IP Library › Granted Patent US 12,703,865
Granted Patent B2
US 12,703,865 · App. 18/650,710 · Granted Aug 11, 2026

Small interfering RNA targeting C3 and uses thereof

Inventors: Chunyang Zhang (Burlington, MA); Xin Geng (Belmont, MA); Shiyu Wang (Belmont, MA); Weimin Wang (Winchester, MA)
Assignee: Sanegene Bio USA Inc.
C12N15/113A61P37/06C12N2310/11C12N2310/315C12N2310/321C12N2310/322
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Quick Facts
Patent No.
US 12,703,865
App. No.
18/650,710
Filed
Apr 30, 2024
Granted
Aug 11, 2026
Kind
B2
Art Unit
1637
USPC
514/44A
Abstract

This disclosure relates to isolated oligonucleotides comprising duplex regions targeting human complement C3 mRNA, and delivery systems, kits and compositions comprising the same, and methods of using the same for inhibiting or downregulating C3 gene expression.

Claims (23)

1 . An isolated oligonucleotide comprising:

an antisense strand comprising the nucleic acid sequence 5′

[mUs][fUs][fA][mG][fU][mA][fG][mA][mA][fU][mU][mU][mC][fU][mC][fU][mG][mU][mA][mGs][mGs][mC] 3′ (SEQ ID NO: 451), and

a sense strand comprising the nucleic acid sequence 5′

[mCs][mUs][mA][mC][mA][fG][mA][fG][fA][fA][fA][mU][mU][mC][mU][mA][mC][mUs][mAs][mA][G1b][G1b][G1b] 3′ (SEQ ID NO: 444),

wherein “m” is a 2′-O-methyl modification, “f” is a 2′-F modification, “s” is a phosphorothioate internucleotide linkage, and “G1b” is a GalNAc G1b moiety.

2 . A delivery system comprising the isolated oligonucleotide of claim 1 , wherein the delivery system is a liposome, a nanoparticle, a polymer-based delivery system, a ligand-conjugate delivery system, or a combination thereof.

3 . A pharmaceutical composition comprising the isolated oligonucleotide of claim 1 , and a pharmaceutically acceptable carrier, diluent or excipient.

4 . An isolated oligonucleotide comprising an antisense strand and a sense strand:

the antisense strand consisting of the nucleic acid sequence 5′ [mUs][fUs][fA][mG][fU][mA][fG][mA][mA][fU][mU][mU][mC][fU][mC][fU][mG][mU][mA][mGs][mGs][mC] 3′ (SEQ ID NO: 451), and

the sense strand consisting of the nucleic acid sequence 5′

[mCs][mUs][mA][mC][mA][fG][mA][fG][fA][fA][fA][mU][mU][mC][mU][mA][mC][mUs][mAs][mA][G1b][G1b][G1b] 3′ (SEQ ID NO: 444),

wherein “m” is a 2′-O-methyl modification, “f” is a 2′-F modification, “s” is a phosphorothioate internucleotide linkage, and “G1b” is a GalNAc G1b moiety.

5 . A delivery system comprising the isolated oligonucleotide of claim 4 , wherein the delivery system is a liposome, a nanoparticle, a polymer-based delivery system, a ligand-conjugate delivery system, or a combination thereof.

6 . A pharmaceutical composition comprising the isolated oligonucleotide of claim 4 , and a pharmaceutically acceptable carrier, diluent or excipient.

7 . An isolated oligonucleotide consisting of an antisense strand and a sense strand:

the antisense strand consisting of the nucleic acid sequence 5′

[mUs][fUs][fA][mG][fU][mA][fG][mA][mA][fU][mU][mU][mC][fU][mC][fU][mG][mU][mA][mGs][mGs][mC] 3′ (SEQ ID NO: 451), and

the sense strand consisting of the nucleic acid sequence 5′

[mCs][mUs][mA][mC][mA][fG][mA][fG][fA][fA][fA][mU][mU][mC][mU][mA][mC][mUs][mAs][mA][G1b][G1b][G1b] 3′ (SEQ ID NO: 444),

wherein “m” is a 2′-O-methyl modification, “f” is a 2′-F modification, “s” is a phosphorothioate internucleotide linkage, and “G1b” is a GalNAc G1b moiety.

8 . A delivery system comprising the isolated oligonucleotide of claim 7 , wherein the delivery system is a liposome, a nanoparticle, a polymer-based delivery system, a ligand-conjugate delivery system, or a combination thereof.

9 . A pharmaceutical composition comprising the isolated oligonucleotide of claim 7 , and a pharmaceutically acceptable carrier, diluent or excipient.

Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Dec 18, 2025
From: SANEGENE BIO INC.
To: SANEGENE BIO USA INC.
Reel/Frame 073966/0761 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded May 1, 2024
From: ZHANG, CHUNYANG; GENG, XIN; WANG, SHIYU; WANG, WEIMIN
To: SANEGENE BIO USA INC.
Reel/Frame 067279/0112 →
Continuity (3)
Continuation 18487663 · Oct 16, 2023
Provisional Application 63416070 · Oct 14, 2022
Related Publication 20240301421A1 · Sep 12, 2024
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