Cell and gene therapies (CGTs) are one of the most important developments in modern medicine. They offer new ways to address the underlying biological causes of disease rather than only managing symptoms. Initially focused on cancer and rare genetic disorders, these therapies are now being explored for broader conditions including autoimmune and cardiovascular diseases. More than 25 CGTs have been approved in the U.S., with hundreds more in development, signaling continued growth in this rapidly evolving area.
Two approaches, one vision
CGTs encompass two distinct approaches. Cell therapies use living cells to directly treat disease, while gene therapies modify or replace genetic material to address underlying causes. Together, they represent a new age of precision medicine with the potential for long-term disease control or even functional cures. This creates possibilities that were previously unattainable with traditional treatments.
CAR-T therapy: A breakthrough in cancer care
One of the most recognized CGT categories is CAR-T therapy, which uses a patient's modified immune cells to attack cancer. Since the first CAR-T approval in 2017, these treatments have transformed care for certain blood cancers. Researchers are now testing CAR-T therapies for autoimmune conditions, like lupus and rheumatoid arthritis. This could expand the potential reach of these innovative treatments.
Complexity and access challenges
Despite their promise, CGTs remain highly complex to deliver. Patients typically undergo extensive evaluation and require coordination with specialized treatment centers before receiving therapy. Unlike many traditional medications, CGTs are usually given at centers of excellence. They require multidisciplinary care teams, intensive monitoring and long term follow-up. These operational challenges can contribute to significant delays between diagnosis and treatment.
A shifting regulatory landscape
Access may improve as the treatment landscape evolves. In 2025, the FDA removed the Risk Evaluation and Mitigation Strategies (REMS) for CAR-T therapies. This reduced administrative burden for providers. Changing regulations and growing provider experience may allow more community-based oncology centers to offer these therapies. This could help address geographic and logistical barriers that currently limit patient access.
The cost equation
Cost remains one of the most significant challenges. Cell therapies like CAR-T are generally priced between $400k - $700k and gene therapies as high as $4 million. Additional fees are incurred for the patient workup and provider administration of the drugs. Although CGT are typically given for one dose only, the high cost creates substantial financial exposure for health plans and employers. Simultaneously, long-term outcomes data remain limited, raising questions about durability and overall value. It can be hard to fit CGTs into traditional healthcare financing models because of high upfront costs, specialized care requirements and evolving evidence.
As more people use these treatments, stakeholders are exploring new reimbursement approaches, including bundled payment arrangements and other innovative financing strategies designed to improve cost predictability.
What this means for employers
CGTs represent both a clinical breakthrough and an emerging financial risk. As the pipeline expands and access improves, you should monitor upcoming therapies, evaluate stop loss protections and assess oncology care networks to prepare for the growing impact of these transformative treatments.
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