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First-time Right Approach to Advanced Therapy Process Development

January 18, 2022

A first-time-right approach to cell and gene therapy process development involves designing and executing the process in a way that ensures the highest likelihood of success on the first attempt. This approach is critical because cell and gene therapies are complex and highly regulated, making the cost of failed attempts extremely high.

By adopting a first-time-right approach to cell and gene therapy process development, companies can minimize the risk of failure, reduce development costs, and bring safe and effective products to market more quickly. Download our whitepaper to get proven strategies for driving the success of cell and gene therapy development.

Here are key elements of a first-time-right approach to cell and gene therapy process development:

• Investing in robust cell lines: Unlike the monoclonal antibody (mAb) field where manufacturing is Start with good plasmids: Proper sequencing of plasmids is necessary to ensure that no unwanted mutations are present.

• Explore the entire formulation space: Often the rush to get to market will encourage some companies to skip this important step in developing a drug with all the required quality attributes. But when gene therapy formulation is an afterthought, it can require great time and expense on the back end for reformulation or even further process development.

• What about subvisible particles? Gene and gene-modified cell therapy developers should monitor the presence of subvisible particles beginning at the earliest development stages. As drug products move to later development stages, regulatory expectations regarding subvisible particles (which have immunogenicity potential that can lead to drug degradation) become stricter and more extensive.

• Build in flexible manufacturing solutions: A modular approach to drug product presentation helps to address clinics’ need for access to the exact volumes of products required for infusion. It allows delivery of the specific dosages needed throughout the entire dose-escalation study as efficiently as possible.

• Bridge the analytics gap: Process and product understanding is essential to the successful development of high-quality CGT products. Integration of state-of-the-art analytics from bench-top technology development through GMP manufacturing accelerates process development and scale-up.

• Working towards industrialization: Considering the complexities around industrializing CGTs, innovators and sponsors should endeavor to implement right-first-time processes that utilize industry best practices. CDMOs should provide expert teams that are integrated with customers to ensure that every step of the process from plasmid design to fill/finish is well planned.

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