IP Library Patent Application 17615695
Patent Application
App. No. 17/615,695

APTAMERS AGAINST TRANSFERRIN RECEPTOR (TfR)

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Quick Facts
Patent No.
US None
App. No.
17/615,695
Abstract

Methods of treating or preventing a disease or disorder are disclosed comprising administering to a subject in need thereof an effective amount of a nucleic acid compound comprising, or consisting of, a nucleic acid sequence capable of binding to a transferrin receptor (TfR) and an effective amount of an inhibitor of DNA synthesis. Also disclosed is a nucleic acid compound comprising, or consisting of, a nucleic acid sequence having at least 85% sequence identity to SEQ ID NO: 1, wherein said nucleic acid sequence is at least 30 nucleotides in length and at most 50 nucleotides in length, and wherein the nucleic acid sequence is capable of binding to a transferrin receptor (TfR).

Claims (40)

1 . (canceled)

2 . (canceled)

3 . A method of treating or preventing a disease or disorder in a subject in need thereof, the method comprising administering simultaneously or sequentially to the subject an effective amount of a nucleic acid compound and an effective amount of an inhibitor of DNA synthesis, wherein said nucleic acid compound comprises, or consists of a nucleic acid sequence capable of binding to a transferrin receptor (TfR).

4 . The method according to claim 3 , wherein said nucleic acid sequence has at least 95% sequence identity to a sequence selected from SEQ ID NOs: 1 to 6.

5 . (canceled)

6 . The method according to claim 3 , wherein the nucleic acid sequence has a length of 22 nucleotides.

7 . The method according to claim 3 , wherein the inhibitor of DNA synthesis is a nucleoside analogue, and wherein the nucleoside analogue is gemcitabine.

8 . (canceled)

9 . The method according to claim 3 , wherein the disease or disorder is cancer.

10 . The method according to claim 9 , wherein the cancer is pancreatic cancer.

11 . The method according to claim 3 , wherein the nucleic acid compound further comprises a moiety attached to said nucleic acid sequence, wherein said moiety is a therapeutic moiety.

12 . (canceled)

13 . The method according to claim 11 , wherein said therapeutic moiety is covalently attached to said nucleic acid sequence.

14 . The method according to claim 13 , wherein said therapeutic moiety is an anticancer therapeutic moiety, and wherein said therapeutic moiety is a C/EBPalpha saRNA moiety, a SIRT1 saRNA moiety, or a HNF saRNA moiety.

15 . (canceled)

16 . A nucleic acid compound comprising, or consisting of, a nucleic acid sequence having at least 95% sequence identity to SEQ ID NO: 5, wherein said nucleic acid sequence is at least 30 nucleotides in length and at most 50 nucleotides in length, and wherein the nucleic acid sequence is capable of binding to a transferrin receptor (TfR).

17 . The nucleic acid compound according to claim 16 , wherein said nucleic acid sequence comprises or consists of a sequence having at least 95% sequence identity to SEQ ID NO: 2 or 4.

18 . (canceled)

19 . (canceled)

20 . (canceled)

21 . (canceled)

22 . (canceled)

23 . (canceled)

24 . The nucleic acid compound according to claim 16 , wherein the nucleic acid sequence is capable of binding to both TfR1 and TfR2.

25 . The nucleic acid compound according to claim 16 , further comprising a moiety attached to said nucleic acid sequence, wherein said moiety is a therapeutic moiety.

26 . (canceled)

27 . (canceled)

28 . (canceled)

29 . The nucleic acid compound according to claim 25 , wherein said moiety is a C/EBPalpha saRNA moiety, a SIRT1 saRNA moiety, or a HNF saRNA moiety.

30 . (canceled)

31 . A pharmaceutical composition comprising a nucleic acid compound according to claim 16 , optionally comprising a pharmaceutically acceptable excipient.

32 . A method of delivering a compound moiety into a cell, the method comprising:

a. contacting a cell with the nucleic acid compound according to claim 16 ; and

b. allowing said nucleic acid compound to bind to a transferrin receptor on said cell and pass into said cell thereby delivering said compound moiety into said cell.

33 . (canceled)

34 . (canceled)

35 . (canceled)

36 . A method of treating or preventing a disease or disorder, the method comprising administering to a subject in need thereof an effective amount of a nucleic acid compound according to claim 16 .

37 . The method according to claim 36 , wherein the disease or disorder is cancer, and wherein the method further comprises administering to the subject an anticancer agent.

38 . (canceled)

Assignments (3)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 30, 2025
From: SWIDERSKI, PIOTR MAREK
To: CITY OF HOPE
Reel/Frame 072427/0947 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 3, 2022
From: YOON, SORAH; ROSSI, JOHN J.; HABIB, NAGY
To: APTERNA LIMITED
Reel/Frame 059156/0881 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Dec 1, 2021
From: YOON, SORAH; ROSSI, JOHN J.; HABIB, NAGY
To: CITY OF HOPE; APTERNA LIMITED
Reel/Frame 058262/0702 →