Dicerna Pharmaceuticals, Inc.

Novo Nordisk A/s

Natalie Wayne Pursell

Henryk T Dudek

Cheng Lai

Bob Dale Brown

Utsav Saxena

Weimin Wang

Naim Nazef

Boyoung Kim

Rachel Storr

Birgitte Andersen

Wen Han

Nicole Alexis Spiegelman

Markus Robert Latta

William Geoffrey Haynes

Zhihao Ding

Chengjung Lai

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

Methods and compositions for inhibiting expression of LDHA

Oligonucleotides are provided herein that inhibit MARC1 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with MARC1 expression.

Compositions and methods for inhibiting mitochondria amidoxime reducing component 1 (MARC1) expression

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

Oligonucleotides are provided herein that inhibit NR1H3 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with NR1H3 expression.

Compositions and methods for inhibiting nuclear receptor subfamily 1 group H member 3 (NR1H3) expression

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

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Westborough, MA, United States of America

Natalie Wayne Pursell