Phase 1 Study Evaluating Hydrogel-formulated UC-MSCs Administered Subcutaneously in Addition to Standard of Care as a Treatment for Lupus Nephritis

 

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Phase 1 Study Evaluating Hydrogel-formulated UC-MSCs Administered Subcutaneously in Addition to Standard of Care as a Treatment for Lupus Nephritis

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ALON
YAAR
ALON YAAR alon@livekidney.bio LKBIO CKD TLV Israel *
 
 
 
 
 
 
 
 
 
 
 
 
 
 

SLE is an idiopathic, chronic, inflammatory disease with multifactorial etiology, affecting between 6.5-178 in 100,000 individuals worldwide, with a 9:1 female predominance. Lupus nephritis (LN) is the most common, serious feature of SLE, developing in approximately 40% of patients. LN is associated with significant morbidity, including end-stage renal disease, which occurs in about 15% of patients, and is linked with a 26-fold higher risk of mortality. Lupus treatment relies on the use of glucocorticoids and conventional immunosuppressants (e.g. methotrexate, azathioprine, or mycophenolate mofetil) and targeted therapies (e.g. belimumab, anifrolumab, or voclosporin) to control disease activity and prevent relapse. In addition, several studies have examined the effect of allogeneic Mesenchymal Stromal Cells (MSC) administered IV on lupus symptomology, yet this approach has elicited only modest immunosuppressive effects. To overcome this challenge, LiveKidney is investigating the safety and clinical utility of hydrogel formulated (hf) Umbilical Cord (UC)-derived MSCs administered SC, in addition to SoC, as a treatment for lupus and LN (NCT06737380).

In this open label Phase 1 study, patients are being administered a single dose of 60 x106 hf-UC-MSCs SC and are being evaluated via physical examination, clinical laboratory testing and SLE-activity assessments. Primary endpoints include occurrence rates and severities of grade 2 and above adverse events as well as toxicity determined by clinical laboratory profiling. Secondary endpoints include changes from baseline in SLE activity scores, patient-reported QoL measurements and prednisone administration

As of Oct. 19, 2025, the first two patients demonstrated significant reduction in SLEDAI-2K scores (Pt1: 20 points at baseline to 6 points on Day 168; Pt2: 10 points at baseline to 0 points on Day 84) with only transient, minor injection-site adverse events. Moreover, Pt1 demonstrated a concomitant reduction in Prednisone dosing (25 mg/day (Day 1) to 0 mg/day (Day 168). More data on these and other patients, including nephritis assessments, expected by Jan., 2026.

Despite the small number of patients treated thus far, the preliminary results seem to indicate that hf-UC-MSCs SC depot has induced a sustained, systemic, anti-inflammatory effect which is reflected both in reduction in SLEDI-2K scores and reduction of Prednisone dosing, with only minor local AEs. It is hypothesized that UC-MSCs are exerting their anti-inflammatory effects through the release of extracellular vesicles (EVs) and soluble active molecules in a paracrine fashion. Investigation of the safety and clinical utility of SC-administered hf-UC-MSCs as a treatment for lupus and LN is ongoing. Further conclusions to be based on data collected through Jan., 2026

Kewords