IL285680A - Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp) - Google Patents
Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp)Info
- Publication number
- IL285680A IL285680A IL285680A IL28568021A IL285680A IL 285680 A IL285680 A IL 285680A IL 285680 A IL285680 A IL 285680A IL 28568021 A IL28568021 A IL 28568021A IL 285680 A IL285680 A IL 285680A
- Authority
- IL
- Israel
- Prior art keywords
- crispr
- rho
- rna
- compositions
- treatment
- Prior art date
Links
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
- C12N15/11—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
- 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
- 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
-
- 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
-
- 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
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
-
- 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
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Mycology (AREA)
- Epidemiology (AREA)
- Ophthalmology & Optometry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962810320P | 2019-02-25 | 2019-02-25 | |
| PCT/US2020/019766 WO2020176552A1 (en) | 2019-02-25 | 2020-02-25 | Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp) |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL285680A true IL285680A (en) | 2021-10-31 |
Family
ID=69960739
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL285680A IL285680A (en) | 2019-02-25 | 2021-08-17 | Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp) |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20220133768A1 (https=) |
| EP (1) | EP3931326A1 (https=) |
| JP (1) | JP2022521764A (https=) |
| KR (1) | KR20210133993A (https=) |
| CN (1) | CN113631710A (https=) |
| AU (1) | AU2020227740A1 (https=) |
| CA (1) | CA3130515A1 (https=) |
| IL (1) | IL285680A (https=) |
| WO (1) | WO2020176552A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020096049A1 (ja) * | 2018-11-08 | 2020-05-14 | 国立大学法人東北大学 | 単一のａａｖベクターによるゲノム編集を用いた遺伝子治療 |
| WO2021122944A1 (en) * | 2019-12-18 | 2021-06-24 | Alia Therapeutics Srl | Compositions and methods for treating retinitis pigmentosa |
| WO2022094363A1 (en) * | 2020-11-01 | 2022-05-05 | University of South Alabama Foundation for Research and Commercialization | Barcoded cells engineered with heterozygous genetic diversity |
| EP4323522A1 (en) * | 2021-04-16 | 2024-02-21 | Editas Medicine, Inc. | Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp) |
| WO2023285431A1 (en) * | 2021-07-12 | 2023-01-19 | Alia Therapeutics Srl | Compositions and methods for allele specific treatment of retinitis pigmentosa |
| US20260048074A1 (en) * | 2022-08-16 | 2026-02-19 | The University Of Adelaide | Agent for treating or preventing a dominantly-inherited disease |
| US20260014279A1 (en) * | 2022-09-16 | 2026-01-15 | Alia Therapeutics Srl | Enqp type cas proteins and applications thereof |
| US20260092271A1 (en) * | 2022-09-20 | 2026-04-02 | The Board Of Trustees Of The Leland Stanford Junior University | Methods and compositions to control gene using genome editing |
| CN116555259B (zh) * | 2023-04-20 | 2024-02-27 | 云舟生物科技(广州)股份有限公司 | 核酸分子及其作为启动子的应用 |
| WO2025090637A2 (en) * | 2023-10-23 | 2025-05-01 | Prime Medicine, Inc. | Genome editing compositions and methods for treatment of retinitis pigmentosa |
Family Cites Families (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5443505A (en) | 1993-11-15 | 1995-08-22 | Oculex Pharmaceuticals, Inc. | Biocompatible ocular implants |
| US5725493A (en) | 1994-12-12 | 1998-03-10 | Avery; Robert Logan | Intravitreal medicine delivery |
| US6299895B1 (en) | 1997-03-24 | 2001-10-09 | Neurotech S.A. | Device and method for treating ophthalmic diseases |
| US6416777B1 (en) | 1999-10-21 | 2002-07-09 | Alcon Universal Ltd. | Ophthalmic drug delivery device |
| ATE283013T1 (de) | 1999-10-21 | 2004-12-15 | Alcon Inc | Medikamentenzuführvorrichtung |
| AU2014361781B2 (en) * | 2013-12-12 | 2021-04-01 | Massachusetts Institute Of Technology | Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing |
| WO2015134812A1 (en) | 2014-03-05 | 2015-09-11 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| EP3152319A4 (en) * | 2014-06-05 | 2017-12-27 | Sangamo BioSciences, Inc. | Methods and compositions for nuclease design |
| DK3155101T3 (da) * | 2014-06-16 | 2020-05-04 | Univ Johns Hopkins | Sammensætninger og fremgangsmåder til ekspression af CRISPR-leder-RNA´er under anvendelse af H1-promotoren |
| US20160324987A1 (en) * | 2015-04-15 | 2016-11-10 | Cedars-Sinai Medical Center | Use of crispr/cas9 as in vivo gene therapy to generate targeted genomic disruptions in genes bearing dominant mutations for retinitis pigmentosa |
| EP3289080B1 (en) * | 2015-04-30 | 2021-08-25 | The Trustees of Columbia University in the City of New York | Gene therapy for autosomal dominant diseases |
| WO2017165862A1 (en) | 2016-03-25 | 2017-09-28 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| BR112019000057A2 (pt) * | 2016-07-05 | 2019-04-02 | The Johns Hopkins University | composições e métodos com base em crispr/cas9 para o tratamento de degeneração de retina |
| WO2018106693A1 (en) | 2016-12-05 | 2018-06-14 | Editas Medicine, Inc. | SYSTEMS AND METHODS FOR ONE-SHOT GUIDE RNA (ogRNA) TARGETING OF ENDOGENOUS AND SOURCE DNA |
| EP3622070A2 (en) * | 2017-05-10 | 2020-03-18 | Editas Medicine, Inc. | Crispr/rna-guided nuclease systems and methods |
| MA50877A (fr) * | 2017-11-21 | 2020-09-30 | Bayer Healthcare Llc | Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante |
| WO2019183630A2 (en) * | 2018-03-23 | 2019-09-26 | The Trustees Of Columbia University In The City Of New York | Gene editing for autosomal dominant diseases |
-
2020
- 2020-02-25 WO PCT/US2020/019766 patent/WO2020176552A1/en not_active Ceased
- 2020-02-25 AU AU2020227740A patent/AU2020227740A1/en not_active Abandoned
- 2020-02-25 CN CN202080025160.5A patent/CN113631710A/zh active Pending
- 2020-02-25 KR KR1020217030652A patent/KR20210133993A/ko active Pending
- 2020-02-25 US US17/433,975 patent/US20220133768A1/en active Pending
- 2020-02-25 EP EP20714062.5A patent/EP3931326A1/en active Pending
- 2020-02-25 JP JP2021549698A patent/JP2022521764A/ja active Pending
- 2020-02-25 CA CA3130515A patent/CA3130515A1/en active Pending
-
2021
- 2021-08-17 IL IL285680A patent/IL285680A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2020227740A1 (en) | 2021-10-07 |
| KR20210133993A (ko) | 2021-11-08 |
| EP3931326A1 (en) | 2022-01-05 |
| US20220133768A1 (en) | 2022-05-05 |
| JP2022521764A (ja) | 2022-04-12 |
| CN113631710A (zh) | 2021-11-09 |
| CA3130515A1 (en) | 2020-09-03 |
| WO2020176552A1 (en) | 2020-09-03 |
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