BR112021020183A2 - Terapias gênicas para distúrbios lisossomais - Google Patents
Terapias gênicas para distúrbios lisossomaisInfo
- Publication number
- BR112021020183A2 BR112021020183A2 BR112021020183A BR112021020183A BR112021020183A2 BR 112021020183 A2 BR112021020183 A2 BR 112021020183A2 BR 112021020183 A BR112021020183 A BR 112021020183A BR 112021020183 A BR112021020183 A BR 112021020183A BR 112021020183 A2 BR112021020183 A2 BR 112021020183A2
- Authority
- BR
- Brazil
- Prior art keywords
- gene therapies
- lysosomal
- lysosomal disorders
- ftd
- expression constructs
- Prior art date
Links
Classifications
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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
- 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
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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
- C07K14/475—Growth factors; Growth regulators
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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
- 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
- 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
- A61K48/0083—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 administration regime
-
- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/18—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with another compound as one donor, and incorporation of one atom of oxygen (1.14.18)
- C12Y114/18001—Tyrosinase (1.14.18.1)
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6803—General methods of protein analysis not limited to specific proteins or families of proteins
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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
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
-
- 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
- C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
-
- 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
-
- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
- G01N2333/435—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Urology & Nephrology (AREA)
- Hematology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Psychiatry (AREA)
- Food Science & Technology (AREA)
- Cell Biology (AREA)
- General Physics & Mathematics (AREA)
- Analytical Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Hospice & Palliative Care (AREA)
Abstract
terapias gênicas para distúrbios lisossomais. a presente invenção refere-se a composições e métodos para o tratamento de distúrbios associados à função lisossomal aberrante, como a demência frontotemporal (ftd - frontotemporal dementia). a divulgação também fornece construtos de expressão que compreendem um transgene que codifica a progranulina ou uma porção desta. a divulgação fornece métodos de tratamento da ftd pela administração desses construtos de expressão a um indivíduo em necessidade.
Applications Claiming Priority (6)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962831846P | 2019-04-10 | 2019-04-10 | |
US201962934450P | 2019-11-12 | 2019-11-12 | |
US201962954089P | 2019-12-27 | 2019-12-27 | |
US202062960471P | 2020-01-13 | 2020-01-13 | |
US202062988665P | 2020-03-12 | 2020-03-12 | |
PCT/US2020/027764 WO2020210698A1 (en) | 2019-04-10 | 2020-04-10 | Gene therapies for lysosomal disorders |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112021020183A2 true BR112021020183A2 (pt) | 2022-03-03 |
Family
ID=70554180
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112021020183A BR112021020183A2 (pt) | 2019-04-10 | 2020-04-10 | Terapias gênicas para distúrbios lisossomais |
Country Status (11)
Country | Link |
---|---|
US (1) | US11999974B2 (pt) |
EP (1) | EP3953377A1 (pt) |
JP (1) | JP2022526823A (pt) |
KR (1) | KR20210150486A (pt) |
CN (1) | CN114174324A (pt) |
AU (1) | AU2020273182A1 (pt) |
BR (1) | BR112021020183A2 (pt) |
CA (1) | CA3136004A1 (pt) |
IL (1) | IL286903A (pt) |
MX (1) | MX2021011958A (pt) |
WO (1) | WO2020210698A1 (pt) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
SG11201906947SA (en) | 2017-02-17 | 2019-08-27 | Bristol Myers Squibb Co | Antibodies to alpha-synuclein and uses thereof |
AU2018346105C1 (en) | 2017-10-03 | 2023-10-12 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
EP4192517A1 (en) | 2020-08-10 | 2023-06-14 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
WO2024100633A1 (en) * | 2022-11-13 | 2024-05-16 | Alexion Pharma International Operations Limited | Gene therapy for frontotemporal dementia |
Family Cites Families (30)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU2001255575B2 (en) | 2000-04-28 | 2006-08-31 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
ATE310096T1 (de) | 2000-09-18 | 2005-12-15 | Genzyme Corp | Expressionsvektoren mit hybriden ubiquitin- promotoren |
US20030133924A1 (en) | 2001-12-21 | 2003-07-17 | Novazyme Pharmaceuticals, Inc. | Highly phosphorylated acid beta-glucocerebrosidase and methods of treating gaucher's disease |
AU2003221733A1 (en) | 2002-04-17 | 2003-11-03 | University Of Florida Research Foundation, Inc. | Improved raav vectors |
WO2006036465A2 (en) | 2004-09-03 | 2006-04-06 | University Of Florida | Compositions and methods for treating cystic fibrosis |
EP2037948B1 (en) | 2006-05-30 | 2016-04-13 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
EP2687223B1 (en) | 2006-05-30 | 2017-07-12 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
EP2489733B1 (en) | 2006-06-07 | 2019-02-13 | Genzyme Corporation | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
EP2061891B1 (en) | 2006-08-24 | 2012-04-11 | Virovek, Inc. | Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor |
US20090176729A1 (en) | 2007-12-14 | 2009-07-09 | Alnylam Pharmaceuticals, Inc. | Method of treating neurodegenerative disease |
WO2009089635A1 (en) | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
WO2009120978A2 (en) | 2008-03-27 | 2009-10-01 | The Ohio State University | Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor |
LT2424991T (lt) | 2009-05-02 | 2018-09-25 | Genzyme Corporation | Neurodegeneracinių sutrikimų genų terapija |
EP2640407A4 (en) | 2010-11-16 | 2014-07-09 | Denis G Kay | PROCESS FOR INCREASING THE EXPRESSION AND ACTIVITY OF NEPRILYSIN |
CN104902923A (zh) | 2012-11-05 | 2015-09-09 | 建新公司 | 用于治疗蛋白质病的组合物和方法 |
CN115120745A (zh) | 2013-05-15 | 2022-09-30 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
EP3193942B1 (en) | 2014-08-11 | 2020-03-25 | Shire Human Genetic Therapies, Inc. | Lysosomal targeting and uses thereof |
WO2016081927A2 (en) | 2014-11-21 | 2016-05-26 | University Of Florida Research Foundation, Inc. | Genome-modified recombinant adeno-associated virus vectors |
EP3292206B8 (en) | 2015-05-07 | 2022-02-09 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
PT3411484T (pt) | 2016-02-05 | 2023-11-17 | Univ Emory | Injeção de vírus 9 adeno-associado de cadeia simples ou auto-complementar no líquido cefalorraquidiano |
EP3423109A4 (en) | 2016-03-02 | 2019-08-14 | The Children's Hospital of Philadelphia | THERAPY FOR FRONTOTEMPORAL DEMENTIA |
IL261665B2 (en) | 2016-04-21 | 2023-11-01 | Virovek Inc | AVV production in insect cells, methods and preparations thereof |
AU2018346105C1 (en) | 2017-10-03 | 2023-10-12 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
CN111542549A (zh) | 2017-10-03 | 2020-08-14 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
CA3078501A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
AU2018354195A1 (en) | 2017-10-23 | 2020-06-04 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
SG11202004406VA (en) * | 2017-11-08 | 2020-06-29 | Avexis Inc | Means and method for preparing viral vectors and uses of same |
CA3134841A1 (en) | 2019-04-10 | 2020-10-15 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
EP3953378A1 (en) | 2019-04-10 | 2022-02-16 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
EP4192517A1 (en) | 2020-08-10 | 2023-06-14 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
-
2020
- 2020-04-10 AU AU2020273182A patent/AU2020273182A1/en active Pending
- 2020-04-10 KR KR1020217036234A patent/KR20210150486A/ko unknown
- 2020-04-10 BR BR112021020183A patent/BR112021020183A2/pt unknown
- 2020-04-10 WO PCT/US2020/027764 patent/WO2020210698A1/en active Application Filing
- 2020-04-10 EP EP20724262.9A patent/EP3953377A1/en active Pending
- 2020-04-10 CA CA3136004A patent/CA3136004A1/en active Pending
- 2020-04-10 MX MX2021011958A patent/MX2021011958A/es unknown
- 2020-04-10 CN CN202080041413.8A patent/CN114174324A/zh active Pending
- 2020-04-10 JP JP2021559727A patent/JP2022526823A/ja active Pending
- 2020-04-10 US US16/846,065 patent/US11999974B2/en active Active
-
2021
- 2021-10-03 IL IL286903A patent/IL286903A/en unknown
Also Published As
Publication number | Publication date |
---|---|
US20200332265A1 (en) | 2020-10-22 |
CN114174324A (zh) | 2022-03-11 |
CA3136004A1 (en) | 2020-10-15 |
EP3953377A1 (en) | 2022-02-16 |
WO2020210698A1 (en) | 2020-10-15 |
IL286903A (en) | 2021-10-31 |
JP2022526823A (ja) | 2022-05-26 |
AU2020273182A1 (en) | 2021-12-02 |
MX2021011958A (es) | 2021-12-15 |
KR20210150486A (ko) | 2021-12-10 |
US11999974B2 (en) | 2024-06-04 |
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