WO2024097521A3 - Compositions for treatment of osteogenesis imperfecta - Google Patents
Compositions for treatment of osteogenesis imperfecta Download PDFInfo
- Publication number
- WO2024097521A3 WO2024097521A3 PCT/US2023/076695 US2023076695W WO2024097521A3 WO 2024097521 A3 WO2024097521 A3 WO 2024097521A3 US 2023076695 W US2023076695 W US 2023076695W WO 2024097521 A3 WO2024097521 A3 WO 2024097521A3
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- col1a1
- col1a2
- expression
- compositions
- vectors
- Prior art date
Links
- 206010031243 Osteogenesis imperfecta Diseases 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title abstract 2
- 102100033601 Collagen alpha-1(I) chain Human genes 0.000 abstract 4
- 102100036213 Collagen alpha-2(I) chain Human genes 0.000 abstract 4
- 101000875067 Homo sapiens Collagen alpha-2(I) chain Proteins 0.000 abstract 4
- 108010029483 alpha 1 Chain Collagen Type I Proteins 0.000 abstract 4
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 102000039446 nucleic acids Human genes 0.000 abstract 2
- 239000013598 vector Substances 0.000 abstract 2
- 108091026821 Artificial microRNA Proteins 0.000 abstract 1
- 108700019146 Transgenes Proteins 0.000 abstract 1
- 210000002449 bone cell Anatomy 0.000 abstract 1
- 230000003247 decreasing effect Effects 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 210000000963 osteoblast Anatomy 0.000 abstract 1
- 210000002997 osteoclast Anatomy 0.000 abstract 1
- 210000004409 osteocyte Anatomy 0.000 abstract 1
- 239000013608 rAAV vector Substances 0.000 abstract 1
- -1 rAAV vectors Chemical class 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
-
- 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
- C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmacology & Pharmacy (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
In some aspects, the disclosure relates to compositions and methods for modulating (e.g., increasing and/or decreasing) expression of COL1A1 or COL1A2 in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that decrease the expression of mutant COL1A1 or COL1A2 (e.g., amiRNAs) and/or provide a wildtype copy of COL1A1 or COL1A2 in certain types of bone cells, for example osteoblasts, osteoclasts, osteocytes, etc. In some embodiments, the isolated nucleic acids and vectors described by the disclosure are useful for treating disorders and conditions associated with mutant COL1A1 or COL1A2 expression, such as osteogenesis imperfecta (OI).
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263422062P | 2022-11-03 | 2022-11-03 | |
| US63/422,062 | 2022-11-03 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| WO2024097521A2 WO2024097521A2 (en) | 2024-05-10 |
| WO2024097521A3 true WO2024097521A3 (en) | 2024-06-13 |
Family
ID=90931505
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PCT/US2023/076695 WO2024097521A2 (en) | 2022-11-03 | 2023-10-12 | Compositions for treatment of osteogenesis imperfecta |
Country Status (1)
| Country | Link |
|---|---|
| WO (1) | WO2024097521A2 (en) |
-
2023
- 2023-10-12 WO PCT/US2023/076695 patent/WO2024097521A2/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2024097521A2 (en) | 2024-05-10 |
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