In-vivo CAR-T Therapy for Glioblastoma by Novel Non-Viral Gene Editing System
Project Overview
Challenge
CAR-T therapies have delivered promising results for certain hematological cancers but remain largely ineffective for solid tumours with failure nodes including complex and hostile tumor microenvironments and the lack of adapted technological tools. We propose to adapt a novel gene editing system to develop an in-vivo CAR-T gene editing treatment for glioblastoma (GBM), an aggressive type of brain cancer with a 5-year survival rate of less than 10%, through the targeted delivery of a gene editing protein and CAR encoded DNA donor to T-cells administered intravenously.
Solution
Jenthera Therapeutics has developed a novel gene editing platform featuring the targeted and direct delivery of the CRISPR ribonucleoprotein, a clear differentiation from current methodologies that require external agents, such as viruses or liposomes to achieve delivery of the editing complex to cells. These agents create important precision and safety issues and limit possible therapeutic applications. This approach was previously successfully applied to the direct targeting of solid tumours as well as in a CAR-T program against a hematological target where the complete eradication of the malignancies was observed at minimal starting doses through intravenous administration.
Impact
A systemically-administered immunotherapy for GBM, ready for preclinical evaluation would change the paradigm in treatment and offer a groundbreaking, safe, non-invasive therapy to patients facing a dire prognosis with few options, harnessing the patient’s own immune arsenal and offering extended immune protection against recurrence. For the partners involved, this would represent a clear commercial deliverable of a in-vivo CAR construct and therapeutic approach and a proof of concept for the technology with significant commercial potential translatable to other targets within and outside of oncology. For the Quebec ecosystem, effects range from decreased pressure on health care resources to the recognition of Quebec’s leading innovative force on the world stage.
Principal Investigator
Ovidiu Jumanca , Institut de Recherches Cliniques de Montréal (IRCM)
Team Members
Maxime Rousseaux, University of Ottawa
Christophe Brun-Baronnat, CERASP
Partners and Donors
CQDM
Jenthera Therapeutics