Tips for Transitioning Cell & Gene Therapies to the Clinic
How to circumvent common barriers to bringing cell and gene therapies to the clinic
Dr. Clarke has more than 15 years of experience developing solutions to support cell and gene therapies. Dominic is currently the chief technical officer for cell & gene therapies at Discovery Life Sciences, where he is responsible for providing leadership and direction on their current and future products & services roadmap and overall strategy in CGT. Previous roles include director of global cell therapy strategy & innovation at HemaCare and Charles River Laboratories, global product manager for Charter Medical's cell therapy and bioprocessing single-use systems portfolio, and director of research and development for BioLife Solutions. Dominic currently serves as the chair for the International Society for Cell and Gene Therapies Process Development & Manufacturing Committee, with efforts directed toward translating research and process development from bench to clinic.
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To say the cell and gene therapy space is exploding would be an understatement. These life-changing therapies reimagine biological material as therapeutics and impact the lab bench, hospital bedside, and business board room. People are taking note—the cell and gene market is predicted to reach a value of $25 billion by 2027.
Cell and gene therapies deliver on the promise of precision medicine and have already begun changing patient lives. However, transitioning cell and gene therapies from development to clinical translation is not trivial. Here, we describe the common barriers cell and gene therapies face when stepping into the clinic and how to circumvent them.
Quality in, quality out
From discovery through translation, cell and gene therapies have a complex developmental pipeline. Foundational researchers must first prove the scientific rigor of their therapeutic design. This requires high-quality diseased biospecimens to accurately characterize the therapeutic mechanism of action. Quality is top of mind to ensure accurate and reproducible results as a foundation for therapeutic development.
When transitioning to commercial manufacturing, a common pitfall that cell and gene manufacturers fall into is starting with poor-quality materials. Allogeneic cell therapies are at the mercy of the quality of your starting materials. To ensure the success of your cell or gene therapy, you need to ensure your donor source material is reliable and of the highest quality.
Allogeneic cell therapy donor cells must behave consistently in the lab and the clinic, requiring rigorous quality control and vetting. To reduce the risk inherent in commercializing cell and gene therapies, prioritize using consistent and appropriately characterized human donor starting materials.
How to identify high-quality donor materials
With so many options for sourcing cell and gene therapy material, it can be challenging to differentiate between high- and low-quality materials. However, nothing is more critical to developing a cell therapy.
High-quality donor cells will come from a trusted source with extensive experience providing critical biological samples. The cells will be highly characterized through multi-omic analysis and meet rigorous GMP grade standards. The source must provide a large, recallable donor pool that ensures consistency and scale.
Keep it consistent
It can be tempting to rely on various service providers for your cell and gene therapy needs to save costs. However, what you may save in money you lose in efficiency. Requesting all your cell and gene therapy resources from a central provider streamlines your experience and ensures robust quality throughout your developmental pipeline. Bundling several service providers together creates inefficient transfer points that could derail your therapeutic product.
Cell and gene therapies are poised to revolutionize the clinic. But generating these therapies is an enormous undertaking. It takes the seamless coordination of discovery biomaterials, excellent-quality scalable donor starting materials, and consistent bioproducts and services. You need a trusted service provider with a proven track record to ensure clinical success for your cell and gene therapy.