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Gene Therapy Based Precision Medicine Therapeutics: Clinical Pharmacology & Translational Science Considerations

The era of gene therapy may have started a couple of decades ago, but approvals of agents based on the platform have been relatively recent. In 2017, Spark Therapeutics, Inc. received FDA approval of voretigene neparvovec-rzyl (Luxturna™), a recombinant adeno-associated virus serotype 2 (AAV2) vector expressing the gene for human retinal pigment epithelium 65 kDa … Continued

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