What is Cell & Gene Therapy?
Here we deep dive into what cell & gene therapy is, companies & organizations currently paving the way, as well as the future of cell & gene therapy.
Table of Contents
What are Cells and Genes?
As the building block to living organisms, cells make up our bodies and perform a wide variety of different functions, all while being the smallest unit of the body. Depending on the tissue type, cells can be more or less specialized.
Genes are located deep within our cells, and carry genetic information in the form of DNA that provide the ‘code’ for our physical characteristics, and biological functions. It is important to note that some of our physical traits are not determined by genes alone, and can be caused by a combination of genes and an individual's environment. Genes are inherited from our parents, meaning individuals usually carry two of each gene.
When our DNA is altered, which can include it being missing, damaged, duplicated, and more, it can cause a genetic disease or disorder.
What is Gene Therapy?
In the grand scheme of pharmaceutical treatments, cell & gene therapies are relatively young methods, meaning there are respectively less treatments available on the market when compared to more traditional treatments. Gene therapy is, in essence, altering, replacing, adding, or removing a gene in order to target and treat a disease or disorder.
Gene therapy is particularly beneficial as many genetic disorders require lifelong treatments and management to maintain a quality of life, whereas gene therapy targets the problem in the gene at the most fundamental level, in hopes of significantly improving or curing the disorder altogether. Although gene therapy is incredibly promising, someone considering gene therapy must take into account the possible negative aspects, including intense side effects, the body's rejection of foreign or altered genes, and incredibly high costs of treatment.
In Vivo vs Ex Vivo Gene Therapy
An important thing to note about gene therapy is that it is usually conducted with one of two main methods: in vivo, or ex vivo.
Due to the complicated nature of cell therapy, a patient's cells are sometimes extracted, with alterations to the gene or genes being done in a lab, then transplanted back into the individual receiving treatment. This is called ex vivo therapy. In comparison, in vivo therapy is done when the corrected or altered genes are delivered directly into the patient's body.
What is Cell Therapy?
Instead of altering DNA or a genetic code, cell therapy is done by transplanting, altering, or resorting sets of human cells to treat an ailment. Cell therapy is actually a relatively old treatment, with some common examples being used as far back as the first recorded blood transfusion in 1665, or more recently, the first bone marrow transplant in 1956. Following those major advancements, cell therapy has since evolved to involve a wide range of cells and treatments.
Cell transplants can be autologous or allogeneic. Autologous cells are harvested from the patient, making them their own donor after their cells are altered. Allogeneic cells are transplants from a universal or other applicable donor. For example, the bone marrow transplant mentioned above involved bone marrow being harvested from one identical twin, and given to the other twin.
As mentioned with gene therapy, there are a variety of possible negative outcomes when receiving cell therapy that must be acknowledged and considered, including cost, side effects, and rejecting cells from a foreign body.
Effective Solutions for Cell and Gene Therapy R&D
Due to the complicated technical nature of cell and gene therapy, it is important to use high-quality and effective solutions when developing and testing. High Purity New England (HPNE), a leading provider of bioprocessing solutions, carries a wide range of products ideal for taking your cell and gene therapies to the next level.
For single-use applications, look to their HPConnexx™ Single-Use Assemblies. With a dedicated team of engineers, brand agnostic designs, and extensive material compatibility resources, HPConnexx™ can be customized to your exact specifications. Additionally, there is no minimum order quantity, so the assembly quantity can be tailored to your needs.
Their FlowMaxx Pro 30 Quaternary Diaphragm Pump is the ideal size for cell and gene therapy applications, with flow rate capabilities of 1mL/min to 500mL/min. It has the capability to integrate both flow and pressure sensors, and features an enlarged HMI touch screen for simple, user-friendly navigation.
When conducting a hollow fiber screening process, look to their MicroMaxx Lab-Scale Screening System, which can be run in automatic mode in order to save time, and your thumbs.
Ensure dynamic separation control for all of your tangential flow filtration (TFF) applications while maintaining a small footprint with their OptiMaxx 30/30 TFF System. It provides the ability to configure process automation using many customizable, pre-programmed UF/DF phases, and can digitally store recipes within the device's memory. With open architecture, OptiMaxx can be tailored to your specific needs.
Finally, HPNE carries the Q-Drive-Alpha from Quantex. It is compatible with both microdosing and low flow pumps. With a 7” color touch screen, operators can easily select from the variety of available functions including self-priming, flow, speed, reverse cycling, and dose volume.
To learn more about the full range of bioprocessing solutions available through HPNE, visit their product catalog.
Companies Making Strides in Cell & Gene Therapy
Sarepta Therapeutics
Global biotechnology company Sarepta Therapeutics has made many major advances in the cell & gene therapy space, including their gene therapy ELEVIDYS which was made to treat Duchenne Muscular Dystrophy. ELEVIDYS was approved by the FDA as of June 2024. In addition to this gene therapy, Sarepta also has multiple LGMD gene therapy development programs, and are developing a gene therapy treatment for Charcot-Marie-Tooth disease.
Bristol Myers Squibb
As of 2020, Bristol Myers Squibb (BMS) had already started looking into cell and gene therapies in a variety of categories, including advancing science in and piecing together cell therapy, and discovering and developing CAR T cell therapies. Approved in March of 2021, BMS launched Abecma, a gene therapy that is used to treat multiple myeloma. It is now also approved in the European Union and Japan. Abecma utilizes your T Cells and adds CARs, or receptors, which help the CAR T cell find and fight your multiple myeloma cells.
The Future of Cell & Gene Therapy
Cell & gene therapy is expected to experience massive growth as new treatments are discovered and approved. According to Deloitte, the market is expected to grow to $19.9 billion by 2027. For comparison, the market was valued at $5.8 billion in 2022. When looking further to the future, the projected value of the market in 2033 is expected to reach up to $97.33 billion. This would result in a CAGR of 19.22% from 2024 to 2033.
As for specific ways that cell and gene therapies will evolve, as many of the therapies are young and yet to be approved, it may be a while before we know the full extent of capabilities these therapies have in store.
Organizations for Cell & Gene Therapy Professionals
There are a variety of organizations that aim to provide a space for cell and gene therapy professionals to stay up to date on industry trends, news, findings, and treatments, as well as network with one another.
One such organization is ASGCT, or the American Society of Gene + Cell Therapy. Members of ASGCT range from patient advocates to scientists and more. They also boast a diverse range of work settings amongst members. In addition to memberships, ASGCT also holds multiple events annually, including their Annual Meeting, which was held in Baltimore, Maryland this year. Their website also provides outreach and advocacy resources.
Another popular organization for cell and gene therapy is the International Society for Cell & Gene Therapy (ISCT). They boast a shared mission to improve the lives of patients worldwide through effective and safe advancements in cell and gene therapies. Established in 1992, the organization has years of experience in the CGT space, and has a diverse group of members hailing from a range of industries.
Lastly, the CGT Circle, which is a group whose main hub is on their LinkedIn page, is an organization providing a space for women in cell and gene therapy to harness their collective power and experience. They run multiple events throughout the year in various international locations, which can range from networking, to panelist events, and more. To get involved, you can follow their LinkedIn page, or request to join their LinkedIn group.
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About HPNE
As the industry needs grow, High Purity New England, Inc. continues to supply the biopharmaceutical industry with a range of innovative products, from drug discovery and development to fill-finish, including their flagship product, custom single-use assemblies, as well as pumps, sensors, bioreactor systems, storage and handling solutions and other single-use solutions. Along with their own manufactured products for the global market, they are also a distributor for more than 18 brands in North America.
