Complement factor b-modulating compositions and methods of use thereof
AssigneeADARx Pharmaceuticals, Inc.
InventorsZhen Li, Rui Zhu, Zhiqing (Joel) Zhou, Sean Studer, Kimberly Fultz, Jean da Silva Correia
Life of this patent
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Abstract
Aspects of the disclosure provide compounds, compositions, and methods for modulating the expression or activity of Complement Factor B (CFB). In certain aspects, the compounds, compositions, and methods of the disclosure can be used to reduce the expression of CFB mRNA in a. cell or animal. In certain aspects, the compounds, compositions, and methods of the disclosure can be used to reduce the expression of CFB protein in a cell or animal.
Claims84
A compound comprising a modified oligonucleotide 14 to 23 linked nucleosides in length having a nucleobase sequence comprising at least 14, at least 15, at least 16, at least 17, at least 18, at least 19, or at least 20 contiguous nucleobases of any one of the nucleobase sequences of SEQ ID NOs:
- 11-24, 26-44, 96, 99, and 101-102.
A compound comprising a modified oligonucleotide 14 to 23 linked nucleosides in length having a nucleobase sequence comprising the nucleobase sequence of any one of SEQ ID NOs:
- 11-24, 26-44, 96, 99, and 101-102.
A compound comprising a modified oligonucleotide having a nucleobase sequence selected from the nucleobase sequences of any one of SEQ ID NOs:
- 11-24, 26-44, 96, 99, and 101-102.
A compound comprising a first modified oligonucleotide 14 to 23 linked nucleosides in length having a nucleobase sequence comprising at least 14, at least 15, at least 16, at least 17, at least 18, at least 19, or at least 20 contiguous nucleobases of any of the nucleobase sequences of SEQ ID NOs:
- 11-24, 26-63, 65-96, 99, 101-105, 108, and 110, and a second modified oligonucleotide 14 to 23 linked nucleosides in length having a region of complementarity to the first modified oligonucleotide.
A compound comprising a first modified oligonucleotide 14 to 23 linked nucleosides in length having a nucleobase sequence comprising the nucleobase sequence of any one of SEQ ID NOs:
- 11-24, 26-63, 65-96, 99, 101-105, 108, and 110, and a second modified oligonucleotide 14 to 23 linked nucleosides in length having a region of complementarity to the first modified oligonucleotide.
A compound comprising a first modified oligonucleotide having a nucleobase sequence selected from the nucleobase sequences of any one of SEQ ID NOs:
- 11-24, 26-63, 65-96, 99, 101-105, 108, and 110, and a second modified oligonucleotide 19 to 23 linked nucleosides in length having a region of complementarity to the first modified oligonucleotide.
The compound of any one of the preceding claims, comprising a conjugate group.
A compound comprising a first modified oligonucleotide having a nucleobase sequence selected from any one of the nucleobase sequences of SEQ ID NOs:
- 11-24, 26-63, 65-96, 99, 101-105, 108, and 110, and a second modified oligonucleotide 14 to 21 linked nucleosides in length having a portion fully complementary to the first modified oligonucleotide.
A compound comprising a first modified oligonucleotide selected from any one of the Ref ID NOs:
- IA0478-490, IA0840, IA0843-846, IA0852-862, IA0872-882, IA0887-890, IA1010, IA1015, and IA1017-1018, and a second modified oligonucleotide 14 to 21 linked nucleosides in length fully complementary to the first modified oligonucleotide.
A compound comprising a first modified oligonucleotide selected from any one of the Ref ID NOs:
- IA0478-490, IA0840, IA0843-846, IA0852-862, IA0872-882, IA0887-890, IA1010, IA1015, and IA1017-1018, and a second modified oligonucleotide selected from any one of the Ref ID NOs:
- IS0570-587, IS0969, IS0972-973, IS0975-977, IS1023-1027, IS1029, IS1031-1037, IS1042-1046, IS1082, IS1085, IS1086, IS1091-1095, IS1108-1111, IS1236-1238, IS1249, and IS1253-1254.
A compound comprising a first modified oligonucleotide selected from any one of the Ref ID NOs:
- IA0840, IA0888, and IA1010, and a second modified oligonucleotide selected from any one of the Ref ID NOs:
- IS0972, IS1109, and IS1236.
45-46. (canceled)
A modified oligonucleotide according to the following chemical structure:
- or a pharmaceutically acceptable salt or stereoisomer thereof.
A modified oligonucleotide according to the following chemical structure:
- or a pharmaceutically acceptable salt or stereoisomer thereof.
A modified oligonucleotide according to the following chemical structure:
- or a pharmaceutically acceptable salt or stereoisomer thereof.
A modified oligonucleotide according to the following chemical structure:
- or a pharmaceutically acceptable salt or stereoisomer thereof.
51-52. (canceled)
54-55. (canceled)
60-62. (canceled)
A compound according to the following chemical structure:
- or a pharmaceutically acceptable salt or stereoisomer thereof.
A compound according to the following chemical structure:
- or a pharmaceutically acceptable salt or stereoisomer thereof.
(canceled)
(canceled)
(canceled)
87-95. (canceled)
Dependencies are inferred from the claim text.