Zimislecel

Seattle-based Sana Biotechnology announced 14-month follow-up data from a first-in-human study of UP421, an allogeneic islet cell therapy engineered with the company’s hypoimmune (HIP) platform, in a patient with Type 1 diabetes. The data support proof of concept for immunosuppression-free islet transplantation but remain early and not yet generalizable. Trial specifics The investigator-sponsored study, conducted at Uppsala University Hospital in Sweden, involved intramuscular transplantation of a single low dose of HIP-modified primary islet cells into the forearm of one patient with Type 1 diabetes. UP421 was derived from the islet cells of a deceased donor and engineered using Sana’s hypoimmune platform at Oslo University Hospital. The study was supported by a grant from The Leona M. and Harry B. Helmsley Charitable Trust. The primary objective was safety, with secondary endpoints including cell survival, immune evasion, and C-peptide production — a circulating biomarker of endogenous insulin secretion. At baseline, the patient had undetectable C-peptide both fasting and during a mixed meal tolerance test (MMTT). At 14 months, fasting and MMTT-stimulated C-peptide levels were detectable and, according to the company, comparable to levels observed during the first six months of the study. C-peptide levels exceeded those measured at months 9 and 12, a difference the company attributed to tighter glycemic control achieved between months 12 and 14. A 52-week PET-MRI scan confirmed the presence of islet cells at the transplant site. No safety issues were identified through 14 months, the company said. The study was designed to establish safety and proof-of-concept at a low dose and was not intended to demonstrate glycemic improvement or reduction in exogenous insulin use. Sana said it plans to file an investigational new drug (IND) application for SC451, a HIP-modified stem cell-derived islet cell therapy designed as a more scalable product, and to initiate a Phase I trial as early as this year. Full 14-month data were presented at the Advanced Technologies & Treatments for Diabetes (ATTD) conference. Research context Type 1 diabetes results from autoimmune destruction of insulin-producing pancreatic beta cells. Islet cell transplantation from deceased donors is an established procedure that can restore endogenous insulin production, but it has historically required lifelong immunosuppression to prevent both allogeneic rejection and recurrence of the autoimmune attack. Sana’s HIP technology modifies cells to evade detection by the recipient’s immune system, potentially eliminating the need for immunosuppressive drugs. The UP421 data, from a single patient, provide early evidence that HIP-modified cells can evade both allogeneic and autoimmune rejection, though the single-patient design limits generalizability. The treatment landscape for Type 1 diabetes centers on exogenous insulin delivery via injections or pumps, continuous glucose monitoring, and automated insulin delivery systems. Teplizumab (Tzield), developed by Provention Bio/Sanofi, received US FDA approval in 2022 to delay the onset of stage 3 Type 1 diabetes in at-risk individuals, but it does not restore beta cell function in patients with established disease. No stem cell-derived or genetically engineered islet cell therapy has received regulatory approval for established Type 1 diabetes. Another notable competitor in the same field is Vertex Pharmaceuticals’ VX-880, an allogeneic stem cell-derived islet cell therapy requiring immunosuppression, evaluated in a Phase I/II trial in Type 1 diabetes and now the subject of Phase III study. Leave a Reply Cancel reply Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

Zurich-based NewcelX Ltd. (Nasdaq: NCEL) and Irvine, California-based Eledon Pharmaceuticals, Inc. (Nasdaq: ELDN) have entered a collaborative research agreement to evaluate a combination strategy pairing NewcelX’s NCEL-101, a stem cell derived islet therapy, with Eledon’s tegoprubart (AT-1501), an anti-CD40L monoclonal antibody designed to modulate T-cell-mediated immune activation. The aim is to evaluate the pairing as a durable, immune-protected islet replacement and potential functional cure for type 1 diabetes. No financial terms linked to the deal were disclosed. The agreement is structured as a research collaboration rather than a traditional licensing arrangement. Eledon contributes clinical transplant data generated from over 100 patients treated with tegoprubart across kidney, heart, and diabetes-related transplant settings under multiple US FDA-cleared Investigational New Drug applications. NewcelX contributes NCEL-101, its lead program built on a human pluripotent stem cell platform and designed as a scalable, off-the-shelf cell replacement therapy intended to restore endogenous insulin production. Deal context NCEL-101 is a stem cell-derived islet cell therapy that aims to replace pancreatic β-cells destroyed by the autoimmune process underlying type 1 diabetes. Rather than targeting a single molecular pathway, the therapy delivers functional insulin-producing cells to restore glycemic control. Tegoprubart is a next-generation monoclonal antibody that binds CD40 ligand (CD40L/CD154), blocking its interaction with CD40 on antigen-presenting cells and thereby inhibiting downstream T-cell activation. Earlier-generation anti-CD40L antibodies were associated with thromboembolic complications; tegoprubart was designed to mitigate that risk. The rationale for the NewcelX collaboration is that pairing cell replacement with targeted immune modulation could promote durable islet graft survival without the broad immunosuppression typically required in transplant settings. NewcelX was formed through the October 2025 merger of NLS Pharmaceutics Ltd. (Switzerland) and Kadimastem Ltd. (Israel). The company’s pipeline is centered on cell therapy type 1 diabetes applications, with NCEL-101 as its lead asset. The cell therapy type 1 diabetes space has attracted substantial deal activity. Vertex Pharmaceuticals acquired Semma Therapeutics in September 2019 for approximately USD 950 million to obtain stem cell-derived islet cell therapy technology that later advanced as VX-880. Vertex subsequently acquired ViaCyte in July 2022 for approximately USD 320 million, consolidating additional islet cell programs. In 2023, Novo Nordisk entered a partnership with Aspect Biosystems valued at up to approximately USD 725 million per bioprinted stem cell-derived islet cell therapy emerging from the partnership. Of note, Lantidra (from CellTrans) is an allogeneic pancreatic islet cell therapy that received US FDA approval in 2023, becoming the first donor-derived islet cell transplant product approved for certain adults with type 1 diabetes. Also under development in the space is Vertex Pharmaceuticals’ zimislecel (formerly VX-880) — a stem cell-derived islet cell therapy in Phase I/II clinical development for type 1 diabetes Your email address will not be published. Required fields are marked * Comment * Name * Email * Website