death protein 1 receptor)CD274 (Programmed cell death protein 1 ligand 1)

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