IL313726A - Medical treatment of fragile X-linked disorder - Google Patents
Medical treatment of fragile X-linked disorderInfo
- Publication number
- IL313726A IL313726A IL313726A IL31372624A IL313726A IL 313726 A IL313726 A IL 313726A IL 313726 A IL313726 A IL 313726A IL 31372624 A IL31372624 A IL 31372624A IL 313726 A IL313726 A IL 313726A
- Authority
- IL
- Israel
- Prior art keywords
- fragile
- therapeutic treatment
- associated disorder
- disorder
- therapeutic
- Prior art date
Links
- 230000001225 therapeutic effect Effects 0.000 title 1
Classifications
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
- C12N2310/11—Antisense
-
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- 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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
-
- 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
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163265989P | 2021-12-23 | 2021-12-23 | |
| PCT/US2022/082380 WO2023122800A1 (en) | 2021-12-23 | 2022-12-23 | Therapeutic treatment for fragile x-associated disorder |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL313726A true IL313726A (en) | 2024-08-01 |
Family
ID=85199427
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL313726A IL313726A (en) | 2021-12-23 | 2022-12-23 | Medical treatment of fragile X-linked disorder |
Country Status (9)
| Country | Link |
|---|---|
| EP (1) | EP4453212A1 (https=) |
| JP (1) | JP2025501755A (https=) |
| KR (1) | KR20240133725A (https=) |
| CN (1) | CN118679257A (https=) |
| AU (1) | AU2022417615A1 (https=) |
| CA (1) | CA3240593A1 (https=) |
| IL (1) | IL313726A (https=) |
| MX (1) | MX2024007908A (https=) |
| WO (1) | WO2023122800A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025240947A1 (en) * | 2024-05-17 | 2025-11-20 | University Of Massachusetts | Therapeutic treatment for fragile x-associated disorder |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3854480A (en) | 1969-04-01 | 1974-12-17 | Alza Corp | Drug-delivery system |
| US4452775A (en) | 1982-12-03 | 1984-06-05 | Syntex (U.S.A.) Inc. | Cholesterol matrix delivery system for sustained release of macromolecules |
| US5039660A (en) | 1988-03-02 | 1991-08-13 | Endocon, Inc. | Partially fused peptide pellet |
| WO1998010090A1 (en) | 1996-09-06 | 1998-03-12 | St. Elizabeth's Medical Center Of Boston, Inc. | Gata-6 transcription factor: compositions and methods |
| EP2595663A4 (en) * | 2010-07-19 | 2014-03-05 | Isis Pharmaceuticals Inc | MODULATION OF DYSTROPHIA MYOTONICA PROTEIN KINASE (DMPK) EXPRESSION |
| EP2723902A4 (en) * | 2011-06-24 | 2015-02-18 | Murdoch Childrens Res Inst | TREATMENT AND DIAGNOSIS OF EPIGENETIC DISORDERS AND DISEASES |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| AU2013335451C1 (en) | 2012-10-23 | 2024-07-04 | Toolgen Incorporated | Composition for cleaving a target DNA comprising a guide RNA specific for the target DNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof |
| WO2014093655A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains |
| CN113355357B (zh) | 2012-12-12 | 2024-12-03 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
| US8697359B1 (en) | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| US20140310830A1 (en) | 2012-12-12 | 2014-10-16 | Feng Zhang | CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes |
| EP2840140B2 (en) | 2012-12-12 | 2023-02-22 | The Broad Institute, Inc. | Crispr-Cas based method for mutation of prokaryotic cells |
| IL239344B2 (en) | 2012-12-12 | 2024-06-01 | Broad Inst Inc | Systems engineering, methods and optimal guiding components for sequence manipulation |
| CA2921457A1 (en) * | 2013-08-16 | 2015-02-19 | Rana Therapeutics, Inc. | Heterochromatin forming non-coding rnas |
| US9790490B2 (en) | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| WO2018170290A1 (en) * | 2017-03-15 | 2018-09-20 | Fulcrum Therapeutics, Inc. | Compositions and methods for increasing fmr1 expression |
| GB2574243A (en) * | 2018-05-31 | 2019-12-04 | The Govening Council Of The Univ Of Toronto | Adeno-associated viral vector-mediated gene therapy for treating fragile X-associated disorders |
| CA3130854A1 (en) * | 2019-02-26 | 2020-09-03 | Nogra Pharma Limited | Fragile x mental retardation protein interfering oligonucleotides and methods of using same |
| CN120022385A (zh) * | 2019-05-07 | 2025-05-23 | 洛桑联邦理工学院 | Fmrp及癌症治疗 |
| CN113151406B (zh) * | 2021-03-25 | 2022-06-21 | 深圳会众生物技术有限公司 | Fmr1基因cgg重复数及甲基化检测试剂盒和检测方法 |
-
2022
- 2022-12-23 JP JP2024538030A patent/JP2025501755A/ja active Pending
- 2022-12-23 CA CA3240593A patent/CA3240593A1/en active Pending
- 2022-12-23 IL IL313726A patent/IL313726A/en unknown
- 2022-12-23 MX MX2024007908A patent/MX2024007908A/es unknown
- 2022-12-23 KR KR1020247024797A patent/KR20240133725A/ko active Pending
- 2022-12-23 WO PCT/US2022/082380 patent/WO2023122800A1/en not_active Ceased
- 2022-12-23 AU AU2022417615A patent/AU2022417615A1/en active Pending
- 2022-12-23 EP EP22854728.7A patent/EP4453212A1/en active Pending
- 2022-12-23 CN CN202280089598.9A patent/CN118679257A/zh active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| MX2024007908A (es) | 2024-09-10 |
| EP4453212A1 (en) | 2024-10-30 |
| WO2023122800A1 (en) | 2023-06-29 |
| CN118679257A (zh) | 2024-09-20 |
| JP2025501755A (ja) | 2025-01-23 |
| KR20240133725A (ko) | 2024-09-04 |
| CA3240593A1 (en) | 2023-06-29 |
| AU2022417615A1 (en) | 2024-06-27 |
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