IP Library Granted Patent US 11,339,394
Granted Patent B2
US 11,339,394 · App. 16/448,498 · Granted May 24, 2022

Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene

Inventors: Joseph Collard (Delray Beach, FL); Olga Khorkova Sherman (Tequesta, FL)
Assignee: CURNA, INC.
C12N15/113A61K31/713A61L27/3633C07K14/78C12N2310/11C12N2310/113C12N2310/14C12N2310/31C12N2310/313C12N2310/315C12N2310/319C12N2310/3181C12N2310/3231
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Quick Facts
Patent No.
US 11,339,394
App. No.
16/448,498
Granted
May 24, 2022
Kind
B2
Abstract

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of a Collagen gene, in particular, by targeting natural antisense polynucleotides of a Collagen gene. The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of Collagen genes.

Claims (14)

1. A synthetic, modified oligonucleotide of 20-27 nucleotides in length comprising at least one modification wherein the at least one modification is selected from: at least one modified sugar moiety; at least one modified internucleotide linkage; at least one modified nucleotide, and combinations thereof; wherein said oligonucleotide is an antisense compound which is 100% complementary with and specifically hybridizes to a natural antisense polynucleotide of a Collagen gene polynucleotide having SEQ ID NO: 3, said natural antisense polynucleotides having SEQ ID NOS: 6, 8 or 9 and upregulates the function and/or expression of said Collagen gene having SEQ ID NO: 3 in vivo or in vitro as compared to a normal control.

2. The oligonucleotide of claim 1 , wherein the at least one modification comprises an internucleotide linkage selected from the group consisting of phosphorothioate, alkylphosphonate, phosphorodithioate, alkylphosphonothioate, phosphoramidate, carbamate, carbonate, phosphate triester, acetamidate, carboxymethyl ester, and combinations thereof.

3. The oligonucleotide of claim 1 , wherein said oligonucleotide comprises at least one phosphorothioate internucleotide linkage.

4. The oligonucleotide of claim 1 , wherein said oligonucleotide comprises a backbone of phosphorothioate internucleotide linkages.

5. The oligonucleotide of claim 1 , wherein the oligonucleotide comprises at least one modified nucleotide, said modified nucleotide selected from: a peptide nucleic acid, a locked nucleic acid (LNA), analogue, derivative, and a combination thereof.

6. The oligonucleotide of claim 1 , wherein the oligonucleotide comprises a plurality of modifications, wherein said modifications comprise modified nucleotides selected from: phosphorothioate, alkylphosphonate, phosphorodithioate, alkylphosphonothioate, phosphoramidate, carbamate, carbonate, phosphate triester, acetamidate, carboxymethyl ester, and a combination thereof.

7. The oligonucleotide of claim 1 , wherein the oligonucleotide comprises a plurality of modifications, wherein said modifications comprise modified nucleotides selected from: peptide nucleic acids, locked nucleic acids (LNA), and a combination thereof.

8. The oligonucleotide of claim 1 , wherein the oligonucleotide comprises at least one modified sugar moiety selected from: a 2′-O-methoxyethyl modified sugar moiety, a 2′-methoxy modified sugar moiety, a 2′-O-alkyl modified sugar moiety, a bicyclic sugar moiety, and a combination thereof.

9. The oligonucleotide of claim 1 , wherein the oligonucleotide comprises a plurality of modifications, wherein said modifications comprise modified sugar moieties selected from: a 2′-O-methoxyethyl modified sugar moiety, a 2′-methoxy modified sugar moiety, a 2′-O-alkyl modified sugar moiety, a bicyclic sugar moiety, and a combination thereof.

10. The oligonucleotide of claim 1 , wherein said oligonucleotide hybridizes to said natural antisense polynucleotide and upregulates the expression and/or function of at least one Collagen gene polynucleotide in vivo or in vitro, as compared to a normal control selected from a mock-transfected control.

11. The oligonucleotide of claim 1 , wherein the oligonucleotide consists essentially of any one of the sequences set forth as SEQ ID NOS: 22, 25 or 28.

12. A composition comprising one or more oligonucleotides specific for one or more Collagen gene polynucleotides according to claim 1 and a pharmaceutically acceptable excipient.

13. The composition of claim 12 , wherein the oligonucleotides consists essentially of any one of the nucleotide sequences set forth as SEQ ID NOS: 22, 25 or 28.

14. The composition of claim 12 , wherein the one or more modifications are selected from: phosphorothioate, methylphosphonate, peptide nucleic acid, locked nucleic acid (LNA) molecules, and combinations thereof.

Assignments (3)
CHANGE OF NAME Recorded Mar 25, 2022
From: OPKO CURNA, LLC
To: CURNA, INC.
Reel/Frame 059403/0618 →
CORRECTIVE ASSIGNMENT TO CORRECT THE ASSIGNEE NAME PREVIOUSLY RECORDED AT REEL: 051523 FRAME: 0212. ASSIGNOR(S) HEREBY CONFIRMS THE ASSIGNMENT . Recorded Mar 3, 2022
From: COLLARD, JOSEPH; KHORKOVA SHERMAN, OLGA
To: OPKO CURNA, LLC
Reel/Frame 059310/0235 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jan 15, 2020
From: COLLARD, JOSEPH; KHORKOVA SHERMAN, OLGA
To: CURNA, INC.
Reel/Frame 051523/0212 →
Continuity (6)
Division 15364050 · Nov 29, 2016
Continuation 13377888
Provisional Application 61286939 · Dec 16, 2009
Provisional Application 61286965 · Dec 16, 2009
Provisional Application 61187384 · Jun 16, 2009
Related Publication 20190309295A1 · Oct 10, 2019