December 29, 2025
Type 1 diabetes affects approximately 1.6 million people in the United States. For decades, insulin replacement remained the only viable treatment option, addressing symptoms rather than underlying disease progression. Over the past three years, this paradigm has begun to shift, as disease-modifying therapies move from concept to clinical reality.
The FDA approval of teplizumab in 2022 marked the first regulatory validation of immune intervention in type 1 diabetes. Since then, a growing pipeline spanning immunomodulators, cell-derived therapies, and gene-based approaches has emerged, targeting both early disease interception and restoration of insulin independence. The table below summarizes key programs shaping this evolving landscape.
Emerging Disease-Modifying Therapies in Type 1 Diabetes
| Company | Medicine | Modality | Phase or Status | Key Update |
| Sanofi Provention Bio | Teplizumab | Monoclonal antibody, anti-CD3 | FDA approved November 2022 | Accepted for expedited FDA review in October 2025 for Stage 3 type 1 diabetes in newly diagnosed patients |
| CellTrans | Donislecel (Lantidra) | Cell-based therapy, donor-derived pancreatic islets | FDA approved June 2023 | Approved based on Phase 3 single-arm studies in patients with hypoglycemia unawareness; 70 percent achieved at least 12 months of insulin independence |
| Vertex Pharmaceuticals | Zimislecel (VX-880) | Cell-based therapy, stem cell-derived pancreatic islets | Phase 3, active and recruiting | Phase 1 and 2 results showed 10 of 12 patients insulin independent one year after a single full-dose infusion |
| Diamyd Medical | Diamyd | Antigen-specific immunotherapy, GAD65 vaccine | Phase 3, active and recruiting | Phase 3 DIAGNODE-3 topline readout expected March 2026 |
| Immthera Poltreg | PTG-007 | Cell-based therapy, autologous regulatory T cells | Planned Phase 2 and 3, pre-IND | Preparing IND for adaptive trial in presymptomatic type 1 diabetes following positive FDA feedback |
| Sanofi | Brivekimig (SAR442970) | Nanobody-based immunotherapy, anti-TNF alpha and OX40L | Phase 2, active and recruiting | Phase 2 trial ongoing in type 1 diabetes; no results disclosed |
| Sanofi ImmuNext | Frexalimab (SAR441344) | Monoclonal antibody | Phase 2, active and recruiting | Phase 2 trial ongoing in type 1 diabetes; no results disclosed |
| CRISPR Therapeutics | CTX211 (formerly VCTX211) | Cell-based therapy, gene-edited stem cell-derived islets | Phase 1 and 2, active and recruiting | Program update anticipated by end of 2025 |
| Cour Pharmaceuticals | CNP-103 | Antigen-specific immune tolerance therapy | Phase 1 and 2, active and recruiting | First patient dosed in August 2025 |
| Otsuka | OPF-310 | Cell-based therapy, xenotransplantation | Phase 1 and 2, active and recruiting | Phase 1 and 2 trial ongoing; no results disclosed |
| GentiBio | GNTI-122 | Cell-based therapy, autologous regulatory T cells | Phase 1, active and recruiting | IND approved July 2025; Phase 1 trials scheduled to begin Q3 2025 |
| Genprex | GPX-002 | AAV-based gene therapy | Preclinical | Positive preclinical data presented June 2025; no IND filed |
| Sana Biotechnology | SC451 | Cell-based therapy, HIP-modified stem cell-derived pancreatic islets | Preclinical | Positive FDA INTERACT feedback; IND submission planned for early 2026 |
| iTolerance | iTOL-101 | Cell-based therapy, donor-derived pancreatic islets | Preclinical | Advancing through preclinical studies |
| iTolerance and Kadimastem | iTOL-102 | Cell-based therapy, allogeneic stem cell-derived pancreatic islets | Preclinical | FDA pre-IND meeting held February 2025 |
Note: Phase and regulatory status are shown in the context of the US market and FDA interactions.
Immune Interception Sets the Foundation
Teplizumab established immune modulation as a viable strategy in type 1 diabetes by delaying disease progression in high-risk individuals. Its acceptance for expedited FDA review in newly diagnosed Stage 3 patients represents a potential expansion from prevention into earlier disease treatment, significantly broadening its addressable population.
Sanofi continues to build on this foundation with Frexalimab and Brivekimig, extending its autoimmune immunology platform into type 1 diabetes. While efficacy data are pending, these programs reflect a strategic commitment to immune-based disease modification.
Cell Therapy Moves Toward Functional Cure
Cell-based approaches represent the most direct path toward insulin independence. Lantidra demonstrated that donor-derived islet transplantation can restore glycemic control in selected patients, albeit with procedural complexity and limited scalability.
Vertex’s VX-880 significantly raised expectations for stem cell-derived islet replacement. The high rate of insulin independence observed in early trials has supported advancement into Phase 3 development and positioned cell therapy as a central pillar of next-generation type 1 diabetes treatment.
Gene-edited and immune-evasive approaches, including CTX211 and SC451, aim to overcome durability and immunosuppression challenges, though these remain earlier in development.
Precision and Tolerance-Based Strategies
Antigen-specific and regulatory T cell therapies offer a complementary approach focused on restoring immune tolerance rather than replacing beta cells. Diamyd’s GAD65 vaccine targets a genetically defined responder population, with Phase 3 data expected in 2026 that could establish the first genotype-personalized therapy in type 1 diabetes.
Engineered Treg programs from Immthera, GentiBio, and others aim to deliver durable immune regulation with potentially favorable safety profiles, particularly in presymptomatic or early disease settings.
Takeaway
Type 1 diabetes is transitioning from a purely insulin-managed condition to a competitive landscape of disease-modifying strategies. With approved immune interception, late-stage cell therapies, and a growing pipeline of tolerance-based approaches, the next several years are likely to redefine treatment expectations. Clinical milestones anticipated in 2026, particularly from Vertex and Diamyd, may represent critical inflection points for both patient care and investment narratives.
