CA2997909A1 - Treatment of retinitis pigmentosa using engineered meganucleases - Google Patents
Treatment of retinitis pigmentosa using engineered meganucleases Download PDFInfo
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- CA2997909A1 CA2997909A1 CA2997909A CA2997909A CA2997909A1 CA 2997909 A1 CA2997909 A1 CA 2997909A1 CA 2997909 A CA2997909 A CA 2997909A CA 2997909 A CA2997909 A CA 2997909A CA 2997909 A1 CA2997909 A1 CA 2997909A1
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- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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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
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- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
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- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562215460P | 2015-09-08 | 2015-09-08 | |
| US62/215,460 | 2015-09-08 | ||
| PCT/US2016/050809 WO2017044649A1 (en) | 2015-09-08 | 2016-09-08 | Treatment of retinitis pigmentosa using engineered meganucleases |
Publications (1)
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| CA2997909A1 true CA2997909A1 (en) | 2017-03-16 |
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| CA2997909A Pending CA2997909A1 (en) | 2015-09-08 | 2016-09-08 | Treatment of retinitis pigmentosa using engineered meganucleases |
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| EP (2) | EP3347463B1 (OSRAM) |
| JP (5) | JP6775584B2 (OSRAM) |
| AU (3) | AU2016321244B2 (OSRAM) |
| CA (1) | CA2997909A1 (OSRAM) |
| WO (1) | WO2017044649A1 (OSRAM) |
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| EP3114227B1 (en) | 2014-03-05 | 2021-07-21 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa |
| EP3126495A1 (en) | 2014-04-02 | 2017-02-08 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating primary open angle glaucoma |
| WO2016172727A1 (en) | 2015-04-24 | 2016-10-27 | Editas Medicine, Inc. | Evaluation of cas9 molecule/guide rna molecule complexes |
| EP3347463B1 (en) * | 2015-09-08 | 2024-11-13 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
| US11680254B2 (en) * | 2017-04-21 | 2023-06-20 | Precision Biosciences, Inc. | Engineered meganucleases specific for recognition sequences in the PCSK9 gene |
| MA50877A (fr) * | 2017-11-21 | 2020-09-30 | Bayer Healthcare Llc | Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante |
| CA3137975A1 (en) | 2019-05-07 | 2020-11-12 | Precision Biosciences, Inc. | Optimization of engineered meganucleases for recognition sequences |
| US20230033866A1 (en) * | 2019-12-06 | 2023-02-02 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of rhodopsin |
| WO2021212686A1 (zh) * | 2020-04-21 | 2021-10-28 | 北京中因科技有限公司 | RHO-adRP基于基因编辑的方法和组合物 |
| US20230183664A1 (en) | 2020-05-11 | 2023-06-15 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| WO2021231495A1 (en) * | 2020-05-12 | 2021-11-18 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using improved engineered meganucleases |
| EP4419660A2 (en) * | 2021-10-19 | 2024-08-28 | Precision BioSciences, Inc. | Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency |
| WO2025101946A1 (en) | 2023-11-08 | 2025-05-15 | Precision Biosciences, Inc. | Polypeptide linkers for use in engineered meganucleases |
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| US4873192A (en) | 1987-02-17 | 1989-10-10 | The United States Of America As Represented By The Department Of Health And Human Services | Process for site specific mutagenesis without phenotypic selection |
| US6015832A (en) | 1997-12-31 | 2000-01-18 | The Regents Of The University Of Michigan | Methods of inactivating bacteria including bacterial spores |
| US6635676B2 (en) | 1999-04-28 | 2003-10-21 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US7767216B2 (en) | 1999-04-28 | 2010-08-03 | The Regents Of The University Of Michigan | Antimicrobial compositions and methods of use |
| US6559189B2 (en) | 1999-04-28 | 2003-05-06 | Regents Of The University Of Michigan | Non-toxic antimicrobial compositions and methods of use |
| US6506803B1 (en) | 1999-04-28 | 2003-01-14 | Regents Of The University Of Michigan | Methods of preventing and treating microbial infections |
| DK2628794T3 (en) | 2005-10-18 | 2016-08-15 | Prec Biosciences | RATIONALE CONSTRUCTED MECHANUCLEAS WITH CHANGED SEQUENCE SPECIFICITY AND DNA BINDING EFFICIENCY |
| ES2732735T3 (es) | 2007-10-31 | 2019-11-25 | Prec Biosciences Inc | Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas |
| US20130071373A1 (en) * | 2010-02-02 | 2013-03-21 | Christina Zeitz | Methods for the diagnosis and therapy of retinitis pigmentosa |
| US20130183282A1 (en) * | 2010-05-12 | 2013-07-18 | Cellectis | Meganuclease variants cleaving a DNA target sequence from the rhodopsin gene and uses thereof |
| US20120204282A1 (en) | 2011-02-04 | 2012-08-09 | Sangamo Biosciences, Inc. | Methods and compositions for treating occular disorders |
| DK2714936T3 (en) * | 2011-06-01 | 2019-03-25 | Prec Biosciences Inc | METHODS AND PRODUCTS FOR PRODUCING MANIPULATED MAMMAL CELL LINES WITH AMPLIFIED TRANSGENES |
| US20140287510A1 (en) | 2011-11-08 | 2014-09-25 | Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute | Rod cell-specific promoter |
| KR20250068794A (ko) * | 2013-12-12 | 2025-05-16 | 더 브로드 인스티튜트, 인코퍼레이티드 | 게놈 편집을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 용도 및 치료적 응용 |
| WO2015138739A2 (en) * | 2014-03-12 | 2015-09-17 | Precision Biosciences, Inc. | Dystrophin gene oxon deletion using engineered nucleases |
| EP3347463B1 (en) | 2015-09-08 | 2024-11-13 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
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2016
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2020
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| US10603363B2 (en) | 2020-03-31 |
| US20200179495A1 (en) | 2020-06-11 |
| EP3347463A1 (en) | 2018-07-18 |
| EP3347463B1 (en) | 2024-11-13 |
| US20180250370A1 (en) | 2018-09-06 |
| AU2020294264A1 (en) | 2021-01-28 |
| WO2017044649A1 (en) | 2017-03-16 |
| JP7068413B2 (ja) | 2022-05-16 |
| AU2023210619A1 (en) | 2023-08-24 |
| US20250121039A1 (en) | 2025-04-17 |
| EP4530354A2 (en) | 2025-04-02 |
| AU2016321244B2 (en) | 2020-09-24 |
| JP2021054824A (ja) | 2021-04-08 |
| EP3347463C0 (en) | 2024-11-13 |
| JP2018531624A (ja) | 2018-11-01 |
| US20210000928A1 (en) | 2021-01-07 |
| AU2016321244A1 (en) | 2018-04-12 |
| JP2022110004A (ja) | 2022-07-28 |
| US20240238387A1 (en) | 2024-07-18 |
| US20220143155A1 (en) | 2022-05-12 |
| US10758595B2 (en) | 2020-09-01 |
| JP6775584B2 (ja) | 2020-10-28 |
| JP2024023183A (ja) | 2024-02-21 |
| JP2025131592A (ja) | 2025-09-09 |
| EP4530354A3 (en) | 2025-09-10 |
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