Pre-Clinical Data with ALN-GO1, in Development for the Treatment of Primary Hyperoxaluria Type 1

Pre-Clinical Data with ALN-GO1, in Development for the Treatment of Primary Hyperoxaluria Type 1

We presented new pre-clinical data supporting the selection of a Development Candidate (DC) for ALN-GO1, an investigational RNAi therapeutic targeting glycolate oxidase (GO), also referred to as hydroxyacid oxidase 1 (HAO1), for the treatment of primary hyperoxaluria type 1 (PH1).  These data were presented at the 48th European Society of Paediatric Nephrology (ESPN) Annual meeting – held September 3 – 5, 2015, in Brussels – showing up to 99% silencing of the HAO1 mRNA and up to 98% mean reduction of urinary oxalate in animal models of PH1.



We believe that ALN-GO1 represents a truly innovative approach for the treatment of PH1, an ultra-rare orphan disease with enormous unmet need as there are no approved therapeutics available.  From our ongoing human experience with ESC-GalNAc conjugates, we believe that ALN-GO1 has the potential to achieve a once monthly, and possibly once quarterly, subcutaneous dose regimen with a favorable tolerability profile. We look forward to the continued advancement of this novel investigational therapeutic.



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