Roundtable Part 1: What, in your opinion, is the current state of cell and gene therapy in the pharmaceutical industry?

Wednesday, November 30, 2022

Andrew Schaefer, MBA

Manager, Analytical Method Development & Validation, Eurofins BioPharma Product Testing

The modalities provide some of the greatest hope to treat the most challenging disease to afflict humanity, but in terms of results, only a tiny fraction of the clinical candidates have found approval.

Berangere Tissot, Ph.D.

Director, Biochemistry Method Establishment and Biologics Characterization, Eurofins BioPharma Product Testing

From a technical and methodology standpoint, the new modalities are at the same stage as the monoclonal antibodies were 20 to 30 years ago: there is no consensus for mature and established methodologies for any of the CMC assays. There is a lot of information sharing and collaborative work performed between the industry and the regulatory agencies around the world to gain knowledge on how to control the quality of these products which hopefully will bridge that gap relatively quickly.

Stacie Fichthorn

Senior Manager, Cell/Gene Therapy and Viral Clearance, Eurofins BioPharma Product Testing

Eurofins BioPharma Product Testing is seeing a surge in contract laboratory needs as new products are being pursued. There is significant growth in R&D, which in turn is fueling collaborations early on in the drug development process. The need and demands for CGT will continue to increase in the months and years ahead. Treatment of cancers using CAR-T has served as inspiration to the scientific community. We are seeing first hand that individuals are driven to be part of the process- they want to know that they had an impact on the approvals and release of novel therapies.

Mike Stewart

Chief Operations Officer, Matica Biotechnology, Inc.

This industry is an industry of attrition, where you look at a disease state, you test that disease state, you see if the proof of concept is there and, if it is, you move forward. This is what sponsors desire to do, to move that therapy forward, whether that is gene therapy or cell therapy, and to move from the research phase into the clinic phase as quickly and as efficiently as they can. There are many technologies out here today and there are many more in the pipeline that this industry can take full advantage of. What we can do now, we weren’t able to do five years ago. Historically, we haven’t been able to measure everything that we are measuring today.

Peter Marks M.D., Ph.D.

Director, Center for Biologics Evaluation and Research (CBER), FDA

The scientific field for cell and gene therapy products is fast-paced and rapidly evolving. The advances that we are seeing since the first clinical studies in the U.S. that employed human gene transfer for therapeutic purposes in the 1990s are exciting. Currently, there are over 1300 active gene therapy investigational new drug (IND) applications on file with FDA.

Although we can’t predict future growth, we do anticipate continued scientific advancements and technical innovation in this field. Over the next several years, I believe that we will witness a significant evolution in this area, in part because more researchers and product manufacturers are dedicated to investing in the development of gene therapies. Increased availability of these products will result in additional treatment options and potential cures for many devastating and intractable illnesses – in particular, for patients who previously had no other treatment options.

Rob Jones

Vice President, Global BioServices, Cryoport Systems

The cell and gene therapy industry is no longer a fledgling, research focused discipline, but a tangible, and growing sector of the pharmaceutical industry with a strong pipeline of products in development as well as several commercially approved therapies – many of which for the treatment of previously untreatable conditions. The growing number of IND and BLA filings also prove that the industry has established itself as a recognized modality of medicine.

George Buchman, Ph.D.

Research Fellow, Product Development, Catalent Cell and Gene Therapy

These are exciting and pivotal times for the field. It has only been during the last 10 to 15 years that the industry has made significant strides in leveraging the full potential of cell and gene therapies, having only just begun to translate research into viable products. Indeed, so far, only a handful of products have successfully made it through clinical development to approval. These therapies have the potential to treat or even cure thousands of diseases and improve the lives of millions of patients and their families. However, the development and manufacture of these therapies is currently very complex and expensive and will require significant improvements if we are to produce treatments more efficiently, and so make them more accessible to the wider pool of patients that need them.

Until relatively recently the field has been led by research and development, and as an industry we are only just scratching the surface of what can be achieved with cell and gene therapies. As we learn more and demonstrate the safety and efficacy of these treatments, development and manufacturing partners will become increasingly critical in overcoming challenges with CMC, scale-up, and process optimization. We will continue to develop therapeutics that address current challenges, including pre-existing immunity and immunogenicity, improved dosing and tissue targeting, and supply.