IP Library › Granted Patent US 9,562,230
Granted Patent B2
US 9,562,230 · App. 14/801,161 · Granted Feb 7, 2017

Transferrin/transferrin receptor-mediated siRNA delivery

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Quick Facts
Patent No.
US 9,562,230
App. No.
14/801,161
Granted
Feb 7, 2017
Kind
B2
Abstract

The invention provides interfering RNA molecule-ligand conjugates useful as a delivery system for delivering interfering RNA molecules to a cell in vitro or in vivo. The conjugates comprise a ligand that can bind to a transferrin receptor (TfR). Therapeutic uses for the conjugates are also provided.

Claims (15)

1. A method of delivering an interfering RNA molecule to a patient, comprising administering to the patient an interfering RNA molecule-ligand conjugate wherein the ligand comprises fusion protein containing:

a) a transferrin (TfR)-specific antibody or antibody fragment and

b) a transferrin (Tf) or a less than full length Tf that contains a TfR-binding domain.

2. The method of claim 1 , wherein the ligand comprises a TfR binding domain of transferrin.

3. The method of claim 2 , wherein the ligand comprises the Tf C-lobe or a TfR-binding portion thereof.

4. The method of claim 1 , wherein the ligand comprises a TfR-binding peptide, a TR-binding aptamer, or a TfR-specific antibody or a fragment thereof.

5. The method of claim 1 , wherein the conjugate is administered by intraocular injection, ocular topical application, subconjunctival injection, intravitreal injection, or anterior or posterior juxtascleral injection.

6. The method of claim 1 , wherein the interfering RNA molecule is a siRNA, miRNA, or shRNA.

7. A composition comprising: interfering RNA molecule-ligand conjugate wherein the ligand comprises a fusion protein containing:

a) a transferrin (TfR)-specific antibody or antibody fragment and

b) a transferrin or a less than full length Tf that contains a TfR-binding domain.

8. The composition of claim 7 , wherein the ligand comprises a TfR binding domain of transferrin.

9. The composition of claim 8 , wherein the ligand comprises the Tf C-lobe or a TfR-binding portion thereof.

10. The composition of claim 7 , wherein the ligand comprises a TfR-binding peptide, a TfR-binding aptamer, or a TfR-specific antibody or a fragment thereof.

11. The composition of claim 7 , wherein the interfering RNA molecule is a siRNA, miRNA, or shRNA.

Assignments (2)
SECURITY INTEREST Recorded Aug 7, 2024
From: ARROWHEAD PHARMACEUTICALS, INC.
To: SIXTH STREETLENDING PARTNERS, AS THE ADMINISTRATIVE AGENT
Reel/Frame 068510/0363 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jun 28, 2016
From: CHATTERTON, JON E.; CLARK, ABBOT F.
To: ARROWHEAD PHARMACEUTICALS, INC.
Reel/Frame 039185/0589 →