Supporting the Development of Regenerative Medicines

Regenerative medicine can be defined as a branch of translational research which focuses on methods to regrow, repair or replace human cells, tissues or organs. This approach holds promise for revolutionary new cures for devastating conditions such as heart disease, diabetes and cancer. We recently spoke with Michael May, President & CEO of CCRM, a Canadian not-for-profit organization supporting the development of regenerative medicines and associated technologies to learn more about CCRM’s mission, latest projects and collaborations.

Laura Mason (LM): Could you tell us more about CCRM, the company mission and goals?

Michael May (MM): CCRM is a Canadian not-for-profit organization funded by the Government of Canada, the Province of Ontario, and leading academic and industry partners. It supports the development of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy. Our goal is to bridge the commercialization gap and our model is based on developing networks of stakeholders: researchers, leading companies, strategic investors and entrepreneurs. At CCRM, we aim to accelerate the translation of scientific discovery into new companies and marketable products for patients, with specialized teams, dedicated funding and unique infrastructure.

CCRM’s mission is to generate sustainable health and economic benefits through global collaboration in cell and gene therapy, and regenerative medicine technologies.

CCRM’s vision is twofold: first, to be the preferred global destination for the best people, technologies, clinical trials, companies and investments in regenerative medicine; and, secondly, to be the premier global enabler of clinically-tested, revolutionary new medical therapies and foundational technologies.

LM: What is regenerative medicine (RM)?

MM: Regenerative medicine, including cell and gene therapy, harnesses the power of (stem) cells, biomaterials, molecules and genetic modification to repair, regenerate or replace diseased cells, tissues and organs. This approach is disrupting the traditional biotechnology and pharmaceutical industries with the promise of revolutionary new cures for devastating and costly conditions such as heart disease, diabetes and cancer.

The current global market for regenerative medicine is USD$36B and forecasted to grow to reach USD$49.41B by 2021.¹ There were 959 clinical trials in cell, gene and tissue therapy underway worldwide at the close of the first quarter of 2018.² In 2017, the U.S. Food and Drug Administration approved the first cellular immunotherapies and gene therapies, alongside reimbursements by the U.S. Centers for Medicare & Medicaid Services. This has resulted in record-breaking investment and acquisitions in the sector.

LM: A recent press release mentions CCRM’s partnership with GE Healthcare. Could you expand on this partnership? Are there any specific ongoing collaborative projects you could tell us more about?

MM: CCRM and GE Healthcare have been working collaboratively since 2016, when we established the Centre for Advanced Therapeutic Cell Technologies (CATCT). CATCT bridges the gap between pre-clinical research and commercially-ready manufacturing of cell and gene therapies, enabling promising technologies to reach the market. It is an advanced manufacturing solutions facility that is addressing the challenges in CGT production, most notably, in identifying, developing and adopting suitable solutions for the cells and viral vectors required for clinical trials and commercial production.

CATCT was created with the support of both industry and government, reflecting the broad need for the solutions that CATCT will provide. GE Healthcare is the anchor partner for CATCT with a commitment of CAD$20 million for the project, matched with CAD$20 million from the Federal Economic Development Agency for Southern Ontario. CATCT performs technology development in four areas to support the global cell and gene therapy industry: stem cell scale-up, immunotherapy, viral vector production, and disruptive technologies; and, we offer fee-for-service work to customers.

In 2018, GE Healthcare and CCRM announced two fee-for-service projects conducted by the CATCT team.

In January, Cellular Biomedicine Group Inc. (CBMG), a clinical-stage biopharmaceutical firm engaged in the development of immunotherapies for cancer, announced that it will configure part of its facility in Shanghai with GE Healthcare’s FlexFactory™ platform. CCRM is enabling GE Healthcare to provide CBMG with process development services through CATCT. In addition to process development, GE Healthcare’s FlexFactory solution will provide training services, cell processing equipment, semi-automation capabilities and digital connectivity solutions, supporting good manufacturing practices (GMP) manufacturing.

In June, GE Healthcare and CCRM announced they are supporting DiscGenics’ efforts to scale-up and optimize manufacturing of its allogeneic, injectable cell therapy, IDCT. The therapy is being developed to treat degenerative disc disease. The support will include conducting process, assay and media development for DiscGenics’ manufacturing process.

LM: Could you tell us about CCRM’s existing facilities and some of the work that goes on in these facilities?

MM: Recent accomplishments in CATCT have moved projects closer to bridging the gap between pre-clinical research and commercially-ready manufacturing of cell and gene therapies.

Major milestones have been reached in CATCT’s pluripotent stem cell (PSC), immunotherapy and lentiviral vector (LVV) development programs. The PSC team has generated more than 36 billion PSCs in a single 10 L run. The immunotherapy team has developed a closed, integrated, and semi-automated T cell workflow. Further, the LVV team has scaled-up a high-titer production process to the 5 L scale and developed a corresponding downstream workflow.

The capabilities of CATCT’s process development team were expanded with the recent arrival of its largest stirred tank reactor to-date (GE XDR-50), which permits scale-up in cell manufacturing processes to 50 L in a single-use system. The addition of an automated liquid handling and culturing robot (Hamilton Vantage) provides custom high-throughput design-of-experiments capabilities.

In addition, CCRM recently acknowledged the reprogramming of its 100th patient line, marking the generation of over 200 induced pluripotent stem cell (iPSC) lines overall in CCRM’s development lab. The development lab team provides services including cell reprogramming and engineering, technology translation and protocol development. The team's gene editing service has also taken off and staff is busy performing gene modifications in PSC lines representing many different disease indications. Looking forward, the team has started to work with multiple immunotherapies and this will be a focus for the coming year.

LM: CCRM has partnered with the University Health Network (UHN) to design and build the Centre for Cell and Vector Production (CCVP). Could tell us more about this new world-class facility?

MM: The Centre for Cell and Vector Production (CCVP), a 14,000 ft2 (~1,300 m2) Good Manufacturing Practices (GMP) facility located in CCRM’s space in Toronto’s world-class Discovery District, will be operational in October 2018. CCVP will offer cell and viral vector manufacturing, cell banking, fill and finish, technology transfer, assay development, process development and GMP training. The facility is being designed to meet international standards that will be acceptable to regulators such as Health Canada, U.S. FDA and EMA. It will have eight cell therapy suites and two vector production suites, and will contain ISO Class 7/Grade B clean rooms.

The facility will have the current and future technologies that are driving the industry forward, allowing CCRM to deliver regenerative medicine technologies, and cell and gene therapy products for early-phase clinical trials for years to come. CCRM will also assist with the transfer to clients’ manufacturing facilities or to a contract manufacturing organization when their early clinical trials prove successful.

CCVP is being built and will be operated by CCRM. In addition to contributions of its own, financial support for CCVP has been provided by the University Health Network through a Canadian Foundation for Innovation grant for construction, equipment and operations.

Michael May was speaking to Laura Elizabeth Mason, Science Writer for Technology Networks.

References
1. Marketsandmarkets.com. (2017). Regenerative Medicines Market by Therapy, Product & Application - 2021 | MarketsandMarkets. [online] Available at: https://www.marketsandmarkets.com/Market-Reports/regenerative-medicine-market-65442579.html [Accessed 5 Jul. 2018].
2. Alliance for Regenerative Medicine. (2018). Q1 2018 Data Report - Alliance for Regenerative Medicine. [online] Available at: https://alliancerm.org/publication/q1-2018/ [Accessed 5 Jul. 2018].