Novel Vector Potentiators for AAV-mediated Surfactant B Deficiency correction

Surfactant Protein B (SPB) deficiency is a rare genetic disease where surfactant B is not produced in the lung, proper breathing fails and lungs collapse. The disease usually presents early in life, and unless lung transplantation is performed, no other approaches have provided long-term cure. Yet, lung transplantation is not an ideal approach either, as matching donors are limited, and the procedure is extremely invasive for a baby.
Gene therapy can be an alternative as it has been very successful in animal models. The approach consists of delivering the correct SPB gene into lung cells using an adeno-associated viral vector. This virus has shown to be very efficient at infecting lung cells without causing secondary effects. However, it requires to be dosed in high quantities, which could still have risks. In this project, we are aiming to use small molecules (termed VEPOs) developed by Virano Therapeutics that help the virus infect cells more easily, hence reducing the dose and making the therapy safer.
This project will help Virano Therapeutics advance its gene therapy program, exploring how VEPOs can overcome the challenges of this gene therapy, and potentially more. This will also help establish future partnerships, and pave ways to commercialization.

Faculty Supervisor:

Jean-Simon Diallo

Student:

Partner:

Virano;Stem Cell Network

Discipline:

Life Sciences

Sector:

Biotechnology; Health and Related Sciences and Technology; Pharmaceuticals

University:

University of Ottawa

Program: