Drug intelligence / Profile preview

GNTI-122

Development stage
Phase 1
Lead developer
GentiBio
Modality
Cell Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration
Intravenous
01

Overview

GNTI-122 is an autologous engineered regulatory T cell (Treg) therapy developed by GentiBio for the treatment of type 1 diabetes (T1D)[2][3][6]. This cell therapy product is derived from a patient’s own CD4+ T cells and engineered to stably express FOXP3, a key transcription factor for Treg function. It incorporates a pancreatic islet-specific T cell receptor (TCR) to direct the cells to the pancreas and draining lymph nodes, as well as a chemically inducible signaling complex (CISC) that enables selective IL-2 signaling in response to low-dose rapamycin[1][5][6]. The design aims to enhance tissue specificity, stability, and functional support of the infused Tregs. Mechanistically, GNTI-122 suppresses pathogenic effector T cells through both direct antigen-specific suppression and bystander suppression mechanisms. Preclinical studies have shown that this approach can reduce inflammation in pancreatic tissue and protect insulin-producing beta cells from autoimmune destruction[5][6]. The primary indication under development is type 1 diabetes.

Other names
Engineered regulatory T cell therapyEngTreg cell therapy
02

Targets

RCISC (Synthetic interleukin-2 receptor-based chemically inducible signaling complex)FOXP3 (Forkhead box protein P3)Islet-pMHC (Islet antigen–peptide–major histocompatibility complex)

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