With the dawn of the new decade, the healthcare realm ushers in innovative research and treatments that are expected to leverage cell and gene therapies, significantly improving the lives of patients and fulfilling the promise of personalized medicine. The future of biomedical science is fueled by the growth of these therapies, bringing multiple approved products to global markets along with an increase in the number of clinical trials. Despite advances in next-generation cell and gene therapies across the US and Europe, biotech and pharmaceutical companies continue to face fundamental challenges when it comes to successfully and efficiently developing, manufacturing, and launching a cell or gene therapy product to the market.
Set against a highly competitive backdrop of developing cutting-edge therapies and getting them through development quickly to take advantage of patent exclusivity, pharmaceutical and biotech enterprises must navigate a gauntlet of expensive and sophisticated hurdles to achieve success. Moreover, the commercialization of cell and gene therapy products has to keep up with rapid advances in manufacturing technologies, an evolving regulatory landscape, complex mechanisms of action (MOA), and inherent variability in starting cellular material. A keystone is the requirement for analytical testing to ensure the stability and potency of a product before it becomes available in the clinic. This analytical testing not only verifies the biochemistry of the therapy, but also enables better-quality formulations, getting drugs to market faster, and risk reduction.
Absorption Systems seeks to improve the discovery and development landscape for its clients.
As a Pennsylvania-based full-service nonclinical CRO, Absorption Systems provides innovative services and solutions to the pharmaceutical, biotech, medical device, and regenerative medicine industry, accelerating product lifecycle—from concept to approval. Laser-focused on analytical testing—be it for unique therapies yet to be approved or launched products—the company develops validate-able test systems to help therapeutic products meet regulatory requirements.
In the cell and gene therapy space, Absorption Systems specializes in potency assay development, allowing clients to evaluate multiple assays rapidly, generate data on product stability and consistency, and collect data to support correlation studies if needed. To understand a product’s ability to work in patients, Absorption Systems develops assays to understand how the product works in vitro. By developing analytics-enabled, definitive, cell-based test systems that mimic or demonstrate the mechanism of the product’s action, the company empowers its clients by testing a product’s potency outside of humans. The other avenue offered by Absorption Systems is to use animals in which the disease state is created in vivo, followed by applying the cell or gene therapy to determine if and how well it works.
“We develop the test system with the client and assist them from assay development through the GMP process to where they have a fully validated assay at the end”
“In an ideal situation, we would use cell-based assays since they tend to be faster, more controllable, less variable, and scaleable,” says Patrick M. Dentinger, CEO of Absorption Systems. To understand potency outside of humans, the company employs several rigorous steps, leveraging experts across its three sites in the US. “The first step is to identify a biological system that allows us to understand the transduction efficiency and expression of the delivery system. As we parallel the client’s product development, the assay parameters are optimized to understand the mechanism of action of the delivery system and the expression of the genetic payload. Ideally, the ultimate cell type used in the assay system would be of human origin and resemble the desired target tissue in humans. This can be a significant challenge as not all tissue types have corresponding cell lines that we can work with,” says Dentinger.
Once the gene has been successfully delivered inside the cell, mRNA expression and, ultimately, protein production and function (or loss thereof) are monitored. Next, gain or loss of protein function must be demonstrated at robust, consistent levels between assay runs. In the best case, cellular assay development is complicated, as working with cell culture comes with multiple constraints. Additionally, as the study sponsor gains an understanding of their therapeutic, which may lead to slight modifications, analytical testing often has to evolve to reflect those changes.
As a result, Absorption Systems trains all its researchers on Standard Operating Procedures (SOPs) and employs rigorous Quality Control measures. “We develop the test system with the client and assist them from assay development through the GMP process to where they have a fully validated assay at the end,” says Dentinger. “By understanding the practicality of running that assay over and over again, the expertise we bring to the table is not in just developing the assay, but also ensuring that it runs consistently and efficiently,” he says.
The uniqueness of Absorption Systems stems from the experience and expertise it brings to the table in identifying where the real bottlenecks are. The company’s unparalleled knowledge and expertise have been instrumental in the regulatory approvals of numerous unique therapies over its 25-year history. As a testament to its Quality procedures, its sites have been audited and certified by multiple global regulatory agencies and standards organizations.
The many aspects—cell-based assays, gene expression assays, gene delivery systems, and more—of analytical testing in the CRO space have empowered Absorption Systems to expand its knowledge and capabilities to address the unique challenges of cell and gene therapies. To that end, the company is identifying better tools and skills for developing assays faster. “With much room for innovation and optimization available in the analytical testing area, we continuously improve our processes based on learnings from various other projects. The evolution of “best practices” is the result of working on many diverse projects and problems, and is a never-ending process. The accumulation of these practices is of great value to our client base,” concludes Dentinger.