The Process of Bringing a Clinical Discovery to Market – From Concept to Commercial for Cell and Gene Therapy Products

Translating a drug from a concept to a treatment that is scalable and manufacturable can be the biggest challenge for developers of cell and gene therapies (CGT). Particularly in this field, the challenge is even greater because the people who are able to perform the necessary tasks need to be highly skilled cell biologists with extensive experience working with human cells. In addition, CGTs are highly personalized therapies, which means that the scalability challenge for cell manufacturers is even greater. To assist in this process, companies are partnering with contract development and manufacturing organizations (CDMO), which are companies within the biopharmaceutical industry that provide drug development and manufacturing services.

The Clinical Discovery Process

There are 5 steps in the typical clinical development process:

• Discovery and Development 

• Preclinical Research

• Clinical Development

• FDA Review

• FDA Post-Market Safety Monitoring

However, when it comes to cell and gene therapies, the process of bringing a new therapy to market is a bit more complex. We’ve given an overview of the steps in the clinical development process for cell and gene therapies below:

Product Characterization

In the initial phase of a clinical discovery, data is collected to determine factors that would identify the drug, such as sterility, dosage, and any potential side effects. During this very early stage of development, the product composition is defined based on what is acceptable for larger scale commercialization, and what an ideal outcome would be. In other words, what the drug is, who it’s for, how much a person would need, and what the ideal results would be are defined. 

There is also testing done during this phase to determine the effectiveness of the drug, and following Current Good Manufacturing Practice (CGMP) regulations. This phase would align with the Discovery and Development and Preclinical Research steps for an average clinical discovery.

Process Characterization

This phase aims to accumulate enough scientific data throughout the product life cycle that shows the process can consistently produce a quality product and that the process is reproducible. The first step is for a developer to identify the critical quality attributes (CQAs) of the product. These are the attributes that align with the product characteristics and ensure that the drug does what it’s intended to do. The process is then tested keeping these CQAs in mind, and refined to a point where the developer has confidence in the reproducibility of the process and, therefore, the product.

Process Scalability

Once the process has been refined enough to be reproducible, the next stage involves testing its scalability – or how quickly the product can be made available in large quantities. For an allogeneic cell therapy for which large numbers and doses of cells are needed from each batch, the industrial scalability will be largely dependent on the costs associated with the process. Other factors that affect the scalability are the therapeutic dose per patient, how many patients will be treated in a given time period, the stability of the cell product, and the market size, to name a few.

Downstream Processing

Downstream processing is the recovery and purification of cell products. This part of the process involves harvesting and washing the cell product to prepare for use in cell and gene therapies. This is also the part of the process where scalability becomes an even bigger challenge, as it needs to meet cGMP standards, which requires cell processing to be performed in systems closed to outside environments.

Final Formulation and Filling

The final step in the process of bringing a cell and gene therapy discovery to market is the final formulation and filling. Once the cells have been harvested and processed, the formulated product may be cryopreserved (frozen using liquid nitrogen). In this case, cryoprotectant additives will be used to achieve the proper concentration for the therapeutic dose. The cells will remain at the appropriate temperature until the dose is administered so that the cells retain their viability and CQAs.

ThermoGenesis the future of Cell Therapy Manufacturing

ThermoGenesis Holdings Inc. offers global infrastructure and proprietary manufacturing technologies to help alleviate some of the challenges that developers of cell and gene therapies face. A proprietary technology like the CAR-TXpress Platform provides high efficiency cell manufacturing for cGMO grade cell therapies. This state-of-the-art technology increases manufacturing efficiency while reducing manufacturing costs. Our BioArchive System also provides control rate freezing and storage solutions for cell samples. It is the number one ranked cryostorage system for clinical grade cell therapies and offers the best cell viability in the industry. Our BACS technology is a novel, closed, semi-automated system that allows the selection of specific cell types. This is an excellent solution for cell and gene therapies that are in the development phase. 

To learn more about our state-of-the-art technology, contact us today.