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Updated: Jul 9, 2026

Utilizing Murine Inducible Telomerase Alleles in the Studies of Tissue Degeneration/Regeneration and Cancer
08:34

Utilizing Murine Inducible Telomerase Alleles in the Studies of Tissue Degeneration/Regeneration and Cancer

Published on: April 13, 2015

Oligonucleotide conjugate GRN163L targeting human telomerase as potential anticancer and antimetastatic agent

Sergei M Gryaznov1, Shalmica Jackson, Gunnur Dikmen

  • 1Geron Corporation, 230 Constitution Drive, Menlo Park, CA 94025, USA. sgryaznov@geron.com

Nucleosides, Nucleotides & Nucleic Acids
|December 11, 2007
PubMed

Insights

A novel telomerase inhibitor, GRN163L, effectively halts cancer cell proliferation and metastasis in preclinical models. This compound is now undergoing clinical trials for solid tumors and chronic lymphocytic leukemia (CLL).

Area of Science:

  • Oncology
  • Molecular Biology
  • Biochemistry

Background:

  • Telomerase is crucial for cancer cell immortality.
  • Targeting telomerase offers a promising strategy for cancer therapy.

Purpose of the Study:

  • To introduce and evaluate a novel telomerase inhibitor, GRN163L.
  • To assess the anti-cancer efficacy of GRN163L in preclinical models and early-stage clinical trials.

Main Methods:

  • GRN163L, a 13-mer oligonucleotide N3' --> P5'-thio-phosphoramidate lipid conjugate, was synthesized.
  • In vitro assays measured telomerase inhibition in tumor cell lines (IC50: 3-300 nM).
  • In vivo studies evaluated GRN163L's effect on primary tumor growth and metastasis in animal models.

Main Results:

  • GRN163L demonstrated potent, sequence-specific telomerase inhibition without cellular uptake enhancers.
  • The compound significantly inhibited primary tumor growth and reduced metastasis spread in vivo.
  • GRN163L is currently in Phase I and Phase I/II clinical studies.

Conclusions:

  • GRN163L is a potent telomerase inhibitor with significant anti-cancer activity.
  • GRN163L shows promise for treating solid tumors and chronic lymphocytic leukemia (CLL).
  • Further clinical evaluation of GRN163L is warranted.

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