When it comes to research and commercialization in regenerative medicine and other advanced therapies, companies in Ontario are proving they have the innovative technologies and strong talent to compete globally. They are recognizing marketplace needs and striving to meet industry demands by developing new technologies, investing in the right capital projects, forming strategic partnerships and striving for overall excellence in the biotechnology sector.
Ontario companies show they are the ones to watch in the life sciences and biotechnology space as they fill marketplace gaps and draw talent from around the world. Here are just four examples of Ontario companies excelling at meeting the innovative needs of the biotechnology sector.
Recognizing that a marketplace gap exists for patients suffering with ailments such as Parkinson’s disease, degenerative heart disease and autoimmune disorders that have few treatment options, BlueRock Therapeutics, uses state-of-the-art technologies in cell biology and gene editing to create fundamentally new ways to treat these types of diseases. This new technology, the BlueRock CELL+GENETM platform, overcomes the limitations of current methods that rely on harvested primary cells to develop therapeutics.
Where authentic cells enable cell replacement, restorative function, and disease reversal, engineered cells delivered in ways never before thought possible, enable therapies to treat previously intractable common and rare diseases. As noted on their website, BlueRock Therapeutics are “passionate about delivering on the promise of cellular and gene therapy, shaping the future of cellular medicine, and delivering new therapies to millions of patients with few treatment options”.
BlueRock Therapeutics’ authentic cell replacement projects currently underway in the neurology field include research on microglia for neurodegeneration, oligodendrocyte for demyelinating disorders and their lead project, dopaminergic neurons for Parkinson’s disease. BlueRock founders have demonstrated robust preclinical data for dopaminergic neurons restoring motor function and increasing dopamine release as they mature. This project is expected to advance into clinical development by the end of 2019.
Centre for Commercialization of Regenerative Medicine (CCRM)
Mitchel Sivilotti, Chief Operations Officer for CCRM, a leader in developing and commercializing cell and gene therapies, and regenerative medicine-based technologies says, when it comes to the bio-manufacturing space, “There are a number of significant voids, but first amongst those is the affordability of current, highly manual manufacturing methods. Despite major advances in cell and gene therapy clinical efficacy, we have considerable labour costs and open manufacturing systems requiring large manufacturing infrastructure”. Sivilotti is hopeful that “economies of scale and the results of process optimization will begin to impact the cost structure in both autologous (cells and tissues derived from the patient) and allogeneic (donor) products in the near future”.
From its inception, CCRM designed its Centre for Advanced Therapeutic Cell Technologies (CATCT) to address process scale-up and scale-out challenges by incorporating advanced cell therapy manufacturing technology. “Our programs include deep investment in viral vectors, CAR-T, media development/optimization, and pluripotent stem cell expansion/differentiation in 3D suspension culture bioreactors (to name a few),” says Sivilotti. “This experience ties directly to our clinical manufacturing facility, the Centre for Cell and Vector Production (CCVP), which has 10 ISO Class 7/Grade B clean rooms, including two dedicated to viral vector production. The CCVP can manufacture in accordance with a client’s current procedures or, together with the CATCT, provide access to process improvements designed to solve key challenges in scaled manufacturability, product yield and costs.”
Turnstone Biologics is a clinical-stage immune-oncology company leading the next generation of oncolytic viral therapies. In November 2018, they presented pre-clinical data supporting the development of a new therapeutic platform, Vaccinia, developed by Dr. John Bell and his colleagues at the Ottawa Hospital Research Institute and the University of Ottawa. This platform was developed to be potent, immune-stimulatory and highly selective.
“This is an exciting time for Turnstone as we unveil another novel viral immunotherapy platform technology in our continued pursuit to develop transformative technologies that advance the treatment of cancer,” said Mike Burgess, MBChB, Ph.D., President, Research and Development at Turnstone Biologics. “We are rapidly advancing our lead candidate from this proprietary platform, TBio-6517, to the clinic with the goal of achieving effective and durable outcomes for a greater number of cancer patients.”
The Vaccinia virus can be delivered systematically and is designed to target and kill cancer cells throughout the body, modulate the tumor microenvironment and stimulate both innate and adaptive immune responses. Key milestones for Turnstone Vaccinia include rapidly killing more than 80 per cent of cancer cells (in mice) across multiple cell lines while sparing normal cells, replicating in human lung, sarcoma, melanoma, ovarian, gastric and thymic tumor explants and demonstrated selective expression of its three encoded immunomodulators at the sites of tumors in preclinical models with no evidence of exposure in peripheral blood.
The bioengineering technologies and processes created by Octane Biotech are transforming the delivery of reversative medicine solutions and creating market-leading business opportunities for their global partner network. Octane CocoonTM, cell culture and tissue engineering systems producing living implants, is one of those technologies.
Octane CocoonTM is a patient-scale cell therapy system that forms the core of a series of versatile cell and tissue therapy production systems that opens an array of clinical applications. It aims to answer key challenges facing autologous cell therapies as they reach the commercial stage, such as variability in call yields and strict regulations once products are registered. Octane recently partnered with Lonza Group Ltd. in Switzerland to further develop and advance the technology.
Timothy Smith, co-founder of Octane Biotech with Ian Grant, said, “As continuing shareholders and directors of Octane Biotech, we welcome the expanded engagement of Lonza to significantly accelerate and reinforce the role of the Cocoon™ system as a game changer in the autologous cell therapy manufacturing space. Since the inception of the company, the vision of our team has been to bring award-winning innovation to this sector, and we are confident that Lonza will excel in enabling these transformative therapies to reach more patients throughout the world.”
Cocoon™ will be further developed with continued support from both Octane’s current site in Kingston, Ont. and their Maryland, U.S. research and development site.
Ontario is the place to be when it comes to life sciences. Whether it’s innovative technology or an expansive ecosystem of partners, these Ontario companies are proving they have what it takes to succeed on a global level.