Dicerna Pharmaceuticals, Inc.

Cheng Lai

Bob Dale Brown

Henryk T Dudek

Natalie Wayne Pursell

Weimin Wang

Utsav Saxena

Naim Nazef

Rachel Storr

Boyoung Kim

This disclosure relates to oligonucleotides, compositions and methods useful for reducing LDHA expression, particularly in hepatocytes.

Methods and compositions for inhibiting expression of LDHA

This disclosure relates to oligonucleotides, compositions and methods useful for reducing CTNNB1 expression, particularly in hepatocytes, for the treatment of bile duct paucity-associated conditions. Disclosed oligonucleotides for the reduction of CTNNB1 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of CTNNB1 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver.

Methods and compositions for treating bile duct paucity-associated conditions

This disclosure relates to oligonucleotides, compositions and methods useful for reducing GYS2 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of GYS2 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of GYS2 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat glycogen storage diseases (e.g., GSDIa, GSDIII, GSDIV, GSDVI, and GSDIX) and related conditions.

Compositions and methods for inhibiting GYS2 expression

This invention relates to compounds, compositions, and methods useful for reducing lactate dehydrogenase target RNA and protein levels via use of ds RNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

Therapeutic inhibition of lactate dehydrogenase and agents therefor

This invention relates to compounds, compositions, and methods useful for reducing lactate dehydrogenase target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

This invention relates to compounds, compositions, and methods useful for reducing lactact dehydrogenase target RNA and protein levels via use of ds RNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

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Belmont, MA, United States of America

Cheng Lai