Photo credit: Pixabay, Geralt
It’s exciting news that there are close to 2,000 advanced therapies currently in clinical trials around the world. But is it good news? It’s exciting to see how far the cell and gene therapy (CGT) industry has come and how quickly the pipeline is growing. It’s exciting that this potentially represents new treatments for patients in a variety of disease areas. As of August 2023, the Alliance for Regenerative Medicine reports there are 1,687 clinical trials across North America, Asia Pacific and Europe. Further, the U.S. Food and Drug Administration (FDA) could approve as many as nine cell and gene therapies this year – a new record – and five could be gene therapies for rare diseases.
Exciting definitely. But it may not be good news if the sector isn’t equipped to handle large numbers of therapies being approved every year. More clinical trials require substantial manufacturing capacity to produce the materials – especially viral vectors, and especially at commercial stage – a skilled and trained workforce, and regulatory oversight – to name three challenges.
I mention the above to introduce the topic for this year’s blog carnival.
With an impressive number of cell and gene therapies in clinical trials, what hurdles need to be overcome for these therapies to reach the marketplace? Will regulatory approval be a bottleneck? Can the system handle all the new therapies getting approved?
Perhaps more than in previous years, this topic allows our contributors to pick and choose what they want to discuss from hurdles to regulatory affairs (often one and the same). As always, the invited thought leaders provide unique perspectives and much to contemplate. I encourage you to read each post.
Chih Wei Teng, VP of Corporate Development at CCRM Australia, endorses a strategy published by McKinsey & Company and looks at it through the lens of health equity and specifically when it comes to Australia’s Aboriginal communities. McKinsey & Company’s eight imperatives for a successful therapy launch depend on proper preparation of the market, the product and the company itself. Read Dr. Teng’s post here.
Don Gibbons, a new Signals contributor, takes an unexpected approach to the topic by focusing on science communications. He argues that poor communication is a major obstacle to this field achieving success. Do you agree? Read what he has to say.
Josee Champagne and Calley Hirsch, partners in the weCANtranslate network, have addressed the topic by offering an “integrated strategic approach” that they believe supports success from R&D through commercialization. It involves three steps that touch on manufacturing, clinical trial design, and de-risking regulatory uncertainty. Read how they propose to do this.
Phil Vanek, Chief Technology Officer at Gamma Biosciences, also thinks there are three key areas where the industry can “double-down” to see cell and gene therapies reach the marketplace. While I’ll share them here, you need to read Dr. Vanek’s post for all the context, details and optimism he shares. He focused on investment prioritization, process simplification and regulatory convergence. Read why here.
Andrew Haller, Entrepreneur in Residence for Medicine by Design, writes about a new initiative that will make Ontario a hub for rare disease gene therapy. TOGETHER (The Ontario GEne ThERapy Network) was established in response to the growing numbers of approvals in cell and gene therapies to ensure efficient delivery to patients beyond academic hospitals; equitable design and development of gene therapies; and maintaining Toronto, Ontario’s role as an engine of innovation. Read about TOGETHER here.
Dave Kent, Professor of Stem Cell Biology at the University of York, UK, also takes a completely different approach to the topic by looking at the need for better academic-industry partnerships and offering solutions to improve current practices. Universities take note! Read his suggestions here.
Interestingly and surprisingly, Michael May, President and CEO of CCRM, has a secret weapon feeding him information about the industry and where it is headed in the future. Dr. May is confident many of the current problems will be solved, but still worries about adoption and patient access challenges. Read his unusual approach to answering this year’s blog carnival questions.
So there you have it! I hope you find this year’s submitted posts to be entertaining, provocative and useful. Please share your thoughts in the Comments section. See you next year!
Stacey Johnson
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