IP Library Granted Patent US 11,001,845
Granted Patent B2
US 11,001,845 · App. 15/742,093 · Granted May 11, 2021

Nucleic acid molecules targeting superoxide dismutase 1 (SOD1)

Inventors: James Cardia (Franklin, MA); Michael Byrne (Natick, MA); Karen G. Bulock (Mendon, MA); Pamela A. Pavco (Longmont, CO); Lyn Libertine (Framingham, MA)
Assignee: Phio Pharmaceuticals Corp.
C12N15/1137A61K9/0019A61K31/713A61P25/28C12Y115/01001C12N2310/14
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Quick Facts
Patent No.
US 11,001,845
App. No.
15/742,093
Granted
May 11, 2021
Kind
B2
Abstract

Aspects of the invention relate to methods for treating ALS comprising administering to a subject in need thereof a therapeutically effective amount of a nucleic acid molecule that is directed against a gene encoding superoxide dismutase 1 (SOD1).

Claims (11)

1. An isolated double stranded nucleic acid molecule directed against superoxide dismutase 1 (SOD1) comprising an antisense strand and a sense strand, wherein the isolated double stranded nucleic acid molecule includes a double stranded region and a single stranded region, wherein the region of the molecule that is double stranded is 13, 14, or 15 nucleotides long, wherein the sense strand is 13, 14, or 15 nucleotides long, and wherein:

(i) the sense strand comprises SEQ ID NO: 2, SEQ ID NO: 32, or SEQ ID NO: 122, and the antisense strand comprises SEQ ID NO: 61, SEQ ID NO: 91, or SEQ ID NO: 123;

(ii) the sense strand comprises SEQ ID NO: 4, SEQ ID NO: 34, or SEQ ID NO: 126, and the antisense strand comprises SEQ ID NO: 63 or SEQ ID NO: 93;

(iii) the sense strand comprises SEQ ID NO: 9, SEQ ID NO: 38, or SEQ ID NO:135, and the antisense strand comprises SEQ ID NO: 68, SEQ ID NO: 97, or SEQ ID NO: 136;

(iv) the sense strand comprises SEQ ID NO: 10 or SEQ ID NO: 39, and the antisense strand comprises SEQ ID NO: 69 or SEQ ID NO: 98; or

(v) the sense strand comprises SEQ ID NO: 5, SEQ ID NO: 127 or SEQ ID NO: 137, and the antisense strand comprises SEQ ID NO: 64, SEQ ID NO: 128 or SEQ ID NO: 138.

2. The isolated double stranded nucleic acid molecule of claim 1 , wherein the isolated double stranded nucleic acid molecule further comprises a hydrophobic conjugate that is attached to the isolated double stranded nucleic acid molecule.

3. A composition comprising the isolated double stranded nucleic acid molecule of claim 1 .

4. The composition of claim 3 , further comprising a pharmaceutically acceptable carrier.

5. The composition of claim 3 further comprising a second therapeutic agent.

6. A method for treating amyotrophic lateral sclerosis (ALS) comprising administering to a subject in need thereof a therapeutically effective amount of the isolated double stranded nucleic acid molecule of claim 1 .

Assignments (2)
CHANGE OF NAME Recorded Dec 7, 2018
From: RXI PHARMACEUTICALS CORPORATION
To: PHIO PHARMACEUTICALS CORP.
Reel/Frame 048380/0009 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Nov 21, 2018
From: CARDIA, JAMES; BYRNE, MICHAEL; BULOCK, KAREN G.; PAVCO, PAMELA A.; LIBERTINE, LYN
To: RXI PHARMACEUTICALS CORPORATION
Reel/Frame 047616/0786 →
Continuity (2)
Provisional Application 62189050 · Jul 6, 2015
Related Publication 20180195072A1 · Jul 12, 2018
Cited By (1)
US 12,544,344