SG11202002737PA - Adeno-associated virus compositions for restoring hbb gene function and methods of use thereof - Google Patents
Adeno-associated virus compositions for restoring hbb gene function and methods of use thereofInfo
- Publication number
- SG11202002737PA SG11202002737PA SG11202002737PA SG11202002737PA SG11202002737PA SG 11202002737P A SG11202002737P A SG 11202002737PA SG 11202002737P A SG11202002737P A SG 11202002737PA SG 11202002737P A SG11202002737P A SG 11202002737PA SG 11202002737P A SG11202002737P A SG 11202002737PA
- Authority
- SG
- Singapore
- Prior art keywords
- adeno
- restoring
- methods
- associated virus
- gene function
- Prior art date
Links
- 241000702421 Dependoparvovirus Species 0.000 title 1
- 101150013707 HBB gene Proteins 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
-
- 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
- A61K48/0016—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 wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
-
- 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
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
- C07K14/01—DNA viruses
- C07K14/075—Adenoviridae
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/795—Porphyrin- or corrin-ring-containing peptides
- C07K14/805—Haemoglobins; Myoglobins
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
-
- 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
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2207/00—Modified animals
- A01K2207/12—Animals modified by administration of exogenous cells
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/035—Animal model for multifactorial diseases
- A01K2267/0381—Animal model for diseases of the hematopoietic system
-
- 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)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Mycology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Cell Biology (AREA)
- Immunology (AREA)
- Developmental Biology & Embryology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762574163P | 2017-10-18 | 2017-10-18 | |
US201862621102P | 2018-01-24 | 2018-01-24 | |
PCT/US2018/056271 WO2019079437A1 (en) | 2017-10-18 | 2018-10-17 | Adeno-associated virus compositions for restoring hbb gene function and methods of use thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11202002737PA true SG11202002737PA (en) | 2020-05-28 |
Family
ID=64332161
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11202002737PA SG11202002737PA (en) | 2017-10-18 | 2018-10-17 | Adeno-associated virus compositions for restoring hbb gene function and methods of use thereof |
Country Status (12)
Country | Link |
---|---|
US (1) | US20190134118A1 (en) |
EP (1) | EP3697450A1 (en) |
JP (1) | JP2021500070A (en) |
KR (1) | KR20200095462A (en) |
CN (1) | CN111447954A (en) |
AU (1) | AU2018352855B2 (en) |
BR (1) | BR112020007765A2 (en) |
CA (1) | CA3079437A1 (en) |
IL (1) | IL273877A (en) |
MX (1) | MX2020004092A (en) |
SG (1) | SG11202002737PA (en) |
WO (1) | WO2019079437A1 (en) |
Families Citing this family (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN109536494A (en) * | 2017-10-31 | 2019-03-29 | 广东赤萌医疗科技有限公司 | A kind of gRNA for repairing HBB1 point mutation, gene editing system, expression vector and gene editing kit |
WO2021138559A1 (en) * | 2019-12-31 | 2021-07-08 | Swanbio Therapeutics Limited | Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn) |
CN111363756B (en) * | 2020-04-02 | 2023-01-17 | 中国医学科学院血液病医院(中国医学科学院血液学研究所) | Globin gene dual-expression lentiviral vector and application thereof |
CN115484975A (en) * | 2020-04-06 | 2022-12-16 | 同源药物公司 | Adeno-associated virus compositions for IDS gene transfer and methods of use thereof |
EP4323513A2 (en) * | 2021-04-12 | 2024-02-21 | Graphite Bio, Inc. | Methods and compositions for production of genetically modified primary cells |
WO2023019185A1 (en) * | 2021-08-10 | 2023-02-16 | Gentibio, Inc. | Compositions and methods for engineering stable tregs |
Family Cites Families (11)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5449759A (en) * | 1987-05-16 | 1995-09-12 | Somatogen, Inc. | Hemoglobins with intersubunit desulfide bonds |
US5252479A (en) * | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
EP0938578B1 (en) * | 1996-12-05 | 2004-02-04 | Crucell Holland B.V. | Genetic modification of primate hemopoietic repopulating stem cells |
US9725485B2 (en) * | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
CN101348786B (en) * | 2007-07-20 | 2011-05-11 | 谭孟群 | Human beta-globin gene and recombinant adenovirus related virus vector thereof |
WO2009013559A1 (en) * | 2007-07-23 | 2009-01-29 | Cellectis | Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof |
US8628966B2 (en) | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
US11242525B2 (en) * | 2014-03-26 | 2022-02-08 | Editas Medicine, Inc. | CRISPR/CAS-related methods and compositions for treating sickle cell disease |
EP3198018B1 (en) * | 2014-09-24 | 2020-12-09 | City of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
US20180135074A1 (en) * | 2015-02-19 | 2018-05-17 | University Of Florida Research Foundation, Incorporated | Recombinant aav vectors for gene therapy of human hematopoietic disorders |
US11136597B2 (en) * | 2016-02-16 | 2021-10-05 | Yale University | Compositions for enhancing targeted gene editing and methods of use thereof |
-
2018
- 2018-10-17 KR KR1020207012240A patent/KR20200095462A/en not_active Application Discontinuation
- 2018-10-17 SG SG11202002737PA patent/SG11202002737PA/en unknown
- 2018-10-17 WO PCT/US2018/056271 patent/WO2019079437A1/en unknown
- 2018-10-17 BR BR112020007765-8A patent/BR112020007765A2/en unknown
- 2018-10-17 EP EP18804159.4A patent/EP3697450A1/en not_active Withdrawn
- 2018-10-17 CN CN201880079840.8A patent/CN111447954A/en active Pending
- 2018-10-17 MX MX2020004092A patent/MX2020004092A/en unknown
- 2018-10-17 US US16/163,061 patent/US20190134118A1/en active Pending
- 2018-10-17 AU AU2018352855A patent/AU2018352855B2/en active Active
- 2018-10-17 CA CA3079437A patent/CA3079437A1/en active Pending
- 2018-10-17 JP JP2020542536A patent/JP2021500070A/en active Pending
-
2020
- 2020-04-07 IL IL273877A patent/IL273877A/en unknown
Also Published As
Publication number | Publication date |
---|---|
CA3079437A1 (en) | 2019-04-25 |
BR112020007765A2 (en) | 2020-10-20 |
AU2018352855A1 (en) | 2020-04-02 |
US20190134118A1 (en) | 2019-05-09 |
WO2019079437A1 (en) | 2019-04-25 |
MX2020004092A (en) | 2021-01-08 |
EP3697450A1 (en) | 2020-08-26 |
IL273877A (en) | 2020-05-31 |
KR20200095462A (en) | 2020-08-10 |
CN111447954A (en) | 2020-07-24 |
AU2018352855B2 (en) | 2022-07-14 |
JP2021500070A (en) | 2021-01-07 |
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