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09:54
Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
Published on: November 4, 2018
Gene therapy (alpha1-antitrypsin) (GeneMedicine/Vanderbilt)
1Central Research Division, Pfizer Inc, Groton, CT 06340, USA. KRISHR@pfizer.com
Idrugs : the Investigational Drugs Journal
|May 10, 2008
Summary
This non-viral gene therapy for alpha1-antitrypsin (AAT) deficiency is safe and effective. Inhalation delivery of AAT protein increased levels and showed anti-inflammatory effects in a Phase I trial.
Area of Science:
- Biotechnology
- Gene Therapy
- Pulmonology
Background:
- Alpha1-antitrypsin (AAT) deficiency is a genetic disorder that can lead to emphysema.
- AAT inhibits elastase, an enzyme that can damage lung tissue.
- Current treatments for AAT deficiency are limited.
Purpose of the Study:
- To evaluate the safety and efficacy of a non-viral gene therapy for AAT deficiency.
- To assess the ability of inhaled gene therapy to produce therapeutic levels of AAT in the lung.
- To investigate the anti-inflammatory effects of the gene therapy.
Main Methods:
- A physician-initiated Phase I clinical trial was conducted.
- GeneMedicine's non-viral gene therapy, utilizing DOTMA cationic lipid, was administered by inhalation.
- AAT protein levels in nasal lavage fluid and anti-inflammatory effects were measured.
Main Results:
- The gene therapy was found to be safe in all five patients.
- AAT protein levels increased in nasal lavage fluid.
- An anti-inflammatory effect was observed, exceeding that of AAT protein administration alone.
Conclusions:
- Non-viral gene therapy is a safe and potentially effective treatment for AAT deficiency.
- Inhaled gene therapy can achieve therapeutic AAT levels and exert anti-inflammatory effects in the lung.
- This platform may be applicable to other lung diseases.
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