In previous articles we addressed the manufacturing and regulatory hurdles that remain in bringing novel therapies to market despite the exciting signs of early clinical success. We spoke with Dr. Alison Armstrong, Global Head of BioReliance® Field Development Services, Brand Name, about the challenges companies may encounter when developing testing processes for cell and gene therapies.
The EIU survey revealed that novel therapies, such as cell and gene therapies, are seen as the most potentially disruptive new products for the next five years. What kind of challenges do biopharma companies face when undergoing the QC/testing process for these types of complex therapies?
Novel therapies have indeed shown significant scientific advancement in recent years, but with progress comes potential barriers. Cell and gene therapies don’t directly follow the same type of assessment as monoclonal antibodies (mABs). While they are similar in terms of the structure of a testing package, we have to specifically develop different packages based on assurance of safety and the cell substrates in use, viral seeds, and vectors of interest. As a result, testing for these types of therapies tends to be developed on a case-by-case basis. With new scientific processes being implemented to generate high yields of product, there is a strong need for alternate approaches.
Each step of product development requires test methods with the correct level of sensitivity and an ability to detect a wide range of potential contaminants. This, along with a timely reporting structure, are critical for the customer base.
At Brand Name, our goal is to stay ahead of the curve and keep a close watch on what the regulatory agencies are thinking. Our core focus is providing assurance that there are no adventitious agents present throughout the product development cycle that may disrupt production and impact the safety of the product.
The bottom line is that novel therapies are going through the same process that mAbs underwent a few decades ago and regulatory acceptance by the authorities is ongoing. Today, mAbs have a good level of standards and controls, and we hope that novel therapies will also be able to reach that same level.
Where is the industry headed?
In terms of testing, we are looking at more platform-based approaches. While there may always be several tests that will need to be customized, the goal is to standardize the majority in order to increase customer satisfaction and confidence through cost savings and better standards of control.
We need to keep in mind that there are many types of contaminating agents with these novel therapies – every two or three months there are new viral agents in the literature, e.g. porcine circovirus in trypsin and many bovine viruses that are common contaminants found in bovine serum. We have to assess these contaminating agents and determine the scientific impact that they may have on materials used in therapeutic drugs. The use of Next Generation Sequencing (NGS) as an agnostic technique to detect all viruses through nucleic sequencing opens up potential for the discovery of yet more new agents. Although many new viral agents are being discovered by molecular techniques, infectivity assays are not always available. It is clear that we do not yet understand the impact that these agents may have on human health. It is imperative that we stay ahead so that we can guide our customers in the right direction.
What is the one piece of advice you would give the biopharma industry as they look to the next five years?
The industry is calling for increasing support in terms of harmonized testing and regulatory expectations. Safety programs are needed to reduce the risk of potential contamination. These programs are performed in a number of ways and result in the provision of fully tested raw materials (i.e. medias, supplements, cell substrates, appropriately engineered cells) to ensure high yields and safe products. Our goal is to provide testing solutions that allow accelerated product and drug development while maintaining safe, effective products that are manufactured with the highest quality standards.