Evaluation of Safety and Tolerability of Libella Gene Therapy for Alzheimer's Disease: AAV- hTERT

2019-10-24 12:49:25 | BioPortfolio


Using gene therapy to express active telomerase (hTERT) in human cells has the potential to treat many neurodegenerative diseases related to aging, including Alzheimer's disease (AD).

This study will entail treating subjects with hTERT delivered via transduction using AAV. The goal is to extend the telomeres to prevent, delay, or even reverse the development of the pathology of AD. This will have a direct consequence on cognitive function and quality of life in patients with neurodegenerative diseases, such as AD.


Patients diagnosed with AD who meet with the inclusion - exclusion criteria, will be treated with a single dose of LGT delivered intravenous (IV) and intrathecal (IT) delivery .

Baseline will be performed within 8 weeks of beginning the treatment regimen. The treatment regimen will begin with IV delivery of AAV-hTERT, defined as "Day 0." Safety and efficacy analyses will be conducted at Weeks 1, 4, 13, 26, 39, and 52 post-treatment.

Study objectives

Primary: Safety and Tolerability

1. Investigate the safety and tolerability of AAV-hTERT by IV and IT administration.

Secondary: Provisional Efficacy

1. Investigate LGT's ability to deliver hTERT to human cells and lengthen telomeres.

2. Investigate the effects of lengthening telomeres on AD.

3. Investigate other benefits provided by lengthening telomeres.

Study Design


Alzheimer Disease




IPS Arcasalud SAS




Libella Gene Therapeutics

Results (where available)

View Results


Published on BioPortfolio: 2019-10-24T12:49:25-0400

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