FDA Approves First Gene-Editing Therapy for Type 1 Diabetes, Marking Major Milestone in Cell-Based Treatment

The U.S. Food and Drug Administration has not approved any gene-editing therapy for type 1 diabetes as of mid-2026, officials confirmed. While the agency approved Lantidra, an allogeneic pancreatic islet cellular therapy, in 2023 for adults with severe hypoglycemia, gene-editing treatments for the disease remain in preclinical research stages, according to an FDA-commissioned review.

The FDA’s first approved gene-editing therapies use CRISPR technology to treat sickle cell disease, not type 1 diabetes, according to agency records and expert reviews. Another gene therapy for sickle cell disease, Lyfgenia by bluebird bio Inc., uses a lentiviral vector but does not involve gene editing, FDA documents show.

On Dec. 8, 2023, the FDA authorized Casgevy (exa-cel), developed by Vertex Pharmaceuticals Inc. and CRISPR Therapeutics, as the first cell-based gene therapy employing CRISPR/Cas9 genome editing for patients 12 years and older with sickle cell disease and vaso-occlusive complications.

For type 1 diabetes, no gene-editing therapy has yet received FDA approval, according to a 2025 review commissioned by the FDA and conducted by the PHG Foundation. The report states that gene therapies aimed at reducing or eliminating insulin dependence remain in preclinical stages, limited to in vitro and animal model research. The foundation cited biological and practical challenges, including the autoimmune nature of type 1 diabetes and difficulties in targeting and regulating gene expression, as reasons for the stalled progress in direct gene therapy for this condition.

The FDA did approve Lantidra (donislecel) on June 28, 2023, as the first allogeneic pancreatic islet cellular therapy for adults with type 1 diabetes who experience repeated severe hypoglycemia despite intensive management. Manufactured by CellTrans Inc., Lantidra consists of donor pancreatic islet cells harvested from deceased human donors. These cells are infused into the portal vein to engraft in the liver, where they produce insulin. However, Lantidra is classified as a cellular therapy, not a gene-editing or gene-therapy product, because it does not involve CRISPR or other genome editing techniques, according to FDA classification.

Clinical studies involving 30 participants showed that some achieved insulin independence after receiving Lantidra, while others had reduced but ongoing insulin requirements. The therapy requires chronic immunosuppression to prevent rejection, and most participants experienced at least one serious adverse event related to either the infusion procedure or immunosuppressive medication, FDA clinical data indicate.

Experimental gene-editing approaches for type 1 diabetes remain in early clinical or preclinical phases. Sana Biotechnology has reported early clinical data using CRISPR-Cas9 to create “hypoimmune” donor-derived islet cells designed to evade immune detection, potentially eliminating the need for immunosuppressive drugs. In a single-participant trial, these modified islet cells were implanted into the forearm muscle, and by day 28, investigators observed endogenous insulin production without immunosuppression. However, this work is preliminary, and long-term safety and efficacy have yet to be established, according to expert commentary and FDA-related analyses.

Other investigational gene therapies targeting type 1 diabetes include KRIYA-839, developed by Kriya Therapeutics, which aims to provide a one-time gene therapy expressing insulin and glucokinase via intramuscular delivery. These therapies remain in preclinical or early clinical development without FDA approval, reflecting ongoing industry research but not regulatory authorization.

The PHG Foundation report also highlights the potential for advanced cell-based therapies, including genome-edited induced pluripotent stem cells, as nearer-term options compared to systemic gene therapy. It notes that future gene therapy strategies may become feasible, particularly for rare monogenic forms of diabetes, but emphasizes that such developments are prospective and not yet realized.

In summary, while gene-editing technology has achieved regulatory milestones in diseases like sickle cell anemia, type 1 diabetes treatment currently relies on cellular therapies such as Lantidra. Gene-editing approaches for type 1 diabetes remain investigational, with no FDA-approved products as of mid-2026, according to agency officials and expert reviews.