IP Library › Granted Patent US 12668617
Granted Patent B2
US 12668617 · App. 17/254,055 · Granted Jun 30, 2026

Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies

Inventor: Paul Taylor Martin (Bexley, OH)
Assignee: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
C07K14/485A61K48/0058A61P21/00C07K14/4707C07K14/78C12N15/86C07K2319/00C12N2750/14143C12N2830/008
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Quick Facts
Patent No.
US 12668617
App. No.
17/254,055
Granted
Jun 30, 2026
Kind
B2
Abstract

Products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies are provided. In the methods, a protein including a linker domain, such as the heparin-binding domain of Heparin-Binding Epidermal Growth Factor-Like Growth Factor (HBEGF), is delivered to patients.

Claims (28)

1 . A polynucleotide comprising a first nucleotide sequence and a second nucleotide sequence, wherein the first nucleotide sequence comprises the nucleotide sequence of SEQ ID NO: 13 or SEQ ID NO: 14, and wherein the second nucleotide sequence comprises the nucleotide sequence of SEQ ID NO: 15 or SEQ ID NO: 16.

2 . The polynucleotide of claim 1 , wherein

the first nucleotide sequence comprises the nucleotide sequence of SEQ ID NO: 13 and the second nucleotide sequence comprises the nucleotide sequence of SEQ ID NO: 15 or the nucleotide sequence of SEQ ID NO: 16.

3 . The polynucleotide of claim 1 , wherein

the first nucleotide sequence comprises the nucleotide sequence of SEQ ID NO: 14 and the second nucleotide sequence comprises the nucleotide sequence of SEQ ID NO: 15 or the nucleotide sequence of SEQ ID NO: 16.

4 . The polynucleotide of claim 1 , wherein the polynucleotide comprises

(a) the nucleotide sequence of SEQ ID NO: 1,

(b) a nucleotide sequence encoding the amino acid sequence of SEQ ID NO: 19,

(c) the nucleotide sequence of SEQ ID NO: 3,

(d) a nucleotide sequence encoding the amino acid sequence of SEQ ID NO: 20,

(e) the nucleotide sequence of SEQ ID NO: 5,

(f) a nucleotide sequence encoding the amino acid sequence of SEQ ID NO: 21,

(g) the nucleotide sequence of SEQ ID NO: 7, or

(h) a nucleotide sequence encoding the amino acid sequence of SEQ ID NO: 22.

5 . A recombinant adeno-associated virus (rAAV), wherein the genome of the rAAV comprises

(a) the polynucleotide sequence of SEQ ID NO: 1,

(b) the polynucleotide sequence of SEQ ID NO: 3,

(c) the polynucleotide sequence of SEQ ID NO: 5,

(d) the polynucleotide sequence of SEQ ID NO: 7,

(e) nucleotides 3590 to 8215 of SEQ ID NO: 2,

(f) nucleotides 3590 to 8341 of SEQ ID NO: 4,

(g) nucleotides 3609 to 6929 of SEQ ID NO: 6,

(h) nucleotides 3590 to 7036 of SEQ ID NO: 8, or

(i) the nucleotide sequence set out in SEQ ID NO: 25.

6 . The rAAV of claim 5 , wherein the genome of the rAAV further comprises a muscle-specific transcriptional control element.

7 . A recombinant host cell comprising the polynucleotide of claim 1 .

8 . A protein encoded by the polynucleotide of claim 1 , wherein the protein comprises an amino acid sequence of SEQ ID NO: 19, SEQ ID NO: 20, SEQ ID NO: 21 or SEQ ID NO: 22.

9 . A composition comprising the rAAV of claim 5 .