Workflow
iPSC来源的CAR-NK细胞疗法
icon
Search documents
Cell重磅:全球首例!启函生物iPSC-CAR-NK细胞疗法成功治疗一种难治性自身免疫疾病
生物世界· 2025-06-24 23:52
Core Viewpoint - The article discusses a significant breakthrough in the treatment of systemic sclerosis (SSc) through the use of an iPSC-derived CD19/BCMA CAR-NK cell therapy, QN-139b, which has shown promising results in a patient with refractory diffuse cutaneous systemic sclerosis (dcSSc) [2][4][10]. Group 1: Research and Development - Systemic sclerosis is characterized by vascular abnormalities, immune activation, and progressive tissue fibrosis, presenting challenges in clinical treatment due to limited drug efficacy and high disease heterogeneity [2]. - The study published in Cell highlights the first successful clinical translation of iPSC-derived CAR-NK therapy in a patient with dcSSc, marking a global first in this area [3][4]. - QN-139b targets both CD19 and BCMA to eliminate pathogenic B cells and plasma cells, developed using a high-throughput gene editing technology platform [6]. Group 2: Clinical Application - A 36-year-old female patient with a 20-year history of severe dcSSc was treated with QN-139b, receiving a total of four doses [8][9]. - After six months of follow-up, the patient exhibited significant clinical improvement, including a notable decrease in autoantibodies and a reduction in the modified Rodnan skin score (mRSS) [9][10]. - The therapy demonstrated good overall safety, with no significant adverse effects reported during the treatment period [9][10]. Group 3: Expert Opinions - Professor Xu Huji, leading the clinical team, emphasized the strong research capabilities and technological platform of Qihang Biotech, expressing confidence in the partnership [12]. - Academician Pei Gang highlighted the innovative advancements in universal off-the-shelf cell therapies, indicating a new direction for treating autoimmune diseases [12][14]. - Dr. Yang Luhan, CEO of Qihang Biotech, expressed satisfaction with the clinical outcomes and reiterated the company's commitment to providing better solutions for patients with autoimmune diseases [13][14].