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Pre-Clinical Results with RNAi Therapeutics Targeting Aminolevulinic Acid Synthase-1 (ALAS-1) Published in Proceedings of the National Academy of Sciences (PNAS)

We have published pre-clinical results with RNAi therapeutics targeting aminolevulinic acid synthase-1 (ALAS-1) for the treatment of hepatic porphyrias in the Proceedings of the National Academy of Sciences (PNAS). The new paper highlights work that was done in collaboration with researchers at the Icahn School of Medicine at Mount Sinai in New York City. Data from studies performed in a mouse model of acute intermittent porphyria (AIP) demonstrate that RNAi therapeutics targeting ALAS-1 can achieve potent, rapid, and durable suppression of aminolevulinic acid (ALA) and porphobilinogen (PBG), the toxic heme biosynthesis intermediates that cause the symptoms and disease pathology of AIP.



These findings, as reported in PNAS, provide key pre-clinical proof-of-concept data for our ALN-AS1 program for the treatment of hepatic porphyrias, including AIP. We believe that ALN-AS1 has the potential to be a transformative therapy for patients with AIP, an ultra-rare genetic disease with significant unmet medical need. A subcutaneously administered RNAi therapeutic targeting ALAS-1 has the potential to be used as a prophylactic approach to prevent attacks associated with AIP, and also as a therapy for acute attacks.