Cell and gene therapies are rapidly evolving fields in medicine that hold tremendous potential for treating and curing diseases that were previously considered untreatable. From cancer to genetic disorders, these innovative therapies are transforming the way we think about disease and treatment.
In this white paper, we’ll begin with an overview of regenerative medicine, to which both cell and gene therapies belong. Next, we’ll cover the top similarities and differences between cell and gene therapies.
We’ll conclude by exploring the overlapping potential of these exciting new treatments.
Regenerative Medicine: An Overview
Let’s begin by exploring the field of regenerative medicine, to which both cell therapies and gene therapies belong.
What is regenerative medicine?
Regenerative medicine is an interdisciplinary field of medicine that focuses on the development and use of treatments that restore the function of damaged tissues and organs.
Regenerative medicine attempts to recreate, repair, or replace diseased or damaged cells and tissues to treat illnesses and injuries. Instead of treating conditions with medication or surgical excision, regenerative medicine aims to replace damaged or disease-causing cells with healthier versions of themselves.
The ultimate goal of regenerative medicine is to provide new treatments for diseases and conditions that were previously considered untreatable or for which there were limited treatment options.
What does regenerative medicine include?
Regenerative medicine includes a wide range of techniques that aim to replace or regenerate damaged cells, tissues, and organs. This includes treatments at the molecular level, such as the tweaking of chromosomes, as well as the regeneration of blood, muscle, and bone at the tissue level.
Regenerative medicine also includes improvements to biomaterial engineering, artificial organs, and the shifting line where medical devices can interact directly with the body.
What are examples of regenerative medicine?
Since regenerative medicine is driven by rapid technological innovation and improvement, its applications are constantly evolving. An emerging field, it encompasses a number of therapies and treatments with individual targets and processes.
Examples of regenerative medicine include:
- Stem cell therapy
- Tissue engineering
- Cartilage regeneration
- Immunomodulation therapy
Frank Maggiore, Chief Futurist at Apprentice, notes that regenerative medicine is a broad outline.
“Regenerative medicine zooms out, in order to look at things on a large scale.”
— Frank Maggiore, Chief Futurist, Apprentice
This holistic approach means that regenerative medicine isn’t solely focused on alleviating symptoms, but also on repairing and regenerating the underlying causes. The body has its own natural healing processes, and regenerative medicine seeks to enhance and support these processes that are already in place.
Cell Therapies vs Gene Therapies
What are cell and gene therapies?
Cell therapy and gene therapy are two broad subdivisions within the domain of regenerative medicine. Together, the two therapies are changing our entire approach to the way we treat disease.
“On a genetic level, we're still living a hunter-gatherer lifestyle. Cell and gene therapies are changing the game. We're now able to modify our underlying biology, right down to our DNA.”
— Frank Maggiore, Chief Futurist, Apprentice
Both cell and gene therapies have the potential to revolutionize the way we approach and treat a range of medical conditions, from genetic disorders to age-related diseases. These therapies offer a more holistic, individualized approach to treatment than other modalities such as small molecule and large molecule.
Read on to learn some of the key similarities and differences between cell and gene therapies.
How are cell and gene therapies the same?
Cell and gene therapies have a number of similarities, including:
- Modifying biology: Both cell and gene therapies aim to modify the underlying biology of a disease by introducing functional cells or genetic material into the body.
- Personalized medicine: Both cell and gene therapies can be tailored to the specific needs of individual patients, providing a level of personalization in treatment.
- Cutting-edge technology: Both cell and gene therapies represent innovative and cutting-edge advancements in medicine.
- Precise delivery: Both require precise delivery methods to ensure the cells or genetic material reach the target site in the body and achieve the desired therapeutic effect.
- Long-term benefits: Both have the potential for long-term benefits by modifying cells or genetic material that persist in the body.
The fundamental similarity between cell and gene therapies is their method of attack. In both cases, the therapies are designed to help a patient’s own body attack the disease itself through their immune system.
How do cell and gene therapies differ?
At its core, the main difference between cell and gene therapies is their level of complexity.
Cell therapies involve the transfer of cells themselves, while gene therapies go a level deeper by transferring genetic material into targeted cells.
Top differences between cell and gene therapies include:
- Approach: Cell therapy focuses on introducing functional cells into the body to replace damaged or diseased cells, while gene therapy focuses on modifying the genetic material within cells to correct disease-causing mutations.
- Therapeutic agents: Cell therapy uses live cells as therapeutic agents, while gene therapy uses genetic material such as DNA or RNA.
- Delivery method: Cell therapy typically involves transplanting cells into the body, while gene therapy often involves the delivery of genetic material directly into cells through methods such as viral vectors or CRISPR.
- Target diseases: Cell therapy is often used to treat conditions such as cancer, autoimmune diseases, and genetic disorders, while gene therapy is primarily used to treat genetic disorders, but is also being explored for its potential in treating cancer and other diseases.
- Short-term vs. long-term: Cell therapy can provide both short-term and long-term benefits, while gene therapy is typically aimed at providing long-term benefits.
Despite these differences, keep in mind that some therapies can be classified as both cell and gene therapies. For example, since ex vivo gene therapies require altered cells to be inserted, they can also be a type of cell therapy.
“Cell therapies target specific cells to use for treatment, while gene therapy is a functional replacement down to the DNA level.”
— Frank Maggiore, Chief Futurist, Apprentice
The Future of Cell and Gene Therapies
Where are cell therapies headed next?
As technology improves, cell therapies will become even more personalized and effective, with treatments customized for each patient's unique needs.
Innovations in biotechnology and materials science will drive the development of new cell therapies, as well as the optimization of delivery methods for current treatments.
This field is poised for significant growth, as cell therapies become more widely adopted for the treatment of an increasing range of medical conditions. With a focus on treating age-related diseases, such as Alzheimer’s, Parkinson’s, and heart disease, cell therapies have the potential to have a profound impact on global health.
Where are gene therapies headed next?
The future of gene therapy is personalized medicine, with treatments tailored to meet the specific needs of each patient.
The use of gene therapies is expected to become more widespread, with increasing adoption to treat a growing number of medical conditions.
As research shifts towards developing treatments for genetic disorders and age-related diseases, such as cancer, cardiovascular disease, and neurodegenerative disorders, gene therapies have the potential to completely transform the way we approach and treat illness.
How will tech shape the future of CGT?
"Gene therapy products now have the potential to cure intractable diseases, and fundamentally alter the trajectory of many other vexing illnesses."
— Scott Gottlieb, M.D., Former Commissioner of the United States Food and Drug Administration (FDA)
Technology will play a critical role in the future of cell and gene therapies, driving innovation and advancement in both fields.
Biotechnology and materials science will continue to advance, leading to new and improved cell and gene therapies. The use of genetic engineering and gene editing technologies will help to usher in the new era of personalized medicine. The optimization of delivery methods such as nanotechnology, will also be key in the success of these treatments.
As technology improves, cell and gene therapies will become even more effective and widely adopted, transforming the way we approach and treat medical conditions.
Our Featured Thought Leader
Frank is all about future-forward approaches here at Apprentice. In fact, he’s our Chief Futurist! Read on to learn more about Frank’s perspective on the promise of cell and gene therapies.
Frank has worked in the pharmaceutical industry for more than 25 years. Frank’s areas of expertise include a number of fields in tech and life science, including augmented reality, artificial intelligence, and cell and gene therapies.
Frank is the inventor of Single-use Biological 3D Printing, 3D printing within sterilizable bioreactors/mixing containers, Single-use robotics, and the Variable Augmented Reality Marker.
Frank has also invented a method of “Contactless Communications” for secure cloud network computing where IoT devices can securely work with smart glasses, smartphones, or other mobile devices.
What connects all of Frank’s inventions and innovations is his core motivation: pushing technologies to the limit. Frank spends his time developing disruptive technologies, rapidly building functional prototypes, and determining how they can apply to various industries.
Frank’s take on the future of CGT
“Cell and gene therapies hold enormous promise to advance the way we treat disease. But that’s just the start — they could potentially offer a new level of control over our own cognition and the drivers that govern our behavior, much in the way that we are finding out how the microbiome in our gut affects behaviors as well.
By modifying the genetic material that controls our biology and affects our brain function, these therapies have the potential to address both the underlying causes of diseases and disorders, such as using CAR-T cell therapies to attack immune system cells causing auto-immune disorders like multiple sclerosis, as well as aspects of our behavior, cognition, and mental well-being.
While this is still uncharted territory, it’s an important reminder that the current applications of cell and gene therapies are just the beginning of what’s to come.”
The complexities of cell and gene therapies require sustained innovation as they are explored and implemented. The possibilities have only begun to be explored, much less implemented.
This kind of creative learning needs novel technologies and workflows. Companies pursuing such advanced therapies need an advanced platform to help them move from discovery to patent delivery.
Want to find out more about digital solutions for CGT manufacturing? Give us a call!
Reach out to us today to learn how Tempo is furthering the development of cell and gene therapies.