Granted Patent
B2
US 12,263,224 · App. 18/056,664 · Granted Apr 1, 2025
Compositions and methods of treating muscle atrophy and myotonic dystrophy
Inventors:
Andrew John Geall (Carlsbad, CA); Venkata Doppalapudi (San Diego, CA); David Sai-Ho Chu (La Jolla, CA); Michael Caramian Cochran (La Jolla, CA); Michael Hood (San Diego, CA); Beatrice Diana Darimont (San Diego, CA); Rob Burke (Encinitas, CA); Yunyu Shi (San Diego, CA); Gulin Erdogan Marelius (San Diego, CA); Barbora Malecova (La Jolla, CA)
Assignee:
AVIDITY BIOSCIENCES, INC.
A61K47/6807A61K31/712A61K31/713A61K39/395A61K47/6849A61P21/00C07K16/18C12N15/113A61K9/5107C12N2310/14C12N2310/315C12N2310/317C12N2310/3513C12N2310/3515C12N2320/31C12N2320/32
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Abstract
Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle atrophy or myotonic dystrophy.
Claims (18)
1. An antisense oligonucleotide (ASO)-antibody conjugate comprising an anti-transferrin receptor antibody or antigen-binding fragment thereof conjugated to an ASO that hybridizes to a target sequence of human Murf1 mRNA and mediates RNA interference against the human Murf1 mRNA in a muscle cell, and wherein the ASO comprises a sequence selected from SEQ ID NOs: 651-702.
2. The ASO-antibody conjugate of claim 1 , wherein the ASO comprises at least one 2′ modified nucleotide, at least one modified internucleotide linkage, or at least one inverted abasic moiety.
3. The ASO-antibody conjugate of claim 1 , wherein the mediation of RNA interference against the human Murf1 mRNA in the muscle cell modulates muscle atrophy or myotonic dystrophy in a subject.
4. The ASO-antibody conjugate of claim 1 , wherein the anti-transferrin receptor antibody or antigen-binding fragment thereof binds to a transferrin receptor on a cell surface of the muscle cell.
5. The ASO-antibody conjugate of claim 1 , wherein the muscle cell is a skeletal muscle cell or a cardiac muscle cell.
6. The ASO-antibody conjugate of claim 1 , wherein the ASO is from 19 to 30 nucleotides in length.
7. The ASO-antibody conjugate of claim 1 , wherein the ASO-antibody conjugate comprises a linker connecting the anti-transferrin receptor antibody or antigen-binding fragment thereof to the ASO.
8. The ASO-antibody conjugate of claim 2 , wherein the at least one 2′ modified nucleotide comprises;
2′-O-methyl, 2′-O-methoxyethyl (2′-O-MOE), 2′-O-aminopropyl, 2′-deoxy, 2′-deoxy-2′-fluoro, 2′-O-aminopropyl (2′-O-AP), 2′-O-dimethylaminoethyl (2′-O-DMAOE), 2′-O-dimethylaminopropyl (2′-O-DMAP), 2′-O-dimethylaminoethyloxyethyl (2′-O-DMAEOE), or 2′-O—N-methylacetamido (2′-O-NMA) modified nucleotide;
comprises locked nucleic acid (LNA) or ethylene nucleic acid (ENA); or
comprises a combination thereof.
9. The ASO-antibody conjugate of claim 2 , wherein the at least one modified internucleotide linkage comprises a phosphorothioate linkage or a phosphorodithioate linkage.
10. The ASO-antibody conjugate of claim 2 , wherein the ASO comprises 3 or more 2′ modified nucleotides selected from 2′-O-methyl and 2′-deoxy-2′-fluoro.
11. The ASO-antibody conjugate of claim 1 , wherein the ASO-antibody conjugate has an ASO to antibody ratio of from 1 to 4.
12. The ASO-antibody conjugate of claim 1 , wherein the ASO comprises a 5′-terminal vinylphosphonate modified nucleotide.
13. The ASO-antibody conjugate of claim 3 , wherein the muscle atrophy is associated with myotonic dystrophy.
14. The ASO-antibody conjugate of claim 1 , wherein the ASO-antibody conjugate is formulated for parenteral administration.
15. The ASO-antibody conjugate of claim 1 , wherein the ASO hybridizes to at least 8 contiguous bases of the target sequence of the human Murf1 mRNA.
Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Dec 7, 2022
From: GEALL, ANDREW JOHN; DOPPALAPUDI, VENKATA RAMANA; CHU, DAVID SAI-HO; COCHRAN, MICHAEL CARAMIAN; HOOD, MICHAEL DAVID; DARIMONT, BEATRICE DIANA; BURKE, ROB; SHI, YUNYU; MARELIUS, GULIN ERDOGAN; MALECOVA, BARBORA
To: AVIDITY BIOSCIENCES LLC
Reel/Frame 062012/0388 →
CHANGE OF NAME
Recorded Dec 7, 2022
From: AVIDITY BIOSCIENCES LLC
To: AVIDITY BIOSCIENCES, INC.
Reel/Frame 062089/0824 →
Continuity (7)
Continuation
PCTUS2018064359
· Dec 6, 2018
Provisional Application
62725883
· Aug 31, 2018
Provisional Application
62595545
· Dec 6, 2017
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