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NZ747137B2 - Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy - Google Patents
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NZ747137B2 - Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy - Google Patents

Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy

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Publication number
NZ747137B2
NZ747137B2 NZ747137A NZ74713717A NZ747137B2 NZ 747137 B2 NZ747137 B2 NZ 747137B2 NZ 747137 A NZ747137 A NZ 747137A NZ 74713717 A NZ74713717 A NZ 74713717A NZ 747137 B2 NZ747137 B2 NZ 747137B2
Authority
NZ
New Zealand
Prior art keywords
cell
recombinant
seq
nucleotide sequence
aav vector
Prior art date
Application number
NZ747137A
Other versions
NZ747137A (en
Inventor
Jerry R Mendell
Klapac Louise Rodino
Original Assignee
Research Institute At Nationwide Children's Hospital
Filing date
Publication date
Application filed by Research Institute At Nationwide Children's Hospital filed Critical Research Institute At Nationwide Children's Hospital
Priority claimed from PCT/US2017/027583 external-priority patent/WO2017180976A1/en
Publication of NZ747137A publication Critical patent/NZ747137A/en
Publication of NZ747137B2 publication Critical patent/NZ747137B2/en

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0656—Adult fibroblasts
    • C—CHEMISTRY; METALLURGY
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0681—Cells of the genital tract; Non-germinal cells from gonads
    • C12N5/0682—Cells of the female genital tract, e.g. endometrium; Non-germinal cells from ovaries, e.g. ovarian follicle cells
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0684—Cells of the urinary tract or kidneys
    • C12N5/0686—Kidney cells
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0688—Cells from the lungs or the respiratory tract
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0693—Tumour cells; Cancer cells

Abstract

Described herein are recombinant AAV vectors comprising a polynucleotide sequence comprising ß-sarcoglycan and methods of using the recombinant vectors to reduce or prevent fibrosis in a mammalian subject suffering from a muscular dystrophy. Also described herein are combination therapies comprising administering AAV vector(s) expressing ß-sarcoglycan and miR-29c to a mammalian subject suffering from a muscular dystrophy.

Claims (41)

What is claimed is:
1. A recombinant AAV vector comprising a cleotide sequence encoding ß-sarcoglycan, wherein the polynucleotide sequence comprises the nucleotide sequence set forth in SEQ ID NO: 1.
2. The recombinant AAV vector of claim 1, n the cleotide sequence is operably linked to a muscle-specific control element.
3. The recombinant AAV vector of claim 1 or 2, wherein the vector is of the serotype AAV1, AAV2, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAV10, AAV11, AAV12, AAV13 or AAV rh.74, or a variant thereof.
4. The recombinant AAV vector of claim 2 or 3, n the musclespecific control element is human skeletal actin gene element, c actin gene element, myocyte-specific enhancer g factor mef DNA g site, muscle creatine kinase promoter (MCK), truncated MCK promoter (tMCK), myosin heavy chain promoter (MHC), MHCK7 promoter, C5-12 promoter, murine creatine kinase enhancer element, skeletal fast-twitch troponin c gene element, slow-twitch cardiac troponin c gene element, slow-twitch troponin I gene element, hypoxia-inducible nuclear factors, steroid-inducible element or orticoid response element (gre).
5. The recombinant AAV vector of claim 4, wherein the muscle-specific control t is truncated MCK er (tMCK).
6. The recombinant AAV vector of claim 4, wherein the muscle-specific control element is MHCK7 promoter.
7. The recombinant AAV vector of any one of claims 1-6 comprising the nucleotide sequence set forth in SEQ ID NO: 3.
8. The recombinant AAV vector of any one of claims 1-6 comprising the nucleotide sequence set forth in SEQ ID NO: 5.
9. A pharmaceutical composition comprising the rAAV vector of any one of claims 1-8.
10. A composition comprising the recombinant AAV vector of any one of claims 1-8 or the pharmaceutical composition according to claim 9.
11. Use of the inant AAV vector of any one of claims 1-8 or the pharmaceutical composition of claim 9, or the composition of claim 10 in the preparation of a medicament for reducing fibrosis or contraction induced injury in a subject in need thereof.
12. Use of the recombinant AAV vector of any one of claims 1-11, the pharmaceutical composition of claim 12, or the composition of claim 13 in the preparation of a medicament for the treatment of ß-sarcoglycanopathy in a subject in need thereof.
13. The use of claim 11 or claim 12, wherein the subject is suffering from muscular dystrophy.
14. Use of the inant AAV vector of any one of claims 1-8, or the pharmaceutical ition of claim 9, or the composition of claim 10 in the preparation of a ment for increasing muscular force and/or muscle mass in a subject suffering from muscular dystrophy.
15. The use of claim 13 or 14, wherein the muscular dystrophy is limbgirdle muscular dystrophy (LGMD), and optionally wherein the LGMD is LGMD2E.
16. The use of any one of claims 11-15, wherein the subject is a human being.
17. The use of any one of claims 11-16, wherein the medicament further comprises a second recombinant AAV vector comprising the miR-29 nucleotide sequence.
18. The use of claim 17, wherein the second recombinant vector comprises the nucleotide sequence set forth in SEQ ID NO: 9 or the tide sequence set forth in SEQ ID NO: 8.
19. The use of any one of claims 11-18, n the rAAV is formulated for intramuscular injection or intravenous injection.
20. The use of any one of claims 11-19, n the rAAV is formulated for systemic administration.
21. The use of claim 20, wherein the systemic administration is parenteral administration by injection, infusion, or implantation.
22. A inant polynucleotide sing a nucleotide sequence of SEQ ID NO: 1.
23. The recombinant polynucleotide of claim 22, comprising in the 5’ to 3’ direction an inverted terminal repeat (ITR), a muscle-specific control element, a chimeric intron sequence, the nucleotide sequence of SEQ ID NO: 1, and an ITR.
24. The recombinant polynucleotide of claim 23, wherein the musclespecific control element is truncated MCK (tMCK) promoter that comprises the nucleotide sequence set forth in SEQ ID NO: 6.
25. The recombinant polynucleotide of any one of claims 22-24, wherein the polynucleotide sequence comprises the nucleotide sequence of SEQ ID NO: 5.
26. The recombinant cleotide sequence of claim 23, n the -specific control element is MHCK7 promoter that comprises the nucleotide sequence set forth in SEQ ID NO: 4.
27. The recombinant polynucleotide sequence of claim 26, wherein the recombinant polynucleotide comprises the nucleotide sequence set forth in SEQ ID NO: 3.
28. A plasmid comprising the polynucleotide of any one of claims 22-26.
29. The d of claim 28, wherein said plasmid lacks AAV rep and cap genes.
30. The plasmid of claim 28 or 29, further comprising a selectable marker.
31. A cell comprising a plasmid of any one of claims 28-30, wherein the cell is not a human cell inside the human body.
32. The cell of claim 31, wherein the cell is a eukaryotic cell, a ial cell, an insect cell, or a yeast cell.
33. A packaging cell sing the plasmid of any one of claims 28-30, wherein the packaging cell is not a host cell inside the human body.
34. The packaging cell of claim 33, wherein the packaging cell is a 293 cell, a Hela cell, MRC-5 cell, WI-38 cell, Vero cell or FrhL-2 cell.
35. An associated viral (AAV) ing system comprising: (a) the plasmid of any one of claims 28-30; (b) a rep-cap helper plasmid, and (c) an adenovirus helper plasmid.
36. An adeno-associated viral (AAV) packaging system comprising: (a) the plasmid of any one of claims 28-30; and (b) an adenovirus helper plasmid.
37. A method of producing a recombinant AAV (rAAV) particle comprising transfecting a cell with the plasmid of any one of claims 28-30, or the packaging system of claim 35 or 36.
38. The method of claim 37, further comprising the step of culturing the cell.
39. The method of claim 37 or 38, wherein the cell is a eukaryotic cell, a ial cell, an insect cell, or a yeast cell.
40. The method of claim 38 or 39, further comprising the step of recovering an rAAV particle from the supernatant of the cell.
41. A recombinant AAV particle produced by the method of claim 40. GOGPV!UJGGVREPLACEMENT SHEET 2038
NZ747137A 2017-04-14 Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy NZ747137B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662323333P 2016-04-15 2016-04-15
US201662433548P 2016-12-13 2016-12-13
PCT/US2017/027583 WO2017180976A1 (en) 2016-04-15 2017-04-14 Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy

Publications (2)

Publication Number Publication Date
NZ747137A NZ747137A (en) 2025-06-27
NZ747137B2 true NZ747137B2 (en) 2025-09-30

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