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Alnylam submits clinical trial authorization (CTA) application for ALN-HSD for the Treatment of Nonalcoholic Steatohepatitis (NASH)
3 Aug 2020
ALN-HSD (siRNA targeting HSD17B13) – Alnylam Pharma/ Regeneron
- Submitted a clinical trial authorization (CTA) application to the medicines and healthcare products regulatory agency (MHRA) in UK to initiate a Phase 1 study of ALN-HSD for the treatment of nonalcoholic steatohepatitis (NASH)
- Study initiation expected in late 2020, upon obtaining MHRA approval
Alnylam to initiate trial for its first RNAi in NASH in 2020
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CI Scientists Remarks:
- In 2018, Alnylam and Regeneron entered into a fifty-fifty collaboration to research, co-develop and commercialize ALN-HSD is an investigational, subcutaneously administered RNAi therapeutic, targeting HSD17B13 gene for the treatment of nonalcoholic steatohepatitis
- Regeneron identified for the first time a variant in the HSD17B13 gene that is associated with reduced risk of chronic liver diseases and Alnylam leveraged its RNAi therapeutics platform to identify the compound (ALN-HSD) directed to this gene
- ALN-HSD utilizes Alnylam’s Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, which enables subcutaneous dosing with increased selectivity and a wide therapeutic index
– Tarun Raisinghani, CI Scientists