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Aptamer-Engineered Human Islets

Development stage
Preclinical
Lead developer
Virginia Commonwealth University
Modality
DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, Cell Therapies
Administration
Parenteral
01

Overview

Aptamer-Engineered Human Islets represent a novel cell therapy approach aimed at improving the success of islet transplantation, particularly in the context of Type 1 Diabetes. This therapeutic strategy involves modifying human pancreatic islets, which are clusters of cells responsible for insulin production, with specific aptamers. These aptamers are designed to target and block key pro-inflammatory mediators such as Interleukin 1 beta (IL-1β) and Tumor Necrosis Factor-alpha (TNFα), as well as pro-coagulant factors like Factor VIIa (FVIIa). The primary mechanism of action is to mitigate the instant blood-mediated inflammatory reaction (IBMIR), a significant challenge that often leads to early graft failure in islet transplantation. By reducing inflammation and inhibiting coagulation at the transplant site, the aptamer-engineered islets are expected to enhance engraftment and survival of the transplanted cells, thereby improving long-term outcomes for patients. The development is currently in preclinical stages, with in vitro and in vivo studies in animal models demonstrating the potential of this approach.

02

Targets

TNFA (Tumor necrosis factor alpha (soluble))

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