Bioprocessing Summit 2016: Are Novel Therapies on the Path to Standardization?

By Admin
Posted 09 / 20 / 2016

This year’s Bioprocessing Summit in Boston brought a lot of bright minds together to discuss ways to navigate the increasingly complex nature of bioproduction. Brand Name’s Dr. Julie Murrell, Head of Cell Therapy Bioprocessing, participated in this discussion by presenting “Go Beyond: How to Make Novel Therapies a Reality in Tomorrow’s Complex Biopharma Landscape” as part of the Cell and Gene Therapy Production track.

Dr. Murell began her talk by explaining the difference between novel therapies vs. what we now call “standardized” therapies. The type of therapy can often be defined by its mode of action:

Cell therapies: the cell itself generates the therapeutic action.

Gene therapies: a specific gene inside the patient is replaced to achieve the therapeutic effect.

RNA: variants of gene therapies.

CAR-T and Ex-vivo gene therapies reside in a gray area that combines both cell and gene therapy modes of action. On the other hand, therapies that were considered “novel” 25 years ago are now standardized. Small molecule drugs and recombinants are produced at large scales globally and have, for the most part, standard process and manufacturing templates in place as well as regulatory guidelines. So are novel therapies on the path to standardization?

The momentum is certainly there. Unlike the 1990’s when headlines like “Has Gene Therapy Stalled?”1 littered the media following patient deaths, we are now seeing headlines like “Gene Therapy Coming of Age?”2 in the media. Scientists are starting to see high rates of clinical efficacy in cancer patients with these therapies, and with success comes excitement and further investment in science which eventually leads to more innovation. And the cycle continues.

Dr. Murrell also presented some interesting findings from the recent biopharma report3 published by the Economist Intelligence Unit. According to the report, 48% of biotechs reported novel therapies as their main focus for drug development over the next five years. Recombinants are still part of biopharma’s growth strategy in the years ahead, but novel therapies are clearly top of mind as these companies think about future growth plans.

Despite the excitement surrounding the science, process engineering for these therapies is often overlooked. Biopharma needs a cost-effective, reproducible, and scalable manufacturing process. You unfortunately cannot apply the manufacturing process of recombinants to cell and gene therapies. In fact, manufacturing challenges also differ based on the type of therapeutic:

Novel Therapies: Manufacturing Challenges

In addition, any medium chosen for cell therapy applications should be effective across all platforms in the anticipated manufacturing process, and this should be considered early on to prevent costly comparability studies due to changes in later phase clinical trials. Optimizing the process parameters and media formulation is key, and in order to stay ahead of the curve as an innovator, it is critical to have a true understanding of the assays and how they can be applied across different platforms and media formulations. Many of the assays are based on the original discovery platforms, and those assays and analytics may not be the best to use to assess performance as you move to a commercially viable platform.

The complexity that these new therapies bring to the manufacturing process is coupled by the emphasis biopharma is placing on risk management in the coming years. According to the EIU survey, 56% of biopharma plan to mitigate risk by enhancing internal capabilities, while almost half of the survey respondents indicated that they will invest in outside expertise and engage in local partnerships. The industry understands the importance of partnering to enable success of these groundbreaking molecules which may explain their optimism in bringing these new therapies to market: they don’t have to do it alone.

Find out more about novel therapy development and cGMP cell therapy manufacturing at brand-name.

  1. “Has Gene Therapy Stalled?” Oct 9, 1995, Time Magazine:,9171,983532,00.html
  2. “Gene Therapy Coming of Age?” July 11, 2013, The Scientist:
  3. “The Changing Biopharma Risk Equation” June 2016, The Economist Intelligence Unit.