BR112022002794A2 - Terapia combinada de transgene e mirna derivado de íntron para tratamento de sca1 - Google Patents
Terapia combinada de transgene e mirna derivado de íntron para tratamento de sca1Info
- Publication number
- BR112022002794A2 BR112022002794A2 BR112022002794A BR112022002794A BR112022002794A2 BR 112022002794 A2 BR112022002794 A2 BR 112022002794A2 BR 112022002794 A BR112022002794 A BR 112022002794A BR 112022002794 A BR112022002794 A BR 112022002794A BR 112022002794 A2 BR112022002794 A2 BR 112022002794A2
- Authority
- BR
- Brazil
- Prior art keywords
- intron
- sca1
- transgene
- treatment
- expression cassette
- Prior art date
Links
- 102000007372 Ataxin-1 Human genes 0.000 title abstract 3
- 108010032963 Ataxin-1 Proteins 0.000 title abstract 3
- 208000009415 Spinocerebellar Ataxias Diseases 0.000 title abstract 2
- 108700019146 Transgenes Proteins 0.000 title abstract 2
- 238000002648 combination therapy Methods 0.000 title abstract 2
- 201000003624 spinocerebellar ataxia type 1 Diseases 0.000 title abstract 2
- 230000001225 therapeutic effect Effects 0.000 abstract 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 2
- 108091070501 miRNA Proteins 0.000 abstract 2
- 239000002679 microRNA Substances 0.000 abstract 2
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 102000039446 nucleic acids Human genes 0.000 abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 102000004169 proteins and genes Human genes 0.000 abstract 2
- 102000014461 Ataxins Human genes 0.000 abstract 1
- 108010078286 Ataxins Proteins 0.000 abstract 1
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 108020004999 messenger RNA Proteins 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 229940124788 therapeutic inhibitor Drugs 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- 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
-
- 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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
-
- 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
-
- 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
-
- 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 nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/31—Combination therapy
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted 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)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962887209P | 2019-08-15 | 2019-08-15 | |
| PCT/US2020/046499 WO2021030745A1 (en) | 2019-08-15 | 2020-08-14 | Combined transgene and intron-derived mirna therapy for treatment of sca1 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022002794A2 true BR112022002794A2 (pt) | 2022-08-09 |
Family
ID=74570777
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022002794A BR112022002794A2 (pt) | 2019-08-15 | 2020-08-14 | Terapia combinada de transgene e mirna derivado de íntron para tratamento de sca1 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220288101A1 (https=) |
| EP (1) | EP4013414A4 (https=) |
| JP (1) | JP2022545378A (https=) |
| CN (1) | CN114555084A (https=) |
| AU (1) | AU2020330108A1 (https=) |
| BR (1) | BR112022002794A2 (https=) |
| CA (1) | CA3151122A1 (https=) |
| MX (1) | MX2022001984A (https=) |
| WO (1) | WO2021030745A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11008627B2 (en) | 2019-08-15 | 2021-05-18 | Talis Biomedical Corporation | Diagnostic system |
| WO2024044469A1 (en) * | 2022-08-26 | 2024-02-29 | The Children's Hospital Of Philadelphia | Mirnas targeting atnx2 for the treatment of als and sca2 |
| EP4651883A1 (en) * | 2023-01-19 | 2025-11-26 | uniQure biopharma B.V. | Pharmaceutical formulations of gene delivery vehicles |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7001733B1 (en) * | 1998-05-12 | 2006-02-21 | Rigel Pharmaceuticals, Inc. | Methods and compositions for screening for modulations of IgE synthesis, secretion and switch rearrangement |
| US20080274989A1 (en) * | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
| US7605249B2 (en) * | 2002-11-26 | 2009-10-20 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| DK2164967T3 (en) * | 2007-05-31 | 2015-10-19 | Univ Iowa Res Found | Reduction of off-target rna interferenstoksicitet |
| US8865881B2 (en) * | 2011-02-22 | 2014-10-21 | California Institute Of Technology | Delivery of proteins using adeno-associated virus (AAV) vectors |
| PL3628334T3 (pl) * | 2014-03-21 | 2023-12-18 | Genzyme Corporation | Terapia genowa w retinopatii barwnikowej |
| EP3146051B8 (en) * | 2014-05-20 | 2019-11-27 | University of Iowa Research Foundation | Huntington's disease therapeutic compounds |
| MA40782A (fr) * | 2014-10-03 | 2017-08-08 | Hocuslocus Llc | Procédés et compositions utilisant un arn non codant en vue de la culture et de la sélection de cellules |
| EP3261582B1 (en) * | 2015-02-26 | 2021-01-06 | Remodeless CV Ltd. | Methods and compositions relating to leptin antagonists |
| WO2016196507A1 (en) * | 2015-05-29 | 2016-12-08 | University Of Iowa Research Foundation | Methods of delivery of transgenes for treating brain diseases |
| CN115896106A (zh) * | 2016-03-01 | 2023-04-04 | 佛罗里达大学研究基金会有限公司 | 用于治疗显性视网膜色素变性的aav载体 |
| CA3075168A1 (en) * | 2017-09-08 | 2019-03-14 | Generation Bio Co. | Modified closed-ended dna (cedna) |
| KR20200119782A (ko) * | 2017-12-06 | 2020-10-20 | 오비드 테라퓨틱스 인크. | 발작 장애 치료 시 mir101 또는 mir128의 용도 |
| US20210355509A1 (en) * | 2018-08-07 | 2021-11-18 | The Children's Hospital Of Philadelphia | Alternative splicing regulation of gene expression and therapeutic methods |
-
2020
- 2020-08-14 EP EP20851939.7A patent/EP4013414A4/en active Pending
- 2020-08-14 MX MX2022001984A patent/MX2022001984A/es unknown
- 2020-08-14 CN CN202080072237.4A patent/CN114555084A/zh active Pending
- 2020-08-14 WO PCT/US2020/046499 patent/WO2021030745A1/en not_active Ceased
- 2020-08-14 BR BR112022002794A patent/BR112022002794A2/pt unknown
- 2020-08-14 JP JP2022509153A patent/JP2022545378A/ja active Pending
- 2020-08-14 CA CA3151122A patent/CA3151122A1/en active Pending
- 2020-08-14 US US17/632,587 patent/US20220288101A1/en active Pending
- 2020-08-14 AU AU2020330108A patent/AU2020330108A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2022545378A (ja) | 2022-10-27 |
| WO2021030745A1 (en) | 2021-02-18 |
| CN114555084A (zh) | 2022-05-27 |
| EP4013414A4 (en) | 2023-09-27 |
| EP4013414A1 (en) | 2022-06-22 |
| MX2022001984A (es) | 2022-05-19 |
| US20220288101A1 (en) | 2022-09-15 |
| CA3151122A1 (en) | 2021-02-18 |
| AU2020330108A1 (en) | 2022-03-17 |
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