CA3005254A1 - Compositions and methods for treatment of retinitis pigmentosa 18 and retinitis pigmentosa 13 - Google Patents
Compositions and methods for treatment of retinitis pigmentosa 18 and retinitis pigmentosa 13 Download PDFInfo
- Publication number
- CA3005254A1 CA3005254A1 CA3005254A CA3005254A CA3005254A1 CA 3005254 A1 CA3005254 A1 CA 3005254A1 CA 3005254 A CA3005254 A CA 3005254A CA 3005254 A CA3005254 A CA 3005254A CA 3005254 A1 CA3005254 A1 CA 3005254A1
- Authority
- CA
- Canada
- Prior art keywords
- nucleobases
- mrna
- protein
- retained intron
- ric pre
- 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
Links
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
- 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
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- 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
- C12N2310/34—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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Ophthalmology & Optometry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267261P | 2015-12-14 | 2015-12-14 | |
| US62/267,261 | 2015-12-14 | ||
| PCT/US2016/066684 WO2017106364A2 (en) | 2015-12-14 | 2016-12-14 | Compositions and methods for treatment of retinitis pigmentosa 18 and retinitis pigmentosa 13 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3005254A1 true CA3005254A1 (en) | 2017-06-22 |
Family
ID=59057547
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3005254A Pending CA3005254A1 (en) | 2015-12-14 | 2016-12-14 | Compositions and methods for treatment of retinitis pigmentosa 18 and retinitis pigmentosa 13 |
Country Status (5)
| Country | Link |
|---|---|
| EP (1) | EP3390642B1 (enExample) |
| JP (2) | JP7054675B2 (enExample) |
| CA (1) | CA3005254A1 (enExample) |
| ES (1) | ES2903394T3 (enExample) |
| WO (1) | WO2017106364A2 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| CN107109411B (zh) | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| 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 |
| CN116059236A (zh) | 2015-12-14 | 2023-05-05 | 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 |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| AU2017229778A1 (en) | 2016-03-09 | 2018-08-16 | Ionis Pharmaceuticals, Inc. | Methods and compositions for inhibiting PMP22 expression |
| CA3049980A1 (en) | 2017-01-23 | 2018-07-26 | Regeneron Pharmaceuticals, Inc. | Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof |
| CA3059348A1 (en) | 2017-04-11 | 2018-10-18 | Regeneron Pharmaceuticals, Inc. | Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family |
| KR20260009385A (ko) * | 2017-08-25 | 2026-01-19 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| 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 |
| CN112218949B (zh) * | 2018-03-02 | 2025-02-11 | 莱顿大学医学中心附属莱顿教学医院 | 多瘤病毒复制的抑制 |
| MY207352A (en) | 2018-03-21 | 2025-02-21 | Regeneron Pharma | 17-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof |
| JP2021523227A (ja) | 2018-05-04 | 2021-09-02 | ストーク セラピューティクス，インク． | コレステリルエステル蓄積症の処置のための方法及び組成物 |
| EP3897837A4 (en) * | 2018-12-21 | 2023-08-16 | Ionis Pharmaceuticals, Inc. | COMPOUNDS AND METHODS FOR REDUCING PMP22 EXPRESSION |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| US12338437B2 (en) | 2020-05-11 | 2025-06-24 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
Family Cites Families (13)
| 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 |
| DE69334238D1 (de) | 1992-09-25 | 2008-09-25 | 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 |
| US8258109B2 (en) | 2005-10-20 | 2012-09-04 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of LMNA expression |
| EP2229186A2 (en) | 2007-12-04 | 2010-09-22 | Alnylam Pharmaceuticals Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| KR20220084437A (ko) | 2009-06-17 | 2022-06-21 | 바이오젠 엠에이 인코포레이티드 | 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법 |
| CN107365339A (zh) | 2011-07-19 | 2017-11-21 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| CN104004826B (zh) * | 2013-01-07 | 2016-03-02 | 赵晨 | 突变的基因prpf4在制备遗传性视网膜疾病诊断试剂中的应用 |
| WO2015035091A1 (en) | 2013-09-04 | 2015-03-12 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
-
2016
- 2016-12-14 ES ES16876606T patent/ES2903394T3/es active Active
- 2016-12-14 JP JP2018529240A patent/JP7054675B2/ja not_active Expired - Fee Related
- 2016-12-14 WO PCT/US2016/066684 patent/WO2017106364A2/en not_active Ceased
- 2016-12-14 CA CA3005254A patent/CA3005254A1/en active Pending
- 2016-12-14 EP EP16876606.1A patent/EP3390642B1/en active Active
-
2022
- 2022-04-04 JP JP2022062557A patent/JP2022088621A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| ES2903394T3 (es) | 2022-04-01 |
| WO2017106364A2 (en) | 2017-06-22 |
| EP3390642A4 (en) | 2019-06-26 |
| EP3390642A2 (en) | 2018-10-24 |
| JP2022088621A (ja) | 2022-06-14 |
| JP2019500347A (ja) | 2019-01-10 |
| WO2017106364A3 (en) | 2017-07-13 |
| EP3390642B1 (en) | 2021-11-24 |
| JP7054675B2 (ja) | 2022-04-14 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20211213 |