Base Editing in DMD

Innovation Creates Hope

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August 22, 2025

GenAssist Ltd will Present Three Posters at the European Society of Gene and Cell Therapy (ESGCT) 2025 Annual Congress

GenAssist announced that three posters will be released at the at the 32nd ESGCT

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July 23, 2025

Safety Update of Global First DMD Base Editing Therapy—GEN6050X

GenAssist announced that the third patient enrolled in the IIT study of GEN6050X at Peking Union Medical College Hospital has successfully completed the 30-day follow-up with no serious adverse events reported

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June 24, 2025

GenAssist Ltd Receives U.S. FDA Rare Pediatric Disease Designation (RPDD) for GEN6050X in Treating Duchenne Muscular Dystrophy

GenAssist announced that the United States (U.S.) Food and Drug Administration (FDA) is P02506240

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Our Value

Our Vision: To Benefit Human Health through Innovation.
Our Mission: Transforming Cutting-edge Science into Life-changing Medicine.

Our Story

Founded in July 2020, GenAssist Ltd. (GenAssist) is a pioneering gene editing drug company. The operation entity of GenAssist is located in Suzhou, China, with two subsidiaries in Shanghai (China) and Boston (US). GenAssist owns the global rights of Targeted AID-mediated Mutagenesis cytosine base editor technology. The company is dedicated to developing base editing drugs against life-threatening diseases.

Our Pipeline

Utilizing TAM base editing technology, our product can precisely modify target DNA, inducing exon skipping, activating alternative splice sites, and editing splicing regulatory elements, with the ultimate goal of curing multiple diseases.

Our Platform

Our AAV Platform is highly versatile and modular, offering broad potential across therapeutic areas. We are already developing potential treatments spanning discovery to clinical-stage across multiple devastating diseases, and this is just the beginning.