
siRNA Drug Developer
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Recently, according to the announcement on the CDE official website, the VSA006 injection developed by Visirna Biotechnology (Shanghai) Co., Ltd. ("Visirna") has received clinical trial approval by default and is proposed for development to treat adult patients with non-alcoholic steatohepatitis (NASH).

Nonalcoholic Steatohepatitis (NASH), also known as metabolic steatohepatitis, is a highly prevalent and difficult-to-treat chronic liver disease, commonly occurring in middle-aged individuals, especially those who are overweight or obese. Currently, there is a lack of highly effective therapies specifically targeting NASH indications in clinical practice.Studies show that gene mutations in HSD17β13 are associated with the development of non-alcoholic steatohepatitis (NASH).
AndVSA006 is a small nucleic acid drug that canSpecifically binds to HSD17β13 mRNA in hepatocytes, downregulates the expression of HSD17β13 protein, and thereby inhibits the progression of non-alcohol-related liver disease.

Visirna is a small nucleic acid drug therapy company based in China with a global outlook, aiming to build a biopharmaceutical enterprise with comprehensive capabilities in research and development, production, and commercialization. The company was founded in 2022 and has established a long-term strategic partnership with Arrowhead Pharmaceuticals (NASDAQ: ARWR), a leading international small nucleic acid drug company. Currently, the company's pipeline includes three small nucleic acid (siRNA) drugs in clinical development targeting cardiovascular and metabolic diseases. The company’s existing pipeline is in a leading position among similar competing products, with its investigational targets supported by clear genomic and biological evidence, utilizing Arrowhead Pharmaceuticals' proven chemical modification and delivery technology platform.
R&D Pipeline

References:Visirna