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.
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The introduction of gene therapies has been the highest-profile new modality to reach the market, but new RNA-based therapeutics hold significant potential and could be more accessible for patients. Scrip reviews the leading drug developers in this space.
Givosiran was approved by the US FDA for the treatment of acute hepatic porphyria, a rare generic blood disorder, nearly three months ahead of the action date.
Gilead detailed its Phase III failure with ASK1 inhibitor selonsertib in NASH, but anticipates finding a new regimen to take into Phase III in NASH with the ATLAS combination study data expected in December.