CPSA USA Focuses on Personal Health and the Opportunities and Challenges in Cell and Gene Therapies

February 4, 2022 – Newtown, PA (USA) The CPSA USA meeting focused on improving personal health in the clinic and home with its Four Corners series hosted by Mike Lee, founder of the CPSA meetings and CPSA USA 2021 program chair and led by Mark Arnold of LabCorp together with Emily Ehrenfeld of New Objective. Mark led the meeting with a focus on Cell and Gene Therapies (C&GT), the changes in drug development brought on by these innovative solutions and with focus, the challenges that have yet to be solved.

Working within the framework of the Four Corners – Technology, Methodology, Workflow/Process and Regulation and how they are unified via Informatics, Mark launched the lively and active discussion with support from a panel of industry leaders including Steven Eck, Astra Zeneca, and Heather Myler, PPD. Mark’s statement highlighting C&GT as a current topic of interest due to the COVID vaccines, with the reality that analysts have been supporting these therapies for several years unified group and set the stage for the discussion.

Heather Myler highlighted early in the discussion the need for good regulatory standards with Brad Ackerman, Eli Lilly, bringing the focus to a common thread in that most targets are proteins solidifying LC-MS as a key analytical tool. Cost and controlling cost were discussed, with multiple aspects highlighted as impacting cost including regulations (increasing cost) and logistics; this coupled with a limited pool of assays or experienced staff to run the assays were underlined as current challenges. Heather highlighted the prevalence of Gene Therapy in rare disease in it being a good and simple model enabling the technology to be proven with a validated clinical endpoint – connecting this innovation to the patient.

Within the Four Corners discussion, Mark together with Mike and Emily, was able to identify the gaps and challenges that have yet to be solved, so as to “bring these therapies faster to patients in need”. Mark summarized the discussion noting C&GT are a huge space, which is broader because of the therapy – using the (human) body to generate proteins or cells – which is still in its infancy. The challenges which were highlighted outline opportunity and the collaborative discussion today is just one in a series of many within CPSA. The conversation will continue with its next installment planned for Friday June 3^rd, where the CPSA community will continue to come together – to think big and make a difference.