公开(公告)号：US20240344061A1

公开(公告)日：2024-10-17

申请号：US18637630

申请日：2024-04-17

申请人： Omega Therapeutics, Inc.

发明人： Jesse Jerome Smith ,  Jodi Michelle Kennedy ,  Jeremiah D. Farelli ,  Kendrick Alan Goss ,  Adam Walter Scheidegger ,  Yoseph Kassa ,  Christian Wessel Cobaugh ,  Timsi Rao

IPC分类号： C12N15/11 ,  C12N9/22

CPC分类号： C12N15/11 ,  C12N9/22 ,  C07K2319/80 ,  C07K2319/81 ,  C12N2310/20

摘要： The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of a hepatocyte nuclear factor 4 alpha (HNF4α) gene by targeting an HNF4α expression control region and methods of use thereof for treating an HNF4α associated disorder, e.g., cirrhosis.

公开(公告)号：US20220348908A1

公开(公告)日：2022-11-03

申请号：US17029820

申请日：2020-09-23

申请人： Omega Therapeutics, Inc.

发明人： Jesse Jerome Smith ,  Jodi Michelle Kennedy ,  Jeremiah D. Farelli ,  Kendrick Alan Goss ,  Adam Walter Scheidegger ,  Yoseph Kassa ,  Christian Wessel Cobaugh ,  Timsi Rao

IPC分类号： C12N15/11 ,  C12N9/22

摘要： The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of a hepatocyte nuclear factor 4 alpha (HNF4a) gene by targeting an HNF4α expression control region and methods of use thereof for treating an HNF4α associated disorder, e.g., cirrhosis.

公开(公告)号：US11987791B2

公开(公告)日：2024-05-21

申请号：US17029820

申请日：2020-09-23

申请人： Omega Therapeutics, Inc.

发明人： Jesse Jerome Smith ,  Jodi Michelle Kennedy ,  Jeremiah D. Farelli ,  Kendrick Alan Goss ,  Adam Walter Scheidegger ,  Yoseph Kassa ,  Christian Wessel Cobaugh ,  Timsi Rao

IPC分类号： C12N15/11 ,  A61P1/16 ,  C07K14/47 ,  C12N9/22 ,  C12N15/63

CPC分类号： C12N15/11 ,  C12N9/22 ,  C07K2319/80 ,  C07K2319/81 ,  C12N2310/20

摘要： The present invention provides agents and compositions for modulating expression (e.g., enhanced or reduced expression) of a hepatocyte nuclear factor 4 alpha (HNF4α) gene by targeting an HNF4α expression control region and methods of use thereof for treating an HNF4α associated disorder, e.g., cirrhosis.

公开(公告)号：US20220348893A1

公开(公告)日：2022-11-03

申请号：US17754051

申请日：2020-09-23

申请人： OMEGA THERAPEUTICS, INC

发明人： Serena Lunardi ,  Adam Walter Scheidegger ,  Jesse Jerome Smith ,  Jeremiah Dale Farelli ,  Jodi Michelle Kennedy

IPC分类号： C12N9/22 ,  C12N15/11 ,  A61K38/46 ,  A61K31/7105 ,  A61P25/28 ,  C12N15/90 ,  C12N9/10

摘要： The present disclosure relates generally to methods and compositions for modulating frataxin (FXN) expression, e.g., to treat Friedreich ataxia (FRDA).