Drug intelligence / Profile preview

AAV-hTERT

Development stage
Phase 1
Lead developer
Libella Gene Therapeutics
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravenous, Intravascular, Intrathecal
01

Overview

AAV-hTERT is an investigational gene therapy that uses an adeno-associated virus (AAV) vector to deliver the human telomerase reverse transcriptase (hTERT) gene into cells. The goal of this therapy is to induce expression of active telomerase, thereby elongating telomeres and potentially reversing or delaying cellular aging. Preclinical studies and limited case reports suggest that systemic administration of AAV-hTERT can result in significant elongation of cellular telomeres and reduction in biological age as measured by telomere length. The approach has been explored for its potential in treating degenerative diseases and age-related conditions, including dementia, cognitive aging, and critical limb ischemia. However, there are concerns about long-term safety risks such as cancer development or immune responses against transfected cells. Clinical trials are ongoing to evaluate its safety and efficacy[1][3][4][5].

Other names
AAV hTERT gene transfer therapyAAV9 hTERT gene transfer therapyAAV-9 hTERT gene transfer therapyAAV 9 hTERT gene transfer therapy
02

Targets

TERT (Telomerase)

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