Cellular (cell) and gene therapies are part of a personalized medicine growth market for treating, preventing and curing genetic diseases and cancers. ClinicalTrials.gov, a database of privately and publicly funded clinical studies conducted around the world, listed more than 671 clinical trials that were underway as of June 2020. More than $23 billion in investments has been announced for acquisition, expansion or greenfield sites for the period of 2019-2021.
What are Cell and Gene Therapies?
Cell therapy and gene therapy are overlapping fields of biomedical research and emerging treatment. Both approaches seek to treat the disease by targeting its cause. They are collectively known as Cell and Gene Therapy (CGT).
Cells are the fundamental building blocks of the human body and all living organisms. Genes, found within cells, are the basic units of heredity. They contain tiny sections of Deoxyribonucleic Acid (DNA), a complex molecule that contains a unique genetic code for each person. In addition to genetic information, genes contain instructions for making proteins, which instruct cells on how to work and grow. Genetic diseases occur when DNA sections are deleted, substituted or duplicated. Any changes in the DNA are called genetic mutations. Sometimes genetic mutations are inherited.
Cell therapy involves the transfer of restored or altered cells into a patient. Cell therapy products include cellular immunotherapies, cancer vaccines, and other types of both autologous (originating from a patient) and allogeneic (originating from a donor) cells for certain therapeutic indications, including hematopoietic stem cells and adult and embryonic stem cells.
Gene therapy involves the transfer of genetic material, usually in a carrier or vector, and the uploading of the gene into the appropriate cells of a patient’s body. Human gene therapy seeks to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use. Gene therapy holds promise to treat a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS. The process involves replacement, inactivation, or introduction of genes into cells.
Some therapies are considered both cell and gene therapies. These therapies work by altering genes in specific types of cells and inserting them into the body.
One example of cell and gene therapy that might be familiar to people is CAR-T. Chimeric Antigen Receptor T-cell
(CAR-T) therapy is a specific application of CGT. In 2017 the US Food and Drug Administration (FDA) approved the first gene therapy drug for (CAR) T-cell treatment of B-cell precursor acute lymphoblastic leukemia. In this case, a patient’s T cells are reprogrammed to attack tumor cells. The process is described as patient-to-bench-to-patient because the plasma is collected from the patient, sent to a facility for modification, and then returned to the patient. The therapeutic is designed and grown in a bioreactor that is assigned to a single, often very ill, patient. These types of therapeutics are more complex than traditional therapeutics and pose additional challenges to produce.
The Cell and Gene Therapy Landscape
In the United States, The Center for Biologics Evaluation and Research (CBER) regulates cellular therapy products, human gene therapy products, and certain devices related to cell and gene therapy. CBER uses both the Public Health Service Act and the Federal Food, Drug and Cosmetic Act as enabling statutes for oversight.
In Europe, the European Medicines Agency (EMA) regulates and provides guidance on advanced therapy medicinal products (ATMPs)
There are currently many FDA-approved Cellular and Gene Therapy Products, the full list is available on the FDA’s website.
Cell and gene therapies present a new arena of medicine. Most of the advances and technology come from academia or the research laboratories. The processes to scale up these advances and scale them out to manufacturing are new, time consuming and very expensive. In addition, new regulations are being developed for this emerging market segment.
CGT Industry Challenges
Many Biotech and Pharma companies are still new to this technology and are investing in numerous areas, including how to scale, develop new processes, become more agile and flexible, establish appropriate batch genealogy and more. Some of the key topics being explored are novel product development, clinical trials, quality, regulatory, manufacturing, supply chain and data analytics.
The following are the major categories of challenges currently being faced by the cell and gene therapy industry:
- Novel Therapeutic Discovery
- Patient Recruitment and Retention for Clinical Trials
- Quality by Design
- Market Approval & Regulatory
- Batch Genealogy
- Flexible, agile and modular manufacturing and Supply Chain
- Data Analytics
Cellular and Gene Therapy Digitalization Transformation
With the extensive promise of cell and gene therapies, we believe the above challenges can best be addressed through digital transformation. Commonly understood industry processes for small-scale molecules and biologics cannot solve CGT’s challenges without an Industry Renaissance approach, which will produce real transformative business changes. Cell and Gene Therapy companies are actively looking for innovative software and process solutions and are collaborating with different Industry organizations like BioPhorum to drive new ways of working.
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Dassault Systèmes has several solutions that address many of the challenges described, including:
- Designed to Cure
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- ONE Lab
- Collaborative Scientific Innovation
- MEDIDATA solutions on Precision Medicine
For additional information, visit Dassault Systemes’ Life Sciences & Healthcare industry homepage.
 American Society of Gene & Cell Therapy: Different approaches. Available at: https://patienteducation.asgct.org/gene-therapy-101/different-approaches
 US Food and Drug Administration (FDA): What is Gene Therapy? Available at: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy
 Mayo Clinic: Gene therapy. Available at: https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619
 Novartis: What is cell and gene therapy. Available at: https://www.novartis.com/about/innovative-medicines/novartis-pharmaceuticals/novartis-gene-therapies/what-cell-and-gene-therapy
 European Medicines Agency: https://www.ema.europa.eu/en/human-regulatory/overview/advanced-therapy-medicinal-products-overview
 US Food and Drug Administration (FDA): Cellular & Gene Therapy Guidances. Available at:https://www.fda.gov/vaccines-blood-biologics/biologics-guidances/cellular-gene-therapy-guidances