Therapies aiming to restore endogenous insulin production.

Insulin independence in a small early cohort. Stem-cell-derived islets restored insulin production in an early trial; immunosuppression and long-term safety remain major limits.

Years off insulin for some; substantial treatment risks. Islet cells from deceased donors, infused into the liver — the first approved cell therapy for T1D.

Anti-CD40L antibody replacing toxic tacrolimus in donor islet transplants — 12 of 12 insulin-independent at UChicago. An engineered Fc-silent anti-CD40L monoclonal antibody (tegoprubart / AT-1501) that replaces nephrotoxic and beta-cell-toxic tacrolimus in human donor islet transplantation; in the University of Chicago Phase 1/2 trial (NCT06305286), 12 of 12 patients in the initial cohort achieved insulin independence with mean HbA1c ~5.4%.

Off-the-shelf umbilical-cord MSC infusion with positive Phase 2 adult RCT data; pediatric Phase 2 readout pending. An off-the-shelf intravenous infusion of pooled umbilical-cord Wharton's jelly mesenchymal stromal cells that preserved 90% of endogenous insulin production at 12 months (versus a 47% decline on placebo) in a randomized Phase 2 adult trial, with a 66-patient pediatric Phase 1/2 trial now in follow-up.

First-in-human proof that gene-edited donor beta cells can survive 14 months with zero immunosuppression — at a sub-curative dose in 1 patient. Deceased-donor human pancreatic islets engineered with Sana's three-edit hypoimmune platform (B2M-/-, CIITA-/-, CD47+) that survived, secreted glucose-responsive C-peptide, and evaded both alloimmune and autoimmune rejection for 14 months with zero immunosuppression in a landmark New England Journal of Medicine first-in-human study.

A C-peptide signal from five people; regeneration unproven. An oral menin inhibitor with a company-reported C-peptide signal from a very small, uncontrolled trial.

Her own cells — and immunosuppression. The famous "cured with her own cells" case. She was already on anti-rejection drugs.

Islets that last for years — and drugs that last just as long. A retrievable under-the-skin pouch that keeps donor islets alive for years, with standard anti-rejection drugs.

Autologous dendritic cells restoring HLA-E CD8+ Tregs — slowed C-peptide loss at 5 months in a 25-patient Phase 1/2 RCT. Autologous immature dendritic cells pulsed ex vivo with an Hsp60 signal peptide (spHsp60) to restore defective HLA-E-restricted CD8+ regulatory T cells; in a 25-patient randomized double-blind Phase 1/2 trial (NEJM Evidence 2024), AVT001 significantly slowed C-peptide decline at Day 150 with zero serious adverse events.

An oral polyamine-pathway inhibitor being studied to reduce beta-cell stress.

GLP-1 receptor drugs — the semaglutide/tirzepatide family now famous for type 2 diabetes and weight loss — are being explored off-label for T1D.

Early-stage; watch this space. Stem-cell-derived islets in an early trial for severe, long-standing T1D.

Drug-free by design; very early. An ultrathin, retrievable film pouch that protects islet cells under the skin.

An autologous tolerogenic dendritic-cell injection developed at LUMC, now in a recruiting Dutch Phase 1b for adults with T1D diagnosed within five years.

An old drug in a human body — if it really can be re-dosed. A human version of a T-cell-depleting drug that already works once, built so it can be given again.

A regulatory T-cell-directed IL-2 biologic in a Phase 2 trial listed as recruiting for recent-onset T1D.

A short CD154-derived peptide designed to restrain pathogenic CD40 signalling with the aim of avoiding broad immunosuppression.

This approach takes a person's own regulatory T cells (Tregs) — the immune cells that normally keep autoimmunity in check — grows them into the billions in a lab, and infuses them…

Repeated BCG vaccination remains an experimental immune/metabolic approach to T1D.

A repurposed oral immunomodulator best known as the multiple-sclerosis drug Tecfidera, now being tested in two recruiting Chinese studies: a randomized phase 3 trial in 90 adults…

A dialysis-like procedure that circulates a person's blood through a device where their immune cells are briefly co-cultured with cord-blood-derived stem cells, then returned —…

Kriya has opened two recruiting first-in-human Phase 1/2 trials in September–October 2026: PROGRESS (NCT07829302 in Melbourne, Australia) testing intramuscular VV-14300…

Promising first recipient, far too early to generalize. One recipient achieved insulin independence on day 110—with immunosuppression and no evidence yet that the result is durable or repeatable.

A repurposing hypothesis around denosumab, the RANKL-targeting osteoporosis drug, to protect or improve beta-cell function in early T1D.

An antigen-specific immune-tolerance candidate in a recruiting Phase 1b/2a trial.

Regrow your own cells — still in the lab. Drugs that coax the body to regrow its own insulin-producing cells instead of transplanting new ones.

A cure without anti-rejection drugs — unproven. Hypoimmune stem-cell islets designed to avoid immunosuppression; the current candidate remains preclinical.

Pre-vascularized refillable macroencapsulation pouch — cell-loaded MailPan remains preclinical while ExOlin tests insulin diffusion. A planar extraperitoneal macroencapsulation pouch (MailPan) implanted empty for pre-vascularization and loaded or refilled with insulin-producing cells through subcutaneous ports without systemic immunosuppression; cell-loaded MailPan remains preclinical, while its companion acellular membrane device for physiological extraperitoneal insulin diffusion (ExOlin) is in a first-in-human pilot trial (NCT05221359).

The IL-17A antibody ixekizumab is being tested for beta-cell preservation after T1D diagnosis.

Autologous stomach-biopsy stem cells reprogrammed into insulin-secreting organoids in 10 days — preclinical mouse reversal. Adult human stomach stem cells harvested via routine upper GI endoscopic biopsy and transiently reprogrammed with NGN3, PDX1, and MAFA into glucose-responsive Gastric Insulin-Secreting (GINS) organoids within 10 days; reversed diabetes for over 100 days in mice without teratoma risk, with hypoimmune-edited variants reported in 2026.

pMHC-coated nanoparticles that reprogram autoreactive T cells into Tr1 cells in mice — preclinical in T1D. Iron-oxide nanoparticles coated with autoimmune peptide-MHC class II complexes (Navacims, Santamaria Nature 2016) that reprogram cognate autoreactive CD4+ T cells in vivo into IL-10-secreting Tr1 regulatory T cells; partnered with Novartis for T1D (PVT101), while sibling candidates PVT201 (liver) and PVT401 (celiac) have entered human Phase 1 trials.

Seraxis SR-02 is allogeneic pancreatic endocrine cell clusters grown from a proprietary stem-cell line and implanted into the omentum (the fatty apron in the abdomen), where they…

Remygen is Diamyd Medical's controlled-release oral form of GABA, a molecule that in lab and animal studies coaxes beta cells to multiply and calms islet inflammation.

Promising idea, first-in-human, and open only to a rare genotype. Your own regulatory T cells, re-engineered to call off the attack on your beta cells.

First-in-human xenotransplant program using encapsulated neonatal pig islet cells for adults with unstable T1D and repeated severe hypoglycemia despite closed-loop therapy.

Very early — but a genuinely different delivery route. iPSC islets grown into a sheet and implanted under the skin, not infused into the liver.

A macroencapsulation device that addresses encapsulation's classic weakness — oxygen starvation — by building in a refillable oxygen tank that is topped up daily.

An antigen-specific liposome that packages a proinsulin peptide with calcitriol (active vitamin D), being tested in a Queensland Phase 1 trial in adults with stage 3 T1D.

Foxp3-locked islet-targeted CAR-Treg optioned by AstraZeneca — still preclinical in T1D. An autologous multi-modular engineered regulatory T-cell (CAR-Treg) candidate incorporating Quell's Foxp3 Phenotype Lock and an islet-homing chimeric antigen receptor; optioned by AstraZeneca in November 2024 for IND-enabling preclinical development in Type 1 Diabetes.

Immunocore’s first autoimmune ImmTAAI molecule: a soluble TCR that binds a pre-proinsulin peptide on HLA-A*02:01 beta cells and delivers local PD-1 agonism.

Kill the attackers, don't hide from them — a good idea that has never met a human. Microgels that make attacking T cells self-destruct at the graft site, aiming for tolerance in one spot instead of drugs everywhere.

Fibrosis solved in monkeys; the cells inside still fade. An alginate capsule laced with a chemokine that repels attacking T cells and recruits protective ones.

A preclinical successor with limited predecessor human evidence. CRISPR-cloaked islet cells designed to survive without immunosuppression. The Phase 1 was terminated, and the successor, CTX213, has not reached humans.

An in-the-body gene therapy that aims to regrow insulin-making capacity without any transplant.

The next real shot at drug-free islets — still entirely in mice. Off-the-shelf stem-cell islets edited to dodge T cells, NK cells and antibodies, aiming to need no anti-rejection drugs. No human has been dosed.

Very early; little to show yet. Perinatal-tissue cells infused into the pancreas just after a new diagnosis.

An early dose-escalation study of donor-derived CD7-targeted CAR-T cells in stage 2 or 3 T1D.

A City of Hope pilot plans to test a person’s own regulatory T cells engineered to target CD6.

An early registered study of engineered red blood cells in recent-onset autoimmune T1D.

A small investigator-led cell-infusion study in adults with autoimmune T1D and residual insulin secretion.

A first-in-human gene-therapy program using a person's own CD34+ hematopoietic stem and progenitor cells, modified ex vivo with a lentiviral vector to express PD-L1 and…

Phase 1/2 registered; no human outcomes yet. A registered Phase 1/2 islet-cell program designed for eligible people regardless of blood type; human efficacy remains unproven.

A pre-IND stem-cell-derived islet replacement program for insulin-dependent T1D, which NewcelX plans to pair with Eledon's anti-CD40L antibody tegoprubart in a…

Preclinical oxygen support; human benefit unproven. A sealed islet implant that makes its own oxygen, so the protected cells don't suffocate.

A personalized immune-tolerance vaccine made from a person's own dendritic cells loaded with proinsulin peptide, being tested by City of Hope in a small phase 1 T1D…

A pharma retreat rehomed as a startup's flagship. Rodent data only. Bioprinted islet tissue, immune-cloaked and immune-shielded, designed to need no anti-rejection drugs. It has never been in a human — and the assets got here because Novo Nordisk quit.

The most complete answer to immunosuppression anyone has — in mice. It has never reached a human with T1D. Teach the immune system to read the donor as self — then stop the drugs altogether.

A small Egyptian research protocol proposes turning a participant’s fat-derived cells into insulin-producing cells.

A kinase-inhibitor repurposing study asks whether sorafenib can preserve insulin production.

A Japanese first-in-human Phase 1/2a trial of a person's own fat-derived stem cells turned into insulin-producing cells (TUFF-IPC) and placed in the mesentery.

Real idea, real grants, no human evidence yet. Immune-cloaked stem-cell islets with built-in sensors that report whether the graft is alive. Funded, animal-stage, and a long way from a person.

A cautionary tale, not a contender — bought, then buried in silence. Big pharma bought an encapsulation platform, promised a trial in 2024, and has said nothing since.

A discontinued combination programme that preserved more C-peptide during a Phase 2 trial, with benefits diminishing after treatment stopped.

An oral immune-pathway inhibitor tested soon after T1D diagnosis.

Antigen-specific cytolytic CD4+ peptide vaccine — terminated after failing its 110-patient Phase 2 trial. A modified proinsulin peptide containing a thioredoxin oxidoreductase motif (Imotope) engineered to induce antigen-specific cytolytic CD4+ T cells; completed Phase 1b in 41 adults but was terminated after the 110-patient Phase 2 IMPACT trial showed no C-peptide preservation at 1 year.

A clear lesson in what doesn't work yet. Encapsulated stem-cell islets — proved safe drug-free, but the cells did not produce enough insulin.