REGN - Intellia CRISPR gene editing data puts heat on Alnylam; shares down 5%

wildpixel/iStock via Getty Images Shares of Alnylam Pharmaceuticals (ALNY) are under pressure today following this past weekend's release of data on a CRISPR gene editing candidate from Intellia (NTLA) for a liver disorder. The interim Phase 1 data on CRISPR candidate NTLA-2001 showed that it was able to genetically edit cells inside the liver. It is under development for transthyretin amyloidosis ("ATTR"). Alnylam currently markets Onpattro (patisiran), an RNAi therapy for ATTR. In 2020, Onpattro revenues were $306M. Today, Alnylam announced that the FDA accepted its NDA for for vutrisiran, an RNAi therapeutic for polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis. Stifel analyst Paul Matteis said that while Intellia's results will likely have companies engaged in oligonucleotide therapeutics examining their competitive position, "it's premature for Alnylam shareholders to get spooked here." Guggenheim notes that while vutrisiran is dosed once a quarter, NTLA-2001 has the potential to become a "one-time curative therapy," adding that the

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Intellia CRISPR gene editing data puts heat on Alnylam; shares down 5%