Cell and Gene Therapy (CGT) represents a revolutionary approach to the treatment of rare and complex diseases. Cell therapy is the transfer of live cells, into a patient to lessen or cure a disease using cells from the patient or a donor. Cell therapy can be used to treat a variety of conditions, including cancer, autoimmune diseases, and neurological disorders1,4,5.

Gene therapy alters faulty genes or replaces them with healthy ones to correct genetic disorders at the molecular level. Unlike traditional treatments that often focus on managing symptoms, gene therapy targets the underlying cause of a disorder, offering a potential one-time curative intervention that could radically improve the quality of life for patients. With nearly all gene therapies designed to provide durable effects from a single administration, these cutting-edge therapies are considered transformative, particularly for rare and genetic diseases that have long lacked effective treatment options.

Currently, there are over 30 FDA-approved cell and gene therapy treatments5 in the United States, with more than 4,000 therapies at various stages of development. While prevalence and incidence rates are low today, experts predict the treatable population will increase 11.5 times over the next five years, reaching nearly 50,000 patients in the US alone1,5. This growth is driven not only by the increased pace of FDA approvals for more prevalent diseases but also by greater access to qualified providers and facilities.

Cell and gene therapies are closely related and often overlap. In some cases, both are used together to treat diseases. For example, cell-based gene therapy involves removing cells from a patient, modifying them using gene therapy, and then reintroducing the modified cells into the patient’s body. Treatments for Duchenne Muscular Dystrophy (DMD), certain cancers, and spinal fusion are just a few examples3,4.

How Cell and Gene Therapy Will Transform Healthcare in the Next Decade

Cell and gene therapy are poised to radically transform healthcare over the next decade by offering potential cures for currently untreatable diseases, such as genetic disorders, certain cancers, and neurological conditions. These therapies allow for more targeted and potentially life-changing treatments. The ability to address the root cause of diseases, rather than simply managing symptoms, could lead to a paradigm shift in medical treatment. Conditions like sickle cell anemia, cystic fibrosis, Parkinson’s disease, and even HIV may benefit from these breakthroughs. This shift could foster a focus on preventative and curative approaches, moving away from the current treatment protocols that primarily manage symptoms.

These therapies have already caused significant disruptions in the pharmaceutical industry, pushing beyond traditional methods of disease management to fundamentally curative approaches. In the short term, more than a dozen new therapies could gain approval in 2024, including treatments for multiple myeloma and leukemia. In 2025, new treatments for hemophilia A and cutaneous melanoma could be approved. By 2026, there is potential for gene therapies targeting wet age-related macular degeneration and knee osteoarthritis, a condition affecting millions1.

  • Advancements in Cancer Therapy2,3,4: CAR-T cell therapy uses a patient’s immune cells to specifically target and attack cancer cells, creating a personalized approach that could be more effective.
  • Regenerative Medicine2,3,4: Stem cell therapies are being explored to regenerate damaged tissues and organs, offering potential treatment options for conditions like heart disease, diabetes, and neurodegenerative diseases.
  • Personalized Medicine2,3,4: Cell and gene therapy may result in highly customized treatments tailored to an individual’s specific genetic makeup.
  • Improved Treatment Outcomes2,3,4: Patients may no longer need to manage symptoms of chronic diseases, but rather address the underlying genetic causes, providing long-term and potentially curative solutions.

The Future of Treating Chronic Conditions

In the next decade, cell and gene therapies may expand beyond rare genetic conditions and cancers to include areas like cardiology and neurology, including high-profile diseases such as ALS and coronary artery disease. The prospect of next-generation viral-vector therapies for neurodegenerative conditions like Parkinson’s disease suggests the possibility of curing these lifelong diseases rather than simply managing them. Gene therapy could also disrupt transplantation by reducing, or even eliminating, the need for donor organs. Therapies could enable patients’ own cells to regenerate damaged tissues, bypassing immunosuppression or the need for transplantation entirely offering a groundbreaking alternative to transplants and potentially alleviating the donor shortage crisis.

Targeted Cell and Gene Therapy: In Vivo and Ex Vivo Approaches

Both in vivo and ex vivo therapies will play crucial roles in the future of cell and gene therapy. In vivo therapies involve directly administering a therapeutic agent into the patient, allowing for gene modification within the body to treat diseases affecting complex tissues and organs. This approach holds promise for treating conditions like heart disease and central nervous system disorders. In contrast, ex vivo therapies involve removing cells from a patient or donor, editing those cells in a controlled environment, and reintroducing them into the patient. This method has been particularly effective in CAR-T cell therapy for certain cancers, offering precise gene editing in a controlled setting.

As these techniques mature, they will expand into areas beyond oncology and hematology. For instance, 51% of current cell therapy pipelines are focused on CAR-T therapies, while other areas, such as RNA therapies and non-genetically modified cell therapies, are rapidly growing to address conditions ranging from pancreatic cancer to Duchenne Muscular Dystrophy1.

Systemic and Economic Challenges of Scaling Cell and Gene Therapy

Cell and gene therapies are inherently complex, and large-scale adoption requires healthcare systems to navigate both logistical and economic challenges. With over 4,000 therapies currently in development, 650 of which are in Phase II or beyond1, the healthcare ecosystem must adapt quickly to accommodate a surge of new treatments. Key factors include:

  1. Regulatory Adaptation and Oversight4: As therapies approach the market, regulatory bodies like the FDA’s Office of Tissues and Advanced Therapies (OTAT) will need to streamline and update guidelines to ensure safety and effectiveness. With over 100 therapies in Phase III trials, regulatory adaptations may be necessary to expedite approvals while balancing innovation with patient protection.
  2. Cost and Accessibility4: Cell and gene therapies are costly, with many treatments exceeding $1 million per patient. To manage the financial burden, outcome-based payment models, such as value-based pricing, subscription models, and risk-pooling arrangements, are being explored to make life-changing therapies accessible without straining employers and insurers financially. Employers may need to re-evaluate benefit plans to address these high-cost treatments.
  3. Healthcare Delivery Infrastructure2,4: Widespread adoption of cell and gene therapy will require specialized treatment centers and care delivery protocols. Advanced digital infrastructure will also be needed to support long-term patient monitoring, given the durability of gene therapies and the need for consistent data on success. Healthcare providers will need to educate and train professionals across specialties to provide appropriate follow-up and supportive care.

What’s Next for Cell and Gene Therapy?

The cell and gene therapy pipeline is robust, with 348 therapies expected to come to market within the next 3-5 years1. This includes new therapies for neurology and cardiology, areas where gene therapy has historically been less prevalent. As gene and cell therapies diversify into new specialties, they offer new avenues for treating complex diseases that have previously had limited therapeutic options. For example, in oncology, therapies are being developed for hard-to-treat cancers such as pancreatic, liver, and head and neck cancer. In neurology, therapies for conditions like ALS and Huntington’s disease are progressing through clinical trials and could open the door to targeted, long-term treatments for these debilitating diseases.

Moving from Treatment to Cure

Over the next decade, as gene therapies evolve from symptomatic treatments to curative solutions, the approach to chronic and genetic diseases may be forever altered. From reducing the need for transplantation to curing neurodegenerative diseases with a single treatment, cell and gene therapy has the potential to fundamentally redefine healthcare and patient outcomes. By addressing diseases at their genetic roots, gene therapy may offer patients a future free from the limitations of chronic illness, providing transformative solutions, health, and hope for those affected by genetic and complex diseases. Additionally, while the upfront costs of gene therapy can be high, these treatments could ultimately reduce long-term healthcare expenses by minimizing the need for ongoing care and costly treatments for chronic conditions.

As this field advances, cell and gene therapy’s impact on healthcare will be profound, laying the groundwork for a future in which medicine is curative, not just therapeutic. The next 10 years hold the promise of remarkable change, and as cell and gene therapies move from research labs to patient bedsides, the healthcare industry and society at large will need to prepare for a world where “treatment” is redefined by the power of genetic science.

1 https://www.asgct.org/publications/landscape-report
2 https://icer.org/news-insights/press-releases/icer-publishes-final-evidence-report-on-gene-therapies-for-sickle-cell-disease/
3 https://www.mckinsey.com/industries/life-sciences/our-insights/how-could-gene-therapy-change-healthcare-in-the-next-ten-years
4 https://www.milliman.com/-/media/milliman/pdfs/articles/managing_risks_related_to_gene_and_cell_therapies_for_self-insured_employers_with_stop-loss-coverage.ashx
5 https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products