Condition: Gaucher Disease Type 1 · Sponsor: Lingyi Biotech Co., Ltd.
Gaucher disease (GD) is caused by mutations in the GBA1 gene, which leads to a lack or reduction of GCase activity. The consequences of this deficiency are generally attributed to the accumulation of the GCase substrate, Glucosylceramide (GlcCer), in macrophages in the liver, spleen, kidney, bone, lung, and even the brain, inducing their transformation into Gaucher cells whose cell cytoplasm presenting a characteristic "crumpled tissue paper" appearance, leading to pathological changes in involved tissues and organs.LY-M001 Injection is an rAAV8 vector gene therapy product. It can specifically transduce the target organ liver after a single intravenous administration and express the GCase protein in liver cells for a long period of time.
This description comes directly from the study's public registry record.
Qing Lin, PhD · 86+19121572057 · qing.lin@lingyimed.com
Yixiong Chen, PhD · 86+19121572057 · yixiong.chen@lingyimed.com
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| Guangzhou First People's Hospital | Guangzhou, Guangdong, China | Recruiting |
| Shanxi Bethune Hospital | Taiyuan, Shanxi, China | Recruiting |
| Hematology Hospital, Chinese Academy of Medical Sciences | Tianjin, Tianjin Municipality, China | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06818838