How Is the Cell and Gene Therapy Landscape Evolving?
Betty Woo discusses the industry's progress and shares her predictions for the future of CGTs.
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Cell and gene therapies (CGTs) are transforming the treatment landscape for various diseases, offering potential cures for conditions previously considered untreatable. With a rapidly growing number of CGTs entering markets worldwide, these therapies are expanding treatment options for patients with rare and complex conditions. However, as the field matures, challenges around scalable manufacturing, high development costs and equitable patient access continue to pose significant barriers.
To explore how the CGT landscape is evolving, Technology Networks spoke with Betty Woo, vice president of Cell, Gene and Advanced Therapies at Thermo Fisher Scientific. Woo discusses the industry's progress, challenges in scaling manufacturing processes and the need for automation to reduce costs and errors. Additionally, Woo shares her insights on promising advancements in stem cell therapies and in vivo gene therapy applications, as well as her predictions for the future of CGTs.
What does the landscape for CGT look like today, and what does recent progress mean for patients?
Betty Woo, PhD (BW):
While CGTs are still emerging fields, significant progress continues. At the end of 2023, the FDA approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for treating sickle cell disease (SCD) in patients 12 years and older. Though SCD is classified as a rare disease, it is one of the most common inherited blood disorders, with a worldwide impact in the millions. Today, there are more than 30 FDA-approved cell and gene therapies designed to treat conditions that previously had limited or no effective treatment options.
Approximately half of the 1,700 active cell and gene therapy clinical trials are oncology-related. However, promising clinical results using CGT in treating autoimmune diseases are opening the floodgates to the applicability of these therapies for broad patient populations, like the 5–8% of the US population with an autoimmune disorder, according to the National Institutes of Health.
For patients struggling to find a viable treatment, or working to resolve their diagnostic odyssey, CGTs hold immense value since they have the potential to not only treat diseases but also potentially cure inherited disorders. The progress made in cell and gene therapy is giving patients hope.
Still, patients’ access to these therapies remains a challenge, motivating the industry to help make these therapies more widely available, especially as therapies continue to come to market.
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Across the industry, scaling processes to develop enough of a particular therapy in a consistent, robust way is an obstacle, and this can often lead to a delay in getting these treatments to the patients who need them. Because CGT development and manufacturing processes are complex, there is often a large reliance on manual processes, which increases costs and the risk of human error. Workflows can require hours of manual manipulation of cells and these complex processes rely on highly trained professionals. Workforce development is limited, putting more demand on the few experts with specialized training.
Outside of scaling processes, the manufacturing costs can be prohibitive to commercialization, with extended costs to patients in the hundreds of thousands to millions. New reimbursement models in the US are expected this year, a definite step in the right direction to increase the accessibility of these therapies to patients in urgent need.
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Collaboration is the lifeblood of the CGT industry. It extends to academia, biopharma innovators, technology developers, manufacturers, regulators, service providers and other stakeholders and is incredibly important in getting treatments to patients faster. Through cross-industry collaboration, we can glean diverse perspectives and integrate our best thinking from academic thought leaders with industry process scientists to expedite scientific translation. We can also move promising therapeutic candidates efficiently through the clinical trial process, regulatory approval, commercialization, and ultimately, patient access.
Increased collaboration is vital to improving scalability, reducing costs and increasing efficiency. Cell and gene therapy is a fast-growing industry, so sharing knowledge and technology best practices as they are developed will help maximize the potential of the CGT ecosystem and help us deliver innovative therapies to patients more quickly and broadly.
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While it’s hard to predict what specific new therapies will emerge, I imagine we will continue to see investments in treatments for a variety of rare and orphan diseases, including inherited diseases and solid tumor cancers. The industry is already expanding clinical development into new therapeutic indications, which is incredibly important to bring the full potential of CGT to as many patients as possible.
I also think we’ll see advancements in the harmonization of global regulatory requirements and quality standards. As the CGT industry continues to grow, we will need to set up clear, uniform regulatory requirements to reduce variability in manufacturing and ensure safety and quality standards are met. Of course, this harmonization will require cross-industry collaboration and adoption as the market matures.
