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Preparation of IPSC for Cell Gene Editing for the Treatment of AATD

StatusEnrolling by Invitation
PhaseNot applicable
Started
View on ClinicalTrials.gov ↗
Record status
This record was last updated March 24, 2025 (before its estimated February 28, 2026 completion). Its status may not reflect the trial's current state.

Record History

  1. Study First Submitted
Alpha 1-antitrypsin (AAT) deficiency is a genetic condition that leads to lung and/or liver diseases; current treatment of weekly augmentation of AAT addresses only lung diseases with moderate efficacy. Novel treatments based on gene editing can restore physiological levels of AAT and address lung and liver disease. The aim is to generate induced pluripotent stem cells (iPSC) from blood and urine of patients with different severe Alpha1-antitrypsin deficiency (AATD) genotypes. Further, the iPSC will be differentiated into hepatocytes (iHep). Since hepatocytes are the main producers of AAT, the iHep will be used to test different approaches of gene editing to correct various mutations. Gene editing will be conducted at University of Bern (Switzerland)
Trial Details
NCT Number NCT06892236
Lead Sponsor Fondazione IRCCS Policlinico San Matteo di Pavia
Collaborators: University of Bern
Conditions
(from sponsor)		 Alpha1-antitrypsin Deficiency
Related terms alpha 1-Antitrypsin Deficiency
Enrollment 3 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov