Cell and Gene Therapies
Cell and gene therapies have the potential to cure disease in a one-time administration by targeting the root cause of disease at the cellular or genetic level. Since the first FDA approved cell-based gene therapy, a CAR T therapy in 2017, these medical technologies have transformed treatment for patients, like those with cancer and rare pediatric disorders. Rather than using traditional chemical compounds to treat disease, these therapies work by correcting or replacing damaged cells or genes. As of January 2026, the FDA has approved 25 cell and gene therapies overall, including 7 CAR T-cell therapies, which use the body’s own immune system to fight disease. Nine of the 25 approved therapies treat cancer. In cell therapy, cells are cultivated or modified outside the body before being injected into the patient to treat a condition in which the patient’s cells are damaged or diseased. And in gene therapy, a patient’s genetic material (such as DNA) is used to treat, cure or even prevent a disease.
The Complex Cell and Gene Therapy Production Process
Most conventional drugs can be manufactured in bulk, stored and distributed as needed. Cell and gene therapies are different, each treatment is custom-made from an individual patient’s own cells, requiring rapid, time-sensitive production and immediate administration. This highly complex, personalized process demands advanced quality controls and a new level of manufacturing sophistication. Because these technologies are still evolving, many production methods continue to be refined and optimized.
Only highly specialized facilities with rigorous regulatory and quality controls can manufacture cell and gene therapies. Delivering these therapies is challenging, requiring a specially trained workforce and precise, time-sensitive logistics for handling biological materials. Ongoing biopharmaceutical investments are expected to help standardize production, streamline delivery and increase competition in the years ahead. The pipeline reflects this investment: 438 cell and gene therapies are currently in development in the United States, a 70% increase since 2018, spanning hard-to-treat cancers, rare blood disorders, neurologic disorders and autoimmune diseases.
The Value of Cell and Gene Therapies
Value assessments can help shift away from traditional fee-for-service models toward value-driven health care. Unfortunately, traditional value assessments, known as health technology assessments (HTAs), often focus narrowly on costs, and rely on controversial and inherently discriminatory metrics like the quality-adjusted-life-year (QALY). They are also poorly suited for evaluating personalized cell and gene therapies, which may deliver large, long-term benefits and cost offsets that population-level tools fail to capture.
Patients may experience long-lasting or even curative benefits from a single dose of many cell and gene therapies, but evaluating these treatments is challenging because they often target small, rare patient populations. As a result, stakeholders are calling for new ways to measure outcomes that matter most to patients, such as patient-reported outcomes and real-world evidence, to better guide regulatory and payer decisions.
Improving Patient Access and Affordability
Early estimates show that cell and gene therapies will remain a small and affordable share of overall health spending. For example, cell and gene therapy spending is projected to reach just 0.1% of total U.S. health care spending and 1.3% of drug spending by 2030. Innovative payment models and a shift toward value-based care can further help ensure these treatments remain accessible and affordable.
To improve access and affordability, biopharmaceutical companies are working with payers and other stakeholders to develop new ways to pay for medicines, like developing innovative, outcomes-based contracts. In these voluntary agreements, a medicine’s price or discount is tied to the value it delivers for patients. These market-based arrangements can help lower costs and expand access so long as key implementation barriers are addressed. Despite this promise, access gaps remain: just 1 in 10 eligible Medicare fee-for-service patients is currently being treated with CAR T-cell therapy, driven largely by limited treatment centers, inadequate Medicare reimbursement and payer coverage restrictions.
Competitive Pressure from China
The United States leads today in cell and gene therapy innovation, but that lead is narrowing. China’s share of global clinical trial sites for cell and gene therapies rose to 39% in 2024, compared to 19% for the United States, a 15-point increase for China since 2019. Sustaining U.S. leadership will require continued investment in R&D, strong intellectual property protections and a predictable, science-based regulatory framework.
Future of Medicine: Cell & Gene Therapies
Cell and gene therapies address the root causes of disease, offering the potential to change or halt its course rather than simply managing symptoms. These breakthroughs, along with hundreds of promising treatments in development, signal a new frontier for treating cancers, rare genetic conditions, neurological disorders and other debilitating diseases. Continuing to foster U.S. innovation of cell and gene therapies is critical to ensuring American patients have access to these life-changing medicines.
Research and Development,Cancer,Cell and Gene Therapy,Future of Medicine
Report
CAR-T Therapy Revolutionizing Cancer Treatment
https://innovation.org/casestudy/car-t-therapy-revolutionizing-cancer-treatment/