CA3005246A1 - Compositions and methods for treatment of central nervous system diseases - Google Patents
Compositions and methods for treatment of central nervous system diseases Download PDFInfo
- Publication number
- CA3005246A1 CA3005246A1 CA3005246A CA3005246A CA3005246A1 CA 3005246 A1 CA3005246 A1 CA 3005246A1 CA 3005246 A CA3005246 A CA 3005246A CA 3005246 A CA3005246 A CA 3005246A CA 3005246 A1 CA3005246 A1 CA 3005246A1
- Authority
- CA
- Canada
- Prior art keywords
- mrna
- nucleobases
- ric pre
- intron
- retained intron
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
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Classifications
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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
- 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
- C12N15/67—General methods for enhancing the expression
-
- 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
-
- 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
-
- 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
- A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
-
- 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
-
- 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
- A61P27/16—Otologicals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
- C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/158—Expression markers
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Analytical Chemistry (AREA)
- Molecular Biology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Psychiatry (AREA)
- Immunology (AREA)
- Pain & Pain Management (AREA)
- Hospice & Palliative Care (AREA)
- Plant Pathology (AREA)
- Pathology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267256P | 2015-12-14 | 2015-12-14 | |
| US62/267,256 | 2015-12-14 | ||
| US201662319011P | 2016-04-06 | 2016-04-06 | |
| US62/319,011 | 2016-04-06 | ||
| PCT/US2016/066721 WO2017106382A1 (en) | 2015-12-14 | 2016-12-14 | Compositions and methods for treatment of central nervous system diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3005246A1 true CA3005246A1 (en) | 2017-06-22 |
Family
ID=59057524
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3005246A Pending CA3005246A1 (en) | 2015-12-14 | 2016-12-14 | Compositions and methods for treatment of central nervous system diseases |
Country Status (4)
| Country | Link |
|---|---|
| EP (2) | EP3389725B1 (https=) |
| JP (2) | JP7049249B2 (https=) |
| CA (1) | CA3005246A1 (https=) |
| WO (1) | WO2017106382A1 (https=) |
Families Citing this family (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| AU2015327836B2 (en) | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| EP3390635A4 (en) * | 2015-12-14 | 2019-05-01 | Cold Spring Harbor Laboratory | Antisense oligomers for the treatment of a tuberous sclerosis complex |
| WO2017106377A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
| RU2019126483A (ru) | 2017-01-23 | 2021-02-24 | Ридженерон Фармасьютикалз, Инк. | Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение |
| KR20190139869A (ko) | 2017-04-11 | 2019-12-18 | 리제너론 파마슈티칼스 인코포레이티드 | 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정 |
| KR102318434B1 (ko) | 2017-08-25 | 2021-11-01 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| EP3461837A1 (en) * | 2017-09-28 | 2019-04-03 | Secarna Pharmaceuticals GmbH & Co. KG | Inhibitor inhibiting the expression of pprx1 |
| AU2018348195B2 (en) | 2017-10-11 | 2025-05-15 | Regeneron Pharmaceuticals, Inc. | Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation |
| HUE070436T2 (hu) * | 2017-10-23 | 2025-06-28 | Stoke Therapeutics Inc | Antiszensz oligomerek nonszensz-mediált RNS-lebomláson alapuló állapotok és betegségek kezelésére |
| AU2019239971B2 (en) | 2018-03-21 | 2025-09-11 | Regeneron Pharmaceuticals, Inc. | 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof |
| CA3099280A1 (en) | 2018-05-04 | 2019-11-07 | Stoke Therapeutics, Inc. | Methods and compositions for treatment of cholesteryl ester storage disease |
| BR112021003224A2 (pt) | 2018-08-20 | 2021-07-20 | Rogcon, Inc. | oligonucleotídeos antissentido direcionados a scn2a para o tratamento de encefalopatias por scn1a |
| AU2020227825B2 (en) | 2019-02-27 | 2026-03-26 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| US12595479B2 (en) | 2019-03-20 | 2026-04-07 | President And Fellows Of Harvard College | Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases |
| EP3965777A4 (en) * | 2019-05-06 | 2024-08-07 | The Children's Hospital of Philadelphia | MICRORNA SITE-BLOCKING OLIGONUCLEOTIDES FOR THE TREATMENT OF EPILEPTIC ENCEPHALOPATHY AND NEURODEVELOPMENTAL DISORDERS |
| CA3140049A1 (en) * | 2019-05-14 | 2020-11-19 | Duke University | Compositions and methods for the treatment of atpase-mediated diseases |
| US20220333110A1 (en) * | 2019-09-30 | 2022-10-20 | The University Of Tokyo | Nucleic acid that inhibits expression of mex3b gene, mex3b gene expression inhibiting agent, method for inhibiting mex3b gene expression, and prophylactic or therapeutic agent for disease caused by mex3b gene expression |
| EP4143317A1 (en) | 2020-04-27 | 2023-03-08 | Universität Ulm | Antisense oligonucleotides for increasing shank3 expression |
| CN115867657A (zh) | 2020-05-11 | 2023-03-28 | 斯托克制药公司 | 用于治疗疾患和疾病的opa1反义寡聚物 |
| WO2022140264A1 (en) * | 2020-12-23 | 2022-06-30 | Eli Lilly And Company | Rna therapeutics and methods of use thereof |
| WO2022215066A1 (en) * | 2021-04-05 | 2022-10-13 | Itayandbiond Ltd | Small activating rna increasing shank expression and method of treating intellectual disabilities and associated comorbidities associated with shank haploinsufficiency |
| WO2023104693A1 (en) * | 2021-12-07 | 2023-06-15 | F. Hoffmann-La Roche Ag | Antisense oligonucleotides targeting actl6b |
| CN118647405A (zh) * | 2022-01-25 | 2024-09-13 | 上海魁特迪生物科技有限公司 | 改善认知障碍的方法 |
| WO2023168427A1 (en) | 2022-03-03 | 2023-09-07 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
| CN114836532A (zh) * | 2022-04-28 | 2022-08-02 | 福建医科大学附属第一医院 | Eif2b5基因突变作为靶标在诊断消融性白质脑病中的应用 |
| WO2024126654A1 (en) * | 2022-12-14 | 2024-06-20 | F. Hoffmann-La Roche Ag | Antisense oligonucleotides targeting actl6b |
| EP4702143A2 (en) * | 2023-04-24 | 2026-03-04 | BioMarin Pharmaceutical Inc. | Compositions and methods for treating stxbp1 disorders |
| WO2025038842A1 (en) * | 2023-08-15 | 2025-02-20 | The Children's Medical Center Corporation | Systems and methods for modifying a polynucleotide |
| CN117122688B (zh) * | 2023-08-31 | 2024-06-04 | 中国科学院脑科学与智能技术卓越创新中心 | 作用于前脑兴奋性神经元的prrt2及其上调剂的应用 |
| WO2025217494A1 (en) * | 2024-04-11 | 2025-10-16 | The University Of Chicago | Splice-switching oligonucleotides for treating cacna1a-associated disorders |
| WO2025250031A1 (en) * | 2024-05-31 | 2025-12-04 | Bial - Portela & Ca., S.A. | Antisense oligonucleotides for treatment of stxbp1-related developmental epileptic encephalopathy |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4866042A (en) | 1987-11-18 | 1989-09-12 | Neuwelt Edward A | Method for the delivery of genetic material across the blood brain barrier |
| US6294520B1 (en) | 1989-03-27 | 2001-09-25 | Albert T. Naito | Material for passage through the blood-brain barrier |
| US5151510A (en) | 1990-04-20 | 1992-09-29 | Applied Biosystems, Inc. | Method of synethesizing sulfurized oligonucleotide analogs |
| ATE404683T1 (de) | 1992-09-25 | 2008-08-15 | Aventis Pharma Sa | Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn |
| US5656612A (en) | 1994-05-31 | 1997-08-12 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of raf gene expression |
| FR2727867B1 (fr) | 1994-12-13 | 1997-01-31 | Rhone Poulenc Rorer Sa | Transfert de genes dans les motoneurones medullaires au moyen de vecteurs adenoviraux |
| US6936589B2 (en) | 2001-09-28 | 2005-08-30 | Albert T. Naito | Parenteral delivery systems |
| WO2007047913A2 (en) | 2005-10-20 | 2007-04-26 | Isis Pharmaceuticals, Inc | Compositions and methods for modulation of lmna expression |
| WO2009073809A2 (en) | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| JP5608863B2 (ja) | 2007-12-28 | 2014-10-15 | 公立大学法人横浜市立大学 | 新生児期〜乳児期発症の難治性てんかんの検出方法 |
| ES2699827T3 (es) | 2009-06-17 | 2019-02-13 | Biogen Ma Inc | Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto |
| WO2012138487A2 (en) * | 2011-04-07 | 2012-10-11 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
| WO2012178122A2 (en) * | 2011-06-23 | 2012-12-27 | Cold Spring Harbor Laboratory | Phenocopy model of disease |
| CN103796657B (zh) | 2011-07-19 | 2017-07-11 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| EP3041958B1 (en) | 2013-09-04 | 2019-12-04 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
-
2016
- 2016-12-14 JP JP2018529257A patent/JP7049249B2/ja not_active Expired - Fee Related
- 2016-12-14 EP EP16876621.0A patent/EP3389725B1/en active Active
- 2016-12-14 EP EP22166699.3A patent/EP4104867A3/en not_active Withdrawn
- 2016-12-14 CA CA3005246A patent/CA3005246A1/en active Pending
- 2016-12-14 WO PCT/US2016/066721 patent/WO2017106382A1/en not_active Ceased
-
2022
- 2022-02-01 JP JP2022013867A patent/JP2022062141A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3389725A1 (en) | 2018-10-24 |
| JP2019500349A (ja) | 2019-01-10 |
| EP3389725A4 (en) | 2019-04-10 |
| JP7049249B2 (ja) | 2022-04-06 |
| JP2022062141A (ja) | 2022-04-19 |
| EP4104867A3 (en) | 2023-03-01 |
| EP4104867A2 (en) | 2022-12-21 |
| EP3389725B1 (en) | 2022-04-06 |
| WO2017106382A1 (en) | 2017-06-22 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20211213 |