Dicerna Pharmaceuticals, Inc.
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Latest From Dicerna Pharmaceuticals, Inc.
Alnylam shows that lumasiran offers similar safety and efficacy in primary hyperoxaluria type 1 patients who are six or younger as it does in older patients. US and EU regulatory decisions loom.
The top-line data in the ultra-rare disease are also the first to demonstrate safety and efficacy of an RNAi therapy in patients six years and younger.
The UK's Silence Therapeutics is moving its candidate products into early clinical studies in cardiovascular diseases and improving its visibility with a NASDAQ listing.
At EASL 2020, four partnerships updated RNA-interference or antisense efforts to reduce hep B surface antigen, believed to lead to functional cure of the virus. One analyst sees J&J/Arrowhead as the early leader.
- Antisense, Oligonucleotides
- Large Molecule