GenAssist Ltd
- Biotech or pharma, therapeutic R&D
GenAssist Ltd. (GenAssist) is a pioneering gene editing drug company with the operation entity located in China and a subsidiary in Boston (US). The company is dedicated to developing base editing drugs against life-threatening diseases. Duchenne Muscular Disease (DMD) is the first therapeutic area. Our first base editing DMD drug, GEN6050X, got IND approval from FDA in Mar 2025. This is the first gene editing DMD drug approved by FDA. Also, the IIT data of GEN6050X in China showed positive motor and cardiac function improvement as well as dystrophin protein restoration. This marks the milestone of DMD gene therapy. The company has built a human genome-displaying AAV platform with 10B size and achieved multiple new AAV capsids specific to muscles, immune cell, CNS and lung. The muslce tropism and liver-detargeting AAV capsid will be applied to our next-generation DMD pipeline.
We are not in the position to meet CRO/CDMO at this BIO!
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