L2M – A2O

Direct lineage reprogramming (DLR) is the newest advance in the field of cellular reprogramming. It is the forced conversion of one mature cell type to another, without the need for a pluripotent intermediate, directly at the site of injury or disease. We have developed DLR technology to convert astrocytes into new oligodendrocyte lineage cells (iOLCs) for the treatment of central nervous system (CNS) disease and injury. To ensure the success of our technology in clinical trials and increase the likelihood of commercial viability we will source input from stakeholders and key opinion leaders in four main areas. First, we will validate the market fit and clinical need for our technology in our proposed beachhead indication, neuromyelitis optica, through clinicians and patients. Second, we will obtain insight into possible delivery methods (AAV, RNA vaccines, LNPs) for our technology. This will be assessed with groups specializing in these methods to determine optimal efficacy and safety parameters but also with clinicians and patients to identify administration preferences. Additionally, we will meet with regulatory bodies and intellectual property experts to understand the regulatory landscape, clinical trials space and licensing agreements. Finally, we will connect with business strategists and pharmaceutical companies (end buyer) to come up with a market plan and licensing model that supports our vision of a platform technology. As a result of this project we will be able to make evidence-based decisions that de-risk our technology, accelerate our path to clinical trials, and position us for successful partnership negotiations.

Faculty Supervisor:

Jeremy Sivak;Maryam Faiz

Student:

Partner:

DMZ Ventures Inc

Discipline:

Life Sciences

Sector:

Professional, scientific and technical services

University:

University Health Network

Program: