AU2022370552A1 - Compositions and methods for treating myelin deficiency by rejuvenating glial progenitor cells - Google Patents
Compositions and methods for treating myelin deficiency by rejuvenating glial progenitor cells Download PDFInfo
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- AU2022370552A1 AU2022370552A1 AU2022370552A AU2022370552A AU2022370552A1 AU 2022370552 A1 AU2022370552 A1 AU 2022370552A1 AU 2022370552 A AU2022370552 A AU 2022370552A AU 2022370552 A AU2022370552 A AU 2022370552A AU 2022370552 A1 AU2022370552 A1 AU 2022370552A1
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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
- C12N15/11—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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/45—Transferases (2)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
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- 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
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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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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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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- 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
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- 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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- 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
- C12N5/0618—Cells of the nervous system
- C12N5/0622—Glial cells, e.g. astrocytes, oligodendrocytes; Schwann cells
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
- C12N9/1007—Methyltransferases (general) (2.1.1.)
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
- C12N9/80—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
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- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/01—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in linear amides (3.5.1)
- C12Y305/01098—Histone deacetylase (3.5.1.98), i.e. sirtuin deacetylase
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
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- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/60—Transcription factors
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- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- Health & Medical Sciences (AREA)
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- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Cell Biology (AREA)
- Virology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Marine Sciences & Fisheries (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163257827P | 2021-10-20 | 2021-10-20 | |
| US63/257,827 | 2021-10-20 | ||
| US202263350041P | 2022-06-08 | 2022-06-08 | |
| US202263350039P | 2022-06-08 | 2022-06-08 | |
| US202263350042P | 2022-06-08 | 2022-06-08 | |
| US63/350,039 | 2022-06-08 | ||
| US63/350,041 | 2022-06-08 | ||
| US63/350,042 | 2022-06-08 | ||
| PCT/US2022/047083 WO2023069482A1 (en) | 2021-10-20 | 2022-10-19 | Compositions and methods for treating myelin deficiency by rejuvenating glial progenitor cells |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AU2022370552A1 true AU2022370552A1 (en) | 2024-03-28 |
Family
ID=84359179
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2022370552A Pending AU2022370552A1 (en) | 2021-10-20 | 2022-10-19 | Compositions and methods for treating myelin deficiency by rejuvenating glial progenitor cells |
| AU2022369984A Pending AU2022369984A1 (en) | 2021-10-20 | 2022-10-19 | Methods and compositions for rejuvenating cns glial populations by suppression of transcription factors |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2022369984A Pending AU2022369984A1 (en) | 2021-10-20 | 2022-10-19 | Methods and compositions for rejuvenating cns glial populations by suppression of transcription factors |
Country Status (6)
| Country | Link |
|---|---|
| US (4) | US12565648B2 (https=) |
| EP (2) | EP4419685A1 (https=) |
| JP (2) | JP2024539080A (https=) |
| AU (2) | AU2022370552A1 (https=) |
| CA (3) | CA3235458A1 (https=) |
| WO (3) | WO2023069478A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023069478A1 (en) * | 2021-10-20 | 2023-04-27 | University Of Rochester | Methods and compositions for rejuvenating cns glial populations by suppression of transcription factors |
| EP4604985A1 (en) * | 2022-10-19 | 2025-08-27 | University of Rochester | Methods and compositions for rejuvenating cns glial populations with bcl11a transcription factor expression |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US748529A (en) | 1903-09-03 | 1903-12-29 | Troy Laundry Machinery Co Ltd | Marking-machine. |
| US5872005A (en) | 1994-11-03 | 1999-02-16 | Cell Genesys Inc. | Packaging cell lines for adeno-associated viral vectors |
| GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| ES2341926T3 (es) | 1998-03-02 | 2010-06-29 | Massachusetts Institute Of Technology | Poliproteinas con dedos de cinc que tienen enlazadores mejorados. |
| US7013219B2 (en) | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
| US20030104526A1 (en) | 1999-03-24 | 2003-06-05 | Qiang Liu | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
| US7030215B2 (en) | 1999-03-24 | 2006-04-18 | Sangamo Biosciences, Inc. | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
| WO2002088346A2 (en) | 2001-05-01 | 2002-11-07 | National Research Council Of Canada | A system for inducible expression in eukaryotic cells |
| WO2003070171A2 (en) | 2002-02-15 | 2003-08-28 | Cornell Research Foundation, Inc. | Myelination of congenitally dysmyelinated forebrains using oligodendrocyte progenitor cells |
| US20040029269A1 (en) | 2002-05-07 | 2004-02-12 | Goldman Steven A | Promoter-based isolation, purification, expansion, and transplantation of neuronal progenitor cells, oligodendrocyte progenitor cells, or neural stem cells from a population of embryonic stem cells |
| US7485291B2 (en) | 2003-06-03 | 2009-02-03 | Cell Genesys, Inc. | Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof |
| US8728759B2 (en) | 2004-10-04 | 2014-05-20 | National Research Council Of Canada | Reverse cumate repressor mutant |
| WO2011072246A2 (en) | 2009-12-10 | 2011-06-16 | Regents Of The University Of Minnesota | Tal effector-mediated dna modification |
| EP2951588A4 (en) * | 2013-01-30 | 2016-11-16 | Benaroya Res Inst At Virginia Mason | EFFeCtor T-CELL RESISTANCE |
| HUE053191T2 (hu) | 2017-02-09 | 2021-06-28 | Bayer Ag | 2-Heteroaril-3-oxo-2,3-dihidropiridazin-4-karboxamidok rák kezelésére |
| US12365872B2 (en) * | 2018-09-19 | 2025-07-22 | Lineage Cell Therapeutics, Inc. | Methods for differentiating pluripotent stem cells in dynamic suspension culture |
| US20230057355A1 (en) | 2019-02-13 | 2023-02-23 | University Of Rochester | Gene networks that mediate remyelination of the human brain |
| CA3167135A1 (en) | 2020-02-06 | 2021-08-12 | Emerald Health Pharmaceuticals Inc. | Composition and method for the treatment and prevention of cardiac, pulmonary, dermal, and renal fibrosis |
| GB2625500B (en) | 2021-09-21 | 2026-04-01 | Scribe Therapeutics Inc | Engineered CasX repressor systems |
| WO2023069478A1 (en) | 2021-10-20 | 2023-04-27 | University Of Rochester | Methods and compositions for rejuvenating cns glial populations by suppression of transcription factors |
| CA3234809A1 (en) | 2021-10-20 | 2023-04-27 | Steven Goldman | Isolated glial progenitor cells for use in the competition treatment of age-related white matter loss |
-
2022
- 2022-10-19 WO PCT/US2022/047079 patent/WO2023069478A1/en not_active Ceased
- 2022-10-19 JP JP2024523274A patent/JP2024539080A/ja active Pending
- 2022-10-19 WO PCT/US2022/047085 patent/WO2023069484A1/en not_active Ceased
- 2022-10-19 US US17/968,984 patent/US12565648B2/en active Active
- 2022-10-19 US US18/047,744 patent/US20230190961A1/en active Pending
- 2022-10-19 EP EP22823646.9A patent/EP4419685A1/en active Pending
- 2022-10-19 JP JP2024523275A patent/JP2024539081A/ja active Pending
- 2022-10-19 EP EP22803427.8A patent/EP4419679A1/en active Pending
- 2022-10-19 WO PCT/US2022/047083 patent/WO2023069482A1/en not_active Ceased
- 2022-10-19 US US18/047,749 patent/US20230133695A1/en active Pending
- 2022-10-19 CA CA3235458A patent/CA3235458A1/en active Pending
- 2022-10-19 AU AU2022370552A patent/AU2022370552A1/en active Pending
- 2022-10-19 CA CA3235250A patent/CA3235250A1/en active Pending
- 2022-10-19 AU AU2022369984A patent/AU2022369984A1/en active Pending
- 2022-10-19 CA CA3234634A patent/CA3234634A1/en active Pending
-
2023
- 2023-09-14 US US18/467,442 patent/US20240167034A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2024539081A (ja) | 2024-10-28 |
| WO2023069478A8 (en) | 2023-07-27 |
| US20230133695A1 (en) | 2023-05-04 |
| WO2023069478A9 (en) | 2023-08-24 |
| CA3235458A1 (en) | 2023-04-27 |
| JP2024539080A (ja) | 2024-10-28 |
| WO2023069484A1 (en) | 2023-04-27 |
| WO2023069478A1 (en) | 2023-04-27 |
| EP4419679A1 (en) | 2024-08-28 |
| WO2023069482A1 (en) | 2023-04-27 |
| US20230212568A1 (en) | 2023-07-06 |
| CA3234634A1 (en) | 2023-04-27 |
| US12565648B2 (en) | 2026-03-03 |
| US20230190961A1 (en) | 2023-06-22 |
| AU2022369984A1 (en) | 2024-03-28 |
| CA3235250A1 (en) | 2023-04-27 |
| US20240167034A1 (en) | 2024-05-23 |
| EP4419685A1 (en) | 2024-08-28 |
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| Rochat et al. | Expression of a miRNA targeting mutated SOD1 in astrocytes induces motoneuron plasticity and improves neuromuscular function in ALS mice | |
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| Abdalla-Wyse et al. | microRNA Expression in the Late Embryonic Pax6-Null Mouse Cerebellum | |
| Giagnorio | Revealing the involvement of MALAT1, NEAT1, HOTTIP lncRNAs in Amyotrophic Lateral Sclerosis (ALS) via an induced pluripotent stem cell (iPSC)-derived muscle cell model | |
| He | Epigenetic Regulation of Oligodendrocyte Development and Regeneration in the Central Nervous System | |
| Wapinski | Mechanistic Understanding of Cell Fate Decisions Towards Neuronal Lineage |