Technology Integrators in Cell and Gene Therapy: Powering Innovation

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Julien Meissonnier, vice president and chief scientific officer, Catalent

Meissonnier shared information about the expanded role of contract development and manufacturing organizations (CDMOs) in cell and gene therapies (CGTs). Catalent entered the gene therapy space in 2019 through acquisition of Paragon BioServices, adding expertise in adenoassociated viruses (AAVs) and viral vector manufacturing.

Cell therapy capabilities were acquired early in 2020 through acquisition of MaSTherCell, with the goal of providing end-to-end integrated solutions to CGT customers. In addition to expertise in chimeric antigen receptor (CAR) T cells, that acquisition brought experience with other complex cell modalities. Such modalities address issues that cannot be treated by small molecules and monoclonal antibodies (MAbs). CGTs offer new mechanisms, activate new cellular functions, and replace defective genes. He noted that “the speed at which cell modalities are evolving in complexity and specificity is amazing to see,” and he focused the rest of his talk on the next generation of therapeutic approaches.

All innovators face a common need: to ensure that innovative therapies are developable, scalable, and symmetrically manufacturable. Meissonnier spoke about developability and manufacturability assessments; the importance of a flexible, multipurposed manufacturing infrastructure; the integrated toolkit of Catalent’s licensed technologies; and a methodology to translate research-based manufacturing processes into clinical and commercial worlds.

Three out of 10 candidates from small-cap innovators in small-molecule biologics or cell and gene therapy segments are not developable or manufacturable. Approaches have required reengineering that can take a minimum six to nine months to accomplish. A CDMO’s task is to work at early stages with innovators to help design successful development and manufacturing strategies. Catalent experts work with innovators on an open-access model to interact freely, early, and often to advise innovators on the pathway to develop reliable manufacturing technologies. Most cell therapies undergo early phase and academic trials with manual operations and extensive testing, which emphasizes why Catalent wants to help close the technological gaps that separate innovators from bringing products to clinical trials.

Using examples from a previous project related to an autologous T-cell receptor (TCR) cell therapy, Meissonnier showed how his company chose optimal equipment to eliminate some manual operations, reduce variability in critical product parameters (CPPs), and find reliable alternatives to a number of processing steps.

Because companies that develop CGT processes often need to repurpose/customize equipment used in preexisting processes, equipment becomes “the movable variable in your model.” Innovators seldom have the time and capital to keep up with development, delivery, and manufacturing technologies from preferred enablers and to continuously invest in R&D to innovate and evolve in-house platforms. Catalent’s acquisition of Paragon technologies has helped it to bring processes to scale for all 60 of its clients’ programs. Integrating talented scientific teams from MaSTherCell and Paragon is allowing Catalent to develop end-to-end offerings in the gene therapy space.

Meanwhile, the company’s R&D teams are innovating and developing distinct capabilities to simplify and accelerate the path to clinical testing and remain open to partnering with other groups to access new, potentially disruptive technologies.

Catalent’s biological platform has been evolving in complexity and specificity, gradually moving from recombinant proteins into CGTs, and now within a flexible and multipurpose environment. The company always links a quality-by-design concept to manufacturing-by-design concepts, for which both critical quality attributes and critical manufacturing attributes are considered equally in process characterization studies.

Meissonnier concluded by emphasizing the dynamic nature of the CGT space, the disruptive innovations on the horizon, and the promises that new technologies offer to patients. By continuously evolving technologies, partnering models, infrastructures, and expertise in the cell and gene therapeutic field, his company seeks to become a leading development and commercial partner for advanced technologies.

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