Your source for answers to the complex challenges of cell and gene therapy development.
We recently sat down with Maryland Franklin, Vice President and Enterprise Head of Cell & Gene Therapy at Labcorp Drug Development, to discuss key considerations for developing cell or gene therapies in today’s quickly expanding pipeline.
What excites you about the cell and gene therapy landscape?
There’s never been more hope within the cell and gene therapy industry as there is today. Over the last five years or so, we’ve really seen an exponential increase in both cell and gene therapy drug development. I believe this uptick in activity is due to the convergence of the science, technology, funding and the involvement of large pharma.
For example, as we entered 2021, the Alliance for Regenerative Medicine Annual Report1 highlighted that approximately 1,100 organizations globally developing cell-, gene-, or tissue-based therapeutics. In 2020, almost $20 billion of global financing was raised. There are about 1,200 clinical trials ongoing, with more than half in Phase II, and about 150 in Phase III, along with over 1,300 assets in preclinical development that are really continuing to drive this pipeline.
Can you discuss some general considerations for developing cell or gene therapies?
There are a number of considerations that one has to take into account when developing cell or gene therapies. First and foremost is that while cell therapy and gene therapy have some overlapping similarities, they also have many differences from each other. Even within those broad categories, there are really specific nuances depending on the type of cell or gene therapy being developed.
What are some specific considerations for cell therapies?
In general, for a cell therapy, the source of material is very important, whether it’s coming directly from the patient themselves or from a donor. Sponsors must also consider the supply chain and the logistics to coordinate the creation of these very personalized or precision medicines being developed. In many cases, one drug is being developed for only one patient.
How about for gene therapies?
On the gene therapy side, there’s several considerations, such as: the use of viral vectors; the approaches for gene editing or modifications; how to build the safety and toxicology studies; how to evaluate immunogenicity; the analytical tools that are needed, and the translation of those preclinical studies into first in human studies. I think drug development teams must gauge the short- and long-term safety potential of these viral vectors.
Tell us about the role of the patient in these studies.
Managing the clinical trial patient and their experience through the cell or gene therapy process is important, as it is quite different from the patient experience for traditional therapy trials with small molecules or other biologics. A cell therapy or gene therapy requires increased patient engagement and support during a much longer treatment process, along with potential follow up for 5-15 years to meet regulatory requirements.
Do you have any recommendations for the development of cell and gene therapies?
We heard recently from Peter Marks from the FDA about the need for consistency within the development of these types of products, as reported in Biopharma Dive2. We have to consider which attributes need to be evaluated, how to establish the methods and deploy those assays appropriately through the nonclinical and clinical phases of development. There’s also an evolving regulatory landscape. Drug development sponsors need to keep abreast of what’s happening in this global environment.
I think the best strategy to overcome the major development challenges is to plan ahead. Find a reputable partner to work with, like Labcorp Drug Development, who truly understands the challenges across all phases of development.
What can Labcorp Drug Development offer to sponsors?
We have the ability to run bioanalytical and bioassays related to drug substance and drug product during the drug development process for both preclinical and clinical studies. We also have an entire group that specializes in regulatory considerations, market access and commercialization for drug development, including cell and gene therapies. We’re also expanding capabilities at some of our sites, including our Madison, Wisconsin site to help increase the capacity for safety and toxicology studies that are utilized in the important nonclinical development of cell and gene therapy.
As a strategic partner, we help sponsors think ahead. We have dedicated infrastructure of facilities and personnel globally that are well versed in these types of therapeutics. We also have established partnerships to help solve many of the challenges within cell and gene therapy, such as our partnership with Blood Centers of America and a partnership with TrakCel, both aimed at helping supply chain for cell therapies. In addition, we offer true end-to-end services that includes the pharmacology and IND-enabling work, regulatory guidance, clinical development, long-term follow up, commercialization, market access and laboratory services.
Sponsors can work with our cell and gene therapy experts across our business units, as they are embedded into every functional area within our organization. These specialists help provide the needed expertise to assist with individual studies and also serve as key partners when customers are looking for a more programmatic outsourcing approach that’s aimed at reducing risk and shortening timelines.
What are your thoughts about the future of the cell and gene therapy development landscape?
There are so many fantastic areas of scientific research and innovation in this space, and I think these areas will really impact the development of cell and gene therapy in the next three to five years. I do believe that as an industry we will overcome the challenges around allogeneic cell therapy. This would reduce complexity of the studies; it would make the logistics around CAR T-cell therapy a bit less daunting — and it could potentially lower the cost of therapy.
I also think there will be continued advancements in gene editing technologies, in single cell sequencing capabilities and approaches to reduce immunogenicity of AAV capsid proteins, which really could have a big impact on this industry. In addition, our approach to integrating a more patient-centric, decentralized trial solution will be crucial as we move through cell and gene therapy clinical trials and long-term follow up with patients.
Propel your cell and gene therapy development by visiting drugdevelopment.labcorp.com/cgt
1Alliance for Regenerative Medicine. 2020: Growth & Resilience in Regenerative Medicine ANNUAL REPORT. The Alliance for Regenerative Medicine. https://alliancerm.org/sector-report/2020-annual-report/
2Pagliarulo, N. (Ed.). (2021, May 19). FDA seeking more consistency from cell, gene therapy developers, top official says. BioPharma Dive. https://www.biopharmadive.com/news/fda-marks-gene-therapy-consistency/600445/.