AR128239A1 - COMPOSITIONS AND USEFUL METHODS FOR THE TREATMENT OF DISORDERS MEDIATED BY C9ORF72 - Google Patents
COMPOSITIONS AND USEFUL METHODS FOR THE TREATMENT OF DISORDERS MEDIATED BY C9ORF72Info
- Publication number
- AR128239A1 AR128239A1 ARP230100057A ARP230100057A AR128239A1 AR 128239 A1 AR128239 A1 AR 128239A1 AR P230100057 A ARP230100057 A AR P230100057A AR P230100057 A ARP230100057 A AR P230100057A AR 128239 A1 AR128239 A1 AR 128239A1
- Authority
- AR
- Argentina
- Prior art keywords
- c9orf72
- compositions
- treatment
- disorders mediated
- useful methods
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 2
- 230000001404 mediated effect Effects 0.000 title 1
- 108091026890 Coding region Proteins 0.000 abstract 3
- 102000043334 C9orf72 Human genes 0.000 abstract 2
- 108700030955 C9orf72 Proteins 0.000 abstract 2
- 101150014718 C9orf72 gene Proteins 0.000 abstract 2
- 101000989501 Homo sapiens Guanine nucleotide exchange factor C9orf72 Proteins 0.000 abstract 2
- 102000045815 human C9orf72 Human genes 0.000 abstract 2
- 108091070501 miRNA Proteins 0.000 abstract 2
- 239000002679 microRNA Substances 0.000 abstract 2
- 201000011240 Frontotemporal dementia Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
Classifications
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- 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
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- 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
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- 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
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- 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
- 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
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0636—T lymphocytes
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- 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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
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- 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
- C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
- C12N2506/45—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from artificially induced pluripotent stem cells
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- 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
- C12N2510/00—Genetically modified cells
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- 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)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Physics & Mathematics (AREA)
- Neurology (AREA)
- Public Health (AREA)
- Hematology (AREA)
- Cell Biology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
En el presente documento, se proporcionan rAAV y otros vectores y composiciones útiles para tratar un paciente que tiene C9orf72 que comprende una secuencia codificante de hC9orf72 diseñado y la al menos una secuencia codificante de miRNA, donde la secuencia codificante de C9orf72 humano diseñado tiene una secuencia que difiere del C9orf72 humano endógeno en el paciente en el sitio objetivo del miRNA codificado. También se proporcionan métodos para tratar ALS, FTD relacionadas con C9orf72 y trastornos relacionados.Provided herein are rAAV and other vectors and compositions useful for treating a patient having C9orf72 comprising an engineered hC9orf72 coding sequence and the at least one miRNA coding sequence, wherein the engineered human C9orf72 coding sequence has a sequence which differs from endogenous human C9orf72 in the patient at the target site of the encoded miRNA. Methods for treating ALS, C9orf72-related FTDs, and related disorders are also provided.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202263298046P | 2022-01-10 | 2022-01-10 |
Publications (1)
Publication Number | Publication Date |
---|---|
AR128239A1 true AR128239A1 (en) | 2024-04-10 |
Family
ID=85251731
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
ARP230100057A AR128239A1 (en) | 2022-01-10 | 2023-01-10 | COMPOSITIONS AND USEFUL METHODS FOR THE TREATMENT OF DISORDERS MEDIATED BY C9ORF72 |
Country Status (3)
Country | Link |
---|---|
AR (1) | AR128239A1 (en) |
TW (1) | TW202340467A (en) |
WO (1) | WO2023133574A1 (en) |
Family Cites Families (40)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
ES2224375T3 (en) | 1997-04-14 | 2005-03-01 | Cell Genesys, Inc. | METHODS TO INCREASE THE EFFECTIVENESS OF THE RECOMBINANT AAV PRODUCT. |
EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
JP2002538770A (en) | 1998-11-10 | 2002-11-19 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | Viral vectors and methods for their production and administration |
AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
DK1310571T3 (en) | 2001-11-13 | 2006-06-19 | Univ Pennsylvania | Method for identifying unknown adeno-associated virus sequences (AAV sequences) and a kit for the method |
EP1453547B1 (en) | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
ES2411479T3 (en) | 2003-09-30 | 2013-07-05 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus clades (AAV), sequences, vectors that contain them, and uses thereof |
EP3085389A1 (en) | 2005-04-07 | 2016-10-26 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
US8969353B2 (en) | 2008-11-07 | 2015-03-03 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
JP5922095B2 (en) | 2010-03-29 | 2016-05-24 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Pharmacologically induced transgene ablation system |
US20130037977A1 (en) | 2010-04-08 | 2013-02-14 | Paul A. Burke | Preparation of Lipid Nanoparticles |
US8853377B2 (en) | 2010-11-30 | 2014-10-07 | Shire Human Genetic Therapies, Inc. | mRNA for use in treatment of human genetic diseases |
IL280771B2 (en) | 2011-06-08 | 2024-03-01 | Shire Human Genetic Therapies | Lipid nanoparticle compositions and methods for mrna delivery |
FR2977562B1 (en) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | SEALED AND THERMALLY INSULATING TANK INTEGRATED IN A CARRIER STRUCTURE |
EP3800254A1 (en) | 2012-06-08 | 2021-04-07 | Ethris GmbH | Pulmonary delivery of messenger rna |
EP2929035A1 (en) | 2012-12-07 | 2015-10-14 | Shire Human Genetic Therapies, Inc. | Lipidic nanoparticles for mrna delivering |
CN105873902B (en) | 2013-11-18 | 2019-03-08 | 阿克丘勒斯治疗公司 | Ionizable cation lipid for RNA delivery |
WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
WO2017160360A2 (en) | 2015-12-11 | 2017-09-21 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
SG10201912976WA (en) | 2017-02-28 | 2020-02-27 | Univ Pennsylvania | Adeno-associated virus (aav) clade f vector and uses therefor |
WO2018168961A1 (en) | 2017-03-16 | 2018-09-20 | 株式会社デンソー | Own-position estimating device |
CN111819281A (en) * | 2017-10-23 | 2020-10-23 | 普利维尔治疗公司 | Gene therapy for neurodegenerative diseases |
WO2019168961A1 (en) | 2018-02-27 | 2019-09-06 | The Trustees Of The University Of Pennsylvania | Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor |
BR112020017278A2 (en) | 2018-02-27 | 2020-12-22 | The Trustees Of The University Of Pennsylvania | ADENOSOCIATED VIRUS VECTORS (AAV), AAV VECTORS HAVING REDUCED CAPSIDE DAMMATION AND USES FOR THE SAME |
BR112021011143A2 (en) | 2018-12-21 | 2022-01-25 | Univ Pennsylvania | Compositions for drg-specific reduction of transgene expression |
KR20230019063A (en) * | 2019-10-22 | 2023-02-07 | 어플라이드 제네틱스 테크놀로지스 코퍼레이션 | Triple function adeno-associated virus (AAV) vectors for the treatment of C9ORF72 associated diseases |
US20230304034A1 (en) | 2020-05-12 | 2023-09-28 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
TW202229315A (en) | 2020-10-18 | 2022-08-01 | 賓州大學委員會 | Improved adeno-associated virus (aav) vector and uses therefor |
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2023
- 2023-01-10 AR ARP230100057A patent/AR128239A1/en unknown
- 2023-01-10 WO PCT/US2023/060354 patent/WO2023133574A1/en unknown
- 2023-01-10 TW TW112101016A patent/TW202340467A/en unknown
Also Published As
Publication number | Publication date |
---|---|
TW202340467A (en) | 2023-10-16 |
WO2023133574A1 (en) | 2023-07-13 |
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