Alnylam Pharmaceuticals Inc (ALNY)

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“At Alnylam, we’ve been successful in advancing our ESC GalNAc-siRNA conjugate platform with many potent and generally well tolerated investigational RNAi therapeutics in clinical development, including in Phase 3 studies. Nevertheless, we continue to strive to even further optimize our RNAi therapeutics platform to achieve improved target specificity and an even greater therapeutic index. Accordingly, we were pleased to share these new pre-clinical results at this year's OTS meeting, highlighting our ESC+ GalNAc conjugate platform,” said Kevin Fitzgerald, Ph.D., Senior Vice President, Research at Alnylam. “Our goal is to employ the ESC+ chemistry in all future development candidates, beginning with ALN-AAT02, which we plan to advance into clinical development in 2018. We believe that by implementing platform improvements that preemptively address potential off-target effects we can further enhance the therapeutic index of our investigational RNAi therapeutics for all future programs in our pipeline.”