FI3700570T3 - Antisense-oligomeerejä nonsense-välitteiseen rna:n hajoamiseen pohjautuvien tilojen ja sairauksien hoitoon - Google Patents
Antisense-oligomeerejä nonsense-välitteiseen rna:n hajoamiseen pohjautuvien tilojen ja sairauksien hoitoon Download PDFInfo
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- FI3700570T3 FI3700570T3 FIEP18871437.2T FI18871437T FI3700570T3 FI 3700570 T3 FI3700570 T3 FI 3700570T3 FI 18871437 T FI18871437 T FI 18871437T FI 3700570 T3 FI3700570 T3 FI 3700570T3
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- mrna
- antisense oligomer
- nmd exon
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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
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
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- 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
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- 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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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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
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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
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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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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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
- A61P25/06—Antimigraine agents
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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
- A61P25/08—Antiepileptics; Anticonvulsants
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/06—Antiarrhythmics
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- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
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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
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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
- C12N2310/11—Antisense
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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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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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
- C12N2310/30—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
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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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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- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pain & Pain Management (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Cell Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Ophthalmology & Optometry (AREA)
- Mycology (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762575924P | 2017-10-23 | 2017-10-23 | |
| US201862667200P | 2018-05-04 | 2018-05-04 | |
| PCT/US2018/057165 WO2019084050A1 (en) | 2017-10-23 | 2018-10-23 | ANTISENSE OLIGOMERS FOR THE TREATMENT OF CONDITIONS AND DISEASES BASED ON THE DECLINE OF NON-SENSE MEDIATED MRNA |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| FI3700570T3 true FI3700570T3 (fi) | 2025-03-31 |
Family
ID=66247689
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| FIEP18871437.2T FI3700570T3 (fi) | 2017-10-23 | 2018-10-23 | Antisense-oligomeerejä nonsense-välitteiseen rna:n hajoamiseen pohjautuvien tilojen ja sairauksien hoitoon |
Country Status (23)
| Country | Link |
|---|---|
| US (2) | US20210268667A1 (enExample) |
| EP (2) | EP3700570B1 (enExample) |
| JP (2) | JP7475272B2 (enExample) |
| KR (2) | KR102774070B1 (enExample) |
| CN (2) | CN111936163B (enExample) |
| AU (1) | AU2018355237B2 (enExample) |
| BR (1) | BR112020007881A2 (enExample) |
| CA (1) | CA3079729A1 (enExample) |
| DK (1) | DK3700570T3 (enExample) |
| ES (1) | ES3015767T3 (enExample) |
| FI (1) | FI3700570T3 (enExample) |
| GB (2) | GB2610100B (enExample) |
| HR (1) | HRP20250322T1 (enExample) |
| HU (1) | HUE070436T2 (enExample) |
| IL (1) | IL274023A (enExample) |
| LT (1) | LT3700570T (enExample) |
| PL (1) | PL3700570T3 (enExample) |
| PT (1) | PT3700570T (enExample) |
| RS (1) | RS66633B1 (enExample) |
| SG (1) | SG11202003692YA (enExample) |
| SI (1) | SI3700570T1 (enExample) |
| SM (1) | SMT202500127T1 (enExample) |
| WO (1) | WO2019084050A1 (enExample) |
Families Citing this family (60)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013163628A2 (en) | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| WO2016040748A1 (en) | 2014-09-12 | 2016-03-17 | Ionis Pharmaceuticals, Inc. | Compositions and methods for detection of smn protein in a subject and treatment of a subject |
| KR102620328B1 (ko) | 2014-10-03 | 2024-01-02 | 콜드스프링하버러보러토리 | 핵 유전자 산출량의 표적화 증강 |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| KR20220105174A (ko) | 2015-10-09 | 2022-07-26 | 유니버시티 오브 사우스앰톤 | 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝 |
| WO2017066497A2 (en) | 2015-10-13 | 2017-04-20 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| EP4644567A2 (en) | 2015-11-30 | 2025-11-05 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| CN109312343B (zh) | 2015-12-14 | 2022-09-27 | 冷泉港实验室 | 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体 |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| WO2017218884A1 (en) | 2016-06-16 | 2017-12-21 | Ionis Pharmaceuticals, Inc. | Combinations for the modulation of smn expression |
| WO2018017754A1 (en) | 2016-07-19 | 2018-01-25 | Duke University | Therapeutic applications of cpf1-based genome editing |
| SMT202400071T1 (it) | 2017-08-25 | 2024-03-13 | Stoke Therapeutics Inc | Oligomeri antisenso per il trattamento di condizioni e malattie |
| US12060558B2 (en) | 2018-05-04 | 2024-08-13 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| CN109593771B (zh) * | 2018-07-27 | 2022-03-29 | 四川大学华西医院 | 一种人类map2k5第1100位碱基突变基因及其检测试剂盒 |
| WO2020191212A1 (en) | 2019-03-20 | 2020-09-24 | President And Fellows Of Harvard College | Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases |
| UA128437C2 (uk) | 2019-03-29 | 2024-07-10 | Айоніс Фармасьютикалз, Інк. | Сполуки і способи модулювання ube3a-ats |
| BR112022002905A2 (pt) * | 2019-08-19 | 2022-07-12 | Stoke Therapeutics Inc | Composições e métodos para modular splicing e expressão de proteína |
| US11618900B2 (en) * | 2019-11-01 | 2023-04-04 | The Johns Hopkins University | Modulating SYNGAP |
| JP2023504195A (ja) * | 2019-12-06 | 2023-02-01 | ストーク セラピューティクス，インク． | 状態および疾患の処置のためのアンチセンスオリゴマー |
| AU2021225957A1 (en) * | 2020-02-28 | 2022-09-08 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating SMN2 |
| US20230124616A1 (en) * | 2020-03-06 | 2023-04-20 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating kcnq2 |
| US20230201375A1 (en) * | 2020-04-27 | 2023-06-29 | Duke University | Targeted genomic integration to restore neurofibromin coding sequence in neurofibromatosis type 1 (nf1) |
| CA3173647A1 (en) | 2020-05-11 | 2021-11-18 | Isabel AZNAREZ | Opa1 antisense oligomers for treatment of conditions and diseases |
| US20230174984A1 (en) * | 2020-05-11 | 2023-06-08 | The Florey Indtitute of Neuroscience and Mental Health | Compositions and methods for treating disorders associated with loss-of-function mutations in syngap1 |
| CN116368227A (zh) * | 2020-07-22 | 2023-06-30 | 弗洛里神经科学与心理健康研究所 | 用于治疗与scn2a中功能失去突变相关的障碍的组合物和方法 |
| WO2022032060A2 (en) | 2020-08-07 | 2022-02-10 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating scn2a |
| CA3193706A1 (en) * | 2020-10-02 | 2022-04-07 | PYC Therapeutics Limited | Treatment of optic atrophy |
| US20240018522A1 (en) * | 2020-10-26 | 2024-01-18 | Remix Therapeutics Inc. | Oligonucleotides useful for modulation of splicing |
| WO2022169947A2 (en) * | 2021-02-03 | 2022-08-11 | Stoke Therapeutics, Inc. | Compositions for treatment of conditions and diseases associated with polycystin expression |
| WO2022240795A1 (en) * | 2021-05-10 | 2022-11-17 | Q-State Biosciences, Inc. | Compositions targeting sodium channel 1.6 |
| KR20240012377A (ko) * | 2021-05-28 | 2024-01-29 | 빔 테라퓨틱스, 인크. | 염기 편집기의 자기-불활성화용 조성물 및 방법 |
| CN117980479A (zh) * | 2021-06-21 | 2024-05-03 | 斯托克制药公司 | 用于治疗基于无义介导的rna衰变的病状和疾病的反义寡聚体 |
| CN113584082B (zh) * | 2021-06-22 | 2023-07-25 | 复旦大学附属眼耳鼻喉科医院 | CRISPR/Cas9基因编辑系统及其在制备治疗遗传性感音神经性聋的药物中的应用 |
| CN113584038B (zh) * | 2021-09-09 | 2023-11-14 | 深圳雅济科技有限公司 | 一种治疗视网膜疾病的反义寡核苷酸组合及其应用 |
| US20240425882A1 (en) * | 2021-10-18 | 2024-12-26 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of cdkl5 deficiency disorder (cdd) |
| WO2023086342A2 (en) * | 2021-11-09 | 2023-05-19 | Stoke Therapeutics, Inc. | Opa1 antisense oligomers for treatment of conditions and diseases |
| CN114032323B (zh) * | 2021-11-17 | 2023-08-08 | 云南省烟草农业科学研究院 | 一种与雪茄烟抗黑胫病基因紧密连锁的共显性ssr标记及其应用 |
| WO2023102548A1 (en) * | 2021-12-03 | 2023-06-08 | Quralis Corporation | Treatment of neurological diseases using modulators of kcnq2 gene transcripts |
| EP4473109A1 (en) * | 2022-01-31 | 2024-12-11 | Pyc Therapeutics Limited | Method of treatment for optic atrophy |
| JP2025507683A (ja) * | 2022-02-24 | 2025-03-21 | キュー－ステート バイオサイエンシーズ， インコーポレイテッド | Ｓｙｎｇａｐハプロ不全のための治療剤 |
| CN116083427B (zh) * | 2022-04-22 | 2025-10-03 | 齐齐哈尔大学 | 大豆高温诱导型启动子及其应用 |
| CN115029347B (zh) * | 2022-05-11 | 2024-02-20 | 珠海中科先进技术研究院有限公司 | 识别和调控肝肾细胞纤维化的分子监测序列、重组质粒、抑制病毒 |
| WO2023235509A2 (en) * | 2022-06-01 | 2023-12-07 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases |
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