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BioMarin AAV gene therapy trial halted

The FDA has halted testing of BioMarin's AAV based gene therapy drug after liver cancer developed in treated mice. BioMarin's BMN_307 drug is employs an AAV5 vector to treat patients suffering from phenylketonuria. The Phase 1/2 study in patients has been halted after cancers were identified in preclinical mice treated with a high dose of the vector. 5 mice treated developed adenomas and one developed hepatocellular carcinoma. The need to translate the findings to humans is needed. TestAVec's hInGeTox platform provides an in vitro system to test vectors and we are happy to discuss your needs to test your vector in the first patented human in vitro platform to develop safety profile testing of your vector.


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