人体移植基因编辑猪肾规模化临床试验在美国启动

Core Points - The company United Therapeutics has completed the first clinical trial of gene-edited pig kidney transplantation into humans at NYU Langone Medical Center, marking the official launch of large-scale clinical trials [1] - This trial is the first FDA-approved human transplantation of gene-edited pig kidneys, aimed at systematically evaluating the safety and efficacy for patients with end-stage renal disease [1] Summary by Sections - Trial Details - The initial phase of the study includes 6 participants, with surgeries performed at two transplant centers [1] - An independent data monitoring committee will review safety and efficacy data after at least 12 weeks post-surgery to decide on the continuation of the next phase, potentially expanding the participant pool to about 50 [1] - Participant Criteria - Participants are aged between 55 and 70 years, all diagnosed with end-stage renal disease and have undergone dialysis for at least 6 months [1] - Following the transplantation, participants will be monitored for 24 weeks to assess kidney function, survival rates, quality of life, and safety, along with long-term monitoring for organ function and potential animal-source infection risks [1] - Genetic Modifications - The gene-edited pig kidneys developed by the company underwent 10 genetic modifications, removing 4 genes that could cause human rejection and adding 6 human genes to enhance compatibility with the human immune system [1]