Gene therapy can change lives. According to the FDA, gene therapy works by modifying genetic material inside a person’s cells to treat disease — for example, by replacing a faulty gene, inactivating a harmful one, or introducing a new gene to help the body fight illness. Cell therapy, a related approach, uses living cells as the treatment itself, often by modifying or engineering cells to restore, replace, or enhance biological function. For years, these advanced therapies were limited to a small number of ultra‑rare conditions – such as spinal muscular atrophy (SMA), adenosine deaminase–severe combined immunodeficiency (ADA‑SCID) – which constrained their broader impact.
The picture is starting to change.
From “when” to “where”: Cell and gene therapies gain relevance for employer plans
In 2026, the cell and gene therapy pipeline is expanding to more conditions and becoming more complex. As a result, it’s more relevant for employer-sponsored plans. For plan sponsors, the question is no longer when cell and gene therapies may arrive, but where they will be most relevant.
New approvals and pipeline therapies are also showing different delivery methods including localized delivery (intrathecal, intravitreal, direct‑to‑brain and encapsulated ocular cell based therapies). There is growing comfort with technology as a result of improved safety with Adeno‑associated virus (AAV) vectors.
This report looks at cell and gene therapy trends and what they mean for benefits and care strategy.
Broader reach, higher impact
In the first quarter of 2026, cell and gene therapy activity continued to grow. More therapies are now in development, and they target a wider range of conditions and routes of administration. At the same time, recent regulatory delays, product shortages, and market exits underscore the volatility of this space and the uncertainty around timing and availability.
Historically, this level of activity had limited implications for employer‑sponsored plans because gene therapies were largely confined to ultra‑rare diseases affecting very small numbers of members. Today, a broader and more diverse development pipeline changes that calculus. As gene therapies move beyond narrow use cases, the likelihood that employer plans will encounter eligible members increases.
For plans, this means gene therapy is no longer an occasional exception, but an emerging factor that may shape benefit design, cost management, and care strategy over time.
Oncology moves to the forefront
In the past, cancer treatment played a smaller role in gene therapy discussions , even as CAR‑T therapies — a form of cell therapy — were already an important focus within oncology.
Today, their prominence is increasing, alongside broader cell and gene therapy development in cancer care.
This matters because cancer care already brings high costs and complexity for plans. CAR‑T and other oncology‑focused cell and gene therapies may introduce new considerations, including:
- Where and how it’s delivered
- How it fits into current care
- How plans manage high upfront costs
As CAR‑T therapies and other cell and gene therapies become more common in oncology, plan sponsors must look beyond one-time cases. They must plan for it as part of their broader high‑cost care strategy.
Rare disease still central – but less dominant
Rare diseases continue to be a focus of gene therapy development. Many FDA-approved cell and gene therapies on the market today are indicated for small patient populations, often for inherited conditions that require specialized care.
At the same time, rare disease is no longer the only focus.
Plan sponsors must be ready for rare disease cases while also planning for where gene therapy may go next.
Cardiometabolic and neurological gene therapies emerge
Gene therapies for cardiometabolic, and neurological conditions are starting to enter later-stage development. Examples include therapies under investigation for OTOF-related hearing loss and glycogen storage disease type 1, which suggest growing activity beyond historically dominant rare disease areas.
These categories often involve:
- Larger covered populations than ultra-rare diseases
- Chronic or life-long conditions
- Ongoing cost and care considerations
While most of these therapies remain in development, their progression signals that gene therapy could, over time, extend beyond isolated, one‑time cases. For plan sponsors, this points to the need for longer‑term strategic awareness rather than near‑term coverage action.
What the progression of gene therapy means for plan sponsors
As gene therapy reaches new areas, it carries greater weight in long‑term benefit strategy.
For plan sponsors, thinking ahead is essential.
- Near term: Prepare plans and benefits for gene therapy cases, especially those tied to rare diseases.
- Mid term: Get ready for more cancer‑related gene therapies and their impact on oncology care and costs.
- Long term: Plan for possible use in more common conditions. This may require new approaches to coverage, cost, and care coordination.
Watching how treatments change may be as important as following new ones in development.
Preparing for the evolving impact of gene therapy on employer-sponsored plans
Gene therapy is having a greater impact on employer‑sponsored plans. Being aware of how gene therapy is evolving – and where it may head next – can help plan sponsors prepare.
This quarterly outlook tracks changes in gene therapy and what they mean for coverage, cost, and care strategy.
To stay informed between reports, subscribe to the Evernorth monthly Gene Therapy Pipeline Report for updates on emerging therapies and market developments.
To better understand how the cell & gene therapy pipeline could impact your organization specifically, engage your account team directly.
This article was created with the assistance of AI tools. It was reviewed, edited, and fact-checked by Evernorth’s editorial team and subject matter experts.
