Research

The road to a cure

The goal isn't to swap insulin injections for lifelong immune-suppressing drugs. It's cells that make insulin by themselves, and that the body leaves alone. Here's what stands in the way, who is building which part, and what has to go right.

Facts last checked 2026-11-11. Details and sources for each program are in the tracker.

Two problems, solved together

In type 1 diabetes, the immune system destroys the beta cells, the insulin-producing cells in the pancreas's islets. Replacing them has worked for decades: donor islet transplants can make people insulin-free. But two problems keep that from being a cure for everyone.

Problem 1

There are too few cells

Donor pancreases are scarce, and one patient often needs islets from two or three donors.

Answer: grow islet cells from stem cells, in unlimited amounts.

Problem 2

The immune system kills them

Twice over: the body rejects foreign cells, and the autoimmunity that destroyed the original beta cells is still there.

Answer today: lifelong immunosuppression, with real risks. Answer in trials: cells edited to be invisible to the immune system, or shielded in a device.

Both together

Immune-hiding stem-cell islets

Cells that can be made in a factory and are neither rejected nor attacked by the autoimmunity.

That means no insulin and no immunosuppression. That is what a cure would look like.

Eight problems a cure for children has to solve

Every program in the tracker works on some of these. Seeing them side by side shows where the field is crowded, and where almost nobody is working.

ProblemWhere it stands
Cell supply
Beta cells in unlimited amounts
Largely solved. Several groups make working islet cells from stem cells.
Rejection
Stop the immune system rejecting foreign cells
The big bottleneck. Three routes: gene editing that hides the cells, gentler drugs, or a physical barrier.
Autoimmunity
Stop the autoimmune attack on beta cells, including new ones
The fastest-moving area, with much of the progress coming from other autoimmune diseases.
Placement
Where in the body the cells should sit
Forearm muscle, the liver, the abdominal wall, implanted pouches; each a trade-off between survival, monitoring and access.
Manufacturing
Make cells at scale at a reasonable cost
The second bottleneck. Few players can do it, and it has slowed even the largest.
Safety
Kill switches and tumour risk
Cells that hide from the immune system must be safe without it; stem-cell products must contain no wrong cell types.
Durability
Effects that last years, not months
Long-term follow-up is missing for almost everything. The longest without immunosuppression is 14 months in one patient.
Children
Trials and approval for children
Almost all cell-therapy trials are in adults. Children come last, especially for treatments that need immunosuppression.

Where it stands

Each half has been shown separately in people. Stem-cell islets can make people insulin-independent: in Vertex's trial, 10 of 12 people given a full dose no longer needed insulin after a year, but they all take immunosuppressants. And gene-edited islets can survive without immunosuppression: in Uppsala, edited donor islets have been making insulin in one patient's forearm for 14 months with no immune attack and no immunosuppressive drugs, at a deliberately small dose.

The next step is to put the two together: edited stem-cell islets, at a full dose, in a trial. That's what Sana's SC451 is meant to do. It isn't in a trial yet.

From stem cell to forearm

The immune-hiding approach that worked in Uppsala, in four steps:

  1. A stem-cell bank. Induced pluripotent stem cells: adult cells reprogrammed so they can become any cell type. Sana's line is blood group O negative, so anyone could receive it.
  2. Gene editing. The HLA molecules, the cells' "ID tags" that T cells look for, are removed. More CD47, a "don't eat me" signal, is added, so other immune cells don't destroy cells without ID tags.
  3. Maturing and manufacturing. The stem cells are guided step by step into mature, insulin-producing islet cells: hundreds of millions per patient, with the same quality every time. This is the hardest part to scale, for every company in the field.
  4. Transplantation. In Uppsala, a small operation placed the cells in the forearm muscle, where the graft can be followed on PET-MRI scans and is easy to reach.

More in Uppsala's world first.

Three ways to protect new cells

  1. Hide them with gene editing. Remove the molecules the immune system recognises: Sana's approach in Uppsala, and BetaNova's SR-03.
  2. Use gentler drugs. Replace today's harshest anti-rejection drug with something more targeted. With tegoprubart, all 12 donor-islet recipients in a small US study became insulin-free (company data, not yet peer-reviewed). It still means lifelong medicine, which is a hard trade for a child.
  3. Put them behind a barrier. A pouch or capsule that lets insulin and glucose through but keeps immune cells out, like Encellin's. Earlier devices failed because the body walled them off with scar tissue.

A fourth, longer route is to fix the autoimmunity itself, so the cells don't need hiding at all. Seeteaching the immune system to stop.

What has to happen, in order

Dates after 2026 are targets, not forecasts. Most will slip.

  1. 2025done

    Proof in a person. Gene-edited islets survive and make insulin without immunosuppression (UP421, Uppsala, NEJM 2025 and 2026).

  2. Late 2026–2027next

    The stem-cell version enters trials. Sana expects to file for SC451's first trial "as early as" the end of 2026. This is the next big piece of news to watch for.

  3. 2027–2028target

    First answers. Do factory-made cells survive as well as donor cells? Is a full dose enough to stop insulin? Is it safe?

  4. Around 2028–2031target

    Does it last? Larger trials, follow-up over years, and manufacturing for thousands of patients instead of tens.

  5. Early 2030starget

    Approval for adults by the FDA and EMA, then decisions by health systems to offer it.

  6. After thattarget

    Children. Children need their own trials and approvals, which usually come after safety is established in adults.

Our own rough estimate, not a forecast: a cell therapy without immunosuppression is unlikely to be available for children before the mid-to-late 2030s. Treatments that slow the disease are much closer, and one is already approved.

What could go wrong

  • Money. Sana, which owns the immune-hiding technology used in Uppsala, has cash guided to last into mid-2027 and a going-concern warning in its latest report. It needs new capital or a partner.
  • Dose. Uppsala showed the cells survive. A dose large enough to cure has never been tried without immunosuppression.
  • Safety. Cells the immune system can't see are also cells it can't clean up if something goes wrong. Long-term safety has to be shown, and placing the cells somewhere easy to monitor and reach is part of the answer.
  • Durability. Fourteen months is promising. A cure has to last decades.
  • Manufacturing. Making cells consistently at scale has slowed even the biggest player: Vertex paused dosing in 2025 for a manufacturing analysis.
  • Children. Everything starts with adults. Children come when safety is proven.

Other teams on the same road

The more independent attempts there are, the better the odds that one gets there. The tracker follows them all; the main ones:

  • Vertex VX-017: stem-cell islets with immunosuppression, now Vertex's lead, with pivotal studies planned for 2027.
  • BetaNova SR-03: gene-edited, immune-evasive islets; trial application planned for late 2027.
  • Encellin: no gene editing, but a thin pouch meant to shield cells without scarring; early device results in people.
  • Aspect Biosystems: took over Novo Nordisk's stem-cell islet program in 2026; still preclinical.

Encapsulation has a mixed record: Vertex's device-based VX-264 and CRISPR's CTX211 were both stopped without showing enough benefit.

Stopping the attack, and finding it early

A second road works on the immune system itself. It can't replace cells that are already gone, but it can slow the attack: delaying the disease in people on the way to it, or keeping some of a newly diagnosed person's own insulin production. Even a little of that makes glucose easier to manage and lows less likely.

  • Teplizumab is the first approved drug that delays type 1 diabetes: in the key trial, by about two years at the median. It's approved in the US, UK and, since January 2026, the EU. In Sweden, regions have been advised to wait while its cost-effectiveness is assessed.
  • Baricitinib, a daily tablet, preserved insulin production in newly diagnosed young people. Two phase 3 trials started in 2026, both with Swedish sites.
  • Low-dose ATG and verapamil have positive phase 2 results; neither is approved or recommended for type 1 diabetes yet.
  • Not everything works. Sweden's own Diamyd found no effect in its phase 3 trial in 2026, and oral insulin did not prevent the disease in young children.

These treatments work best early, often before symptoms, and that's only possible withscreening. A blood test for autoantibodies finds type 1 diabetes years before it shows. In September 2026 an international consensus recommended screening all children. Sweden has no national programme yet; screening happens in research studies, mostly in Skåne.

An immune treatment alone won't cure someone who has had type 1 diabetes for years: their beta cells are gone. The likely long-term answer combines both roads: new cells, and an immune system that leaves them alone.

Read more

  • Uppsala's world first: islets the immune system ignores: In 2025 a team in Uppsala showed that gene-edited islet cells can survive in a person with type 1 diabetes without immunosuppression. What was done, what it shows, and what Sweden needs for the next step.
  • Teaching the immune system to stop: New cells aren't enough: the immune system remembers beta cells and attacks them again. Hiding the cells is one answer. Teaching the immune system tolerance is the other, and much of the progress comes from other autoimmune diseases.
  • Until the cure: the closed loop, part by part: Pumps and sensors are a bridge until biology takes over. A map of the closed loop, from inputs to the human in it, and where each part is limited today.
  • Sweden: who works on a cure: The research groups, companies and funders in Sweden working toward a cure for type 1 diabetes, and the gap between world-leading results and the industry needed to bring them to patients.
  • China: fast trials, few published results: China runs many of the world's early cell- and immune-therapy trials for type 1 diabetes. A look at the rules, the programs, and how they compare with the results from Uppsala.
  • Unproven stem-cell treatments: warning signs: Clinics and companies sell stem-cell and immune 'cures' for type 1 diabetes outside proper trials. How to tell real research from a commercial offer.