Cell and Gene Therapy Industry Explained: The U.S. View
Cell and gene therapies aim to treat disease at its root: CAR-T therapies re-engineer a patient's immune cells to attack cancer or, in newer trials, autoimmune disease, and gene therapies deliver a working gene to replace a faulty one. The science is promising, but the business is hard. Treatments are often made one patient at a time, can cost millions of dollars, and carry real safety risks. 2026 showed both sides. In August the FDA granted accelerated approval to Ultragenyx's Genglycos, the first gene therapy for glycogen storage disease type Ia, at a list price of about $2.7 million per patient, based on a surrogate endpoint with confirmatory data required. The same month Novartis paused eight trials of an experimental CAR-T therapy in autoimmune and neurological diseases after three patient deaths from a severe immune reaction, and Bristol Myers Squibb paused studies of a rival therapy as a precaution. Established cancer CAR-Ts sell well: Bristol Myers' Breyanzi had $1.36 billion of sales in 2025. Payment is the other bottleneck, and Medicare and Medicaid's regulator CMS is testing outcomes-based payment for sickle cell gene therapies.
- Approval
- FDA approved the first gene therapy for GSDIa in August 2026, about $2.7M
- Safety
- Novartis paused 8 autoimmune CAR-T trials after 3 deaths in August 2026
- Sales
- Bristol Myers' CAR-T therapy Breyanzi had $1.36B of sales in 2025
How the chain works
Cancer CAR-T, autoimmune CAR-T and gene therapy
| CAR-T in cancer | CAR-T in autoimmune disease | Gene therapy | |
|---|---|---|---|
| Stage | Approved and growing | Clinical trials | Approved for several rare diseases |
| 2026 signal | Breyanzi $1.36B sales in 2025 | Novartis paused 8 trials after 3 deaths | Genglycos approved for GSDIa, about $2.7M |
| Main risk | Manufacturing capacity, cost | Safety | Durability, price, payer acceptance |
| What to watch | Sales, new indications | Trial restarts and safety data | Confirmatory data, payment models |
Approvals on new terms
On August 19, 2026, according to Cell & Gene Therapy Insights citing Reuters, the FDA granted accelerated approval to Ultragenyx's Genglycos for patients aged 8 and older with glycogen storage disease type Ia, the first gene therapy for the condition. The approval rests on a surrogate endpoint, reduced need for cornstarch, and the FDA requires confirmatory data. The U.S. list price is about $2.7 million per patient.
Accelerated approvals let patients get treatments sooner but leave questions about long-term benefit, which makes payers cautious about million-dollar prices.
Autoimmune CAR-T hits a safety wall
CAR-T therapies have worked well in some blood cancers, and companies have been testing whether resetting the immune system can treat autoimmune diseases such as lupus. In August 2026 Novartis paused eight trials of its experimental therapy rap-cel in lupus, rheumatoid arthritis, vasculitis, multiple sclerosis and myasthenia gravis after three patients died from a severe immune reaction; its lymphoma and leukemia trials were not affected.
Bristol Myers Squibb then paused studies of a rival CAR-T therapy, zola-cel, out of "an abundance of caution." The episode shows that moving cell therapy into large, non-cancer markets raises the bar on safety.
Manufacturing and money
Many cell therapies are made from each patient's own cells, which limits scale and raises cost. Companies are working to industrialize production: Bristol Myers ended a manufacturing deal worth up to $380 million with Cellares, citing commercial-scale production of Breyanzi, which had $1.36 billion of sales in 2025.
Deals continue despite setbacks. Johnson & Johnson agreed to initial payments of $785 million, including a $465 million equity investment, in a deal with Sail Biomedicines, with an option to acquire it for a further $2.58 billion. On the payment side, CMS's Cell and Gene Therapy Access Model, announced in 2024, lets state Medicaid programs use outcomes-based agreements, currently for sickle cell gene therapies, and may expand to other diseases.
How the U.S. view differs from the China view
The Chinese version of this page looks at Chinese developers of CAR-T and gene therapies, which can run trials and manufacture at lower cost and increasingly license their therapies to Western drugmakers.
From the U.S. side the questions are safety, price and who pays. Million-dollar prices push the government and insurers toward outcomes-based deals, and safety setbacks can quickly change the outlook for whole categories.
August 2026: one approval, one pause
On August 19, 2026, the FDA granted accelerated approval to a gene therapy for glycogen storage disease type Ia, priced at about $2.7 million.
Days later, on August 24, Novartis paused eight autoimmune and neurological CAR-T trials after three deaths, and Bristol Myers paused a rival program.
The same month showed the promise and the risk: one-time treatments can change lives, but safety and long-term evidence decide how far and how fast they spread.
- Safety events can halt whole categories of trials at once.
- Accelerated approvals come with requirements for confirmatory data.
- Price and payment models are as important as science for adoption.
Common misreadings
MythCell therapy has proven safe for all diseases.
RealityNovartis paused 8 autoimmune and neurological CAR-T trials after 3 deaths in August 2026.
MythGene therapy approvals mean proven long-term cures.
RealityGenglycos received accelerated approval on a surrogate endpoint, with confirmatory data required.
MythThese therapies are too niche to sell.
RealityBristol Myers' Breyanzi had $1.36B of sales in 2025.
MythPayers simply pay list price.
RealityCMS is testing outcomes-based payment for sickle cell gene therapies.
Safety, pricing and manufacturing: scenarios
| Scenario | Main effect | How it passes through |
|---|---|---|
| Autoimmune CAR-T trials resume safely | Market size | Large new markets open |
| More safety events | Regulators | Stricter trial rules, slower progress |
| Outcomes-based payment spreads | Access | More patients treated, less upfront risk |
| Off-the-shelf cell therapies succeed | Manufacturing | Lower costs, faster treatment |
| Confirmatory trials disappoint | Approved products | Labels narrowed or withdrawn |
| Biotech funding tightens | Pipeline | Fewer programs, more asset sales |
Supply and demand by segment
| Segment | Supply rigidity | Concentration | New demand | Policy role | What to watch |
|---|---|---|---|---|---|
| CAR-T (cancer) | High (patient-specific manufacturing) | High | Yes | High (FDA) | Sales, capacity |
| CAR-T (autoimmune) | High | Medium | Yes (if safe) | Very high (trial safety) | Trial restarts |
| Gene therapy | High (vector manufacturing) | Medium | Yes | Very high (approval, pricing) | Approvals, payment |
| Contract manufacturing | High | Medium | Yes | Medium | Deals, layoffs |
Supply rigidity: how long new capacity takes and whether money can speed it up. Concentration: whether output sits in a few countries or companies. The more rigid and concentrated, the more prone to shortages.
How to track it
| Indicator | What it tells you | Where to find it |
|---|---|---|
| FDA cell and gene therapy approvals | New products | FDA approved cellular and gene therapy products list, free |
| Clinical holds and trial pauses | Safety risk | Company announcements and FDA notices |
| CMS Cell and Gene Therapy Access Model | Payment approach | CMS Innovation Center, free |
| Deal and licensing announcements | Funding and confidence | Industry news coverage |
| Weight-loss drugs | Another fast-changing drug market | U.S. view: GLP-1 drugs |
Sources
- Cell & Gene Therapy Insights: a CAR-T safety halt for Novartis and a first gene therapy approval for Ultragenyx (2026)
- Milliman: CMS Cell and Gene Therapy Access Model (2024-04-25)
Last updated 2026-10-18.