Alnylam Interview: RNAi Leader Expands Into NASH, CNS And Eye Diseases

Buoyed By $1bn Regeneron Alliance

Feature article: Alnylam’s R&D leader Akshay Vaishnaw sets out plans for next-generation gene-silencing therapies.

Alnylam
Alnylam's Boston, MA headquarters. It will take another 5-10 years for Alnylam to enter biopharma's 'big league' - but it has already put RNAi on the map • Source: Photo Courtesy of BioMed Realty, L.P. All Rights Reserved

Alnylam enjoyed a very successful year in 2019, gaining US approval for its second RNA interference (RNAi) therapy, Givlaari (givosiran), with two more of its drug progressing towards regulatory filing.

The firm was the originator of inclisiran, developed by The Medicines Company, which has just been acquired by Novartis, and the Swiss major hopes to turn the cholesterol-lowering RNAi drug into a blockbuster. Meanwhile, lumisiran, Alnylam’s next in-house molecule, produced pivotal data in primary hyperoxaluria type 1 (PH1), and both drugs are on track for early 2020 filing and approval later this year

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