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AAV-TERT-01

Development stage: Preclinical
Lead developer: Telomere Therapeutics
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies

Overview

AAV-TERT-01 is an adeno-associated virus (AAV) gene therapy being developed by Telomere Therapeutics for the treatment of idiopathic pulmonary fibrosis (IPF). The therapy utilizes an AAV vector to deliver an optimized telomerase reverse transcriptase (TERT) gene sequence, specifically targeting alveolar epithelial cells in the lung. By driving the expression of telomerase, AAV-TERT-01 aims to restore telomere maintenance and address telomere dysfunction, a key driver in the pathogenesis of IPF. The program is currently in the IND-enabling stage and has demonstrated the ability to reverse disease in preclinical animal models of pulmonary fibrosis associated with short telomeres.

Targets

TERT (Telomerase)

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