The results come from the phase 1b/2a RestorAATion-2 study of WVE-006, a GalNAc-conjugated oligonucleotide that is designed to correct a mutation in mRNA associated with AATD that prevents ...
With the exception of N-acetylgalactosamine (GalNAc), which mediates liver targeting, there are no safe and effective means of delivering oligonucleotide drugs to other relevant target cells and ...
The GalNAc platform is made up of two approaches ... Both STP705 and STP707 are made of two siRNA oligonucleotides that hit transforming growth factor beta 1 (TGF-β1) and cyclooxygenase 2 ...
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