About sAAVient Therapeutics
sAAVient Therapeutics is a clinical stage gene therapy company, focused on gene therapy of human liver diseases with the next generation of AAV3 vectors.
Why Liver Diseases?
- Several human liver diseases are ideal targets for gene therapy
- Several AAV serotype vectors have been used in gene therapy for human liver diseases, but with limited success
Why AAV3 Vectors?
- AAV3 vectors are extremely efficient in delivering genes to human liver at much lower doses
- Safety and efficacy of Next Gen AAV3 vectors has been established in “humanized” mice and in monkeys