JP2017512767A5 - - Google Patents
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- JP2017512767A5 JP2017512767A5 JP2016556801A JP2016556801A JP2017512767A5 JP 2017512767 A5 JP2017512767 A5 JP 2017512767A5 JP 2016556801 A JP2016556801 A JP 2016556801A JP 2016556801 A JP2016556801 A JP 2016556801A JP 2017512767 A5 JP2017512767 A5 JP 2017512767A5
- Authority
- JP
- Japan
- Prior art keywords
- nuclease
- medicament according
- nucleases
- seq
- exon
- 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
- 101710163270 Nuclease Proteins 0.000 claims 22
- 239000003814 drug Substances 0.000 claims 14
- 238000000034 method Methods 0.000 claims 5
- 108020004414 DNA Proteins 0.000 claims 3
- 108010069091 Dystrophin Proteins 0.000 claims 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 2
- 108091033409 CRISPR Proteins 0.000 claims 2
- 238000010354 CRISPR gene editing Methods 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 238000010459 TALEN Methods 0.000 claims 2
- 108010043645 Transcription Activator-Like Effector Nucleases Proteins 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 1
- 102000001039 Dystrophin Human genes 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 238000011144 upstream manufacturing Methods 0.000 claims 1
- 108700026220 vif Genes Proteins 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461951648P | 2014-03-12 | 2014-03-12 | |
| US61/951,648 | 2014-03-12 | ||
| PCT/US2015/020205 WO2015138739A2 (en) | 2014-03-12 | 2015-03-12 | Dystrophin gene oxon deletion using engineered nucleases |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019152614A Division JP6832995B2 (ja) | 2014-03-12 | 2019-08-23 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2017512767A JP2017512767A (ja) | 2017-05-25 |
| JP2017512767A5 true JP2017512767A5 (OSRAM) | 2018-04-26 |
Family
ID=54072591
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016556801A Pending JP2017512767A (ja) | 2014-03-12 | 2015-03-12 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2019152614A Active JP6832995B2 (ja) | 2014-03-12 | 2019-08-23 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2021014851A Pending JP2021088563A (ja) | 2014-03-12 | 2021-02-02 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2022154637A Pending JP2023002555A (ja) | 2014-03-12 | 2022-09-28 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2024168117A Pending JP2025013784A (ja) | 2014-03-12 | 2024-09-27 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019152614A Active JP6832995B2 (ja) | 2014-03-12 | 2019-08-23 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2021014851A Pending JP2021088563A (ja) | 2014-03-12 | 2021-02-02 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2022154637A Pending JP2023002555A (ja) | 2014-03-12 | 2022-09-28 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
| JP2024168117A Pending JP2025013784A (ja) | 2014-03-12 | 2024-09-27 | 改変ヌクレアーゼを用いたジストロフィン遺伝子エクソンの欠失 |
Country Status (8)
| Country | Link |
|---|---|
| US (6) | US20170106055A1 (OSRAM) |
| EP (3) | EP3116533B1 (OSRAM) |
| JP (5) | JP2017512767A (OSRAM) |
| AU (3) | AU2015229299A1 (OSRAM) |
| CA (1) | CA2942268A1 (OSRAM) |
| DK (1) | DK3116533T3 (OSRAM) |
| ES (2) | ES2821149T3 (OSRAM) |
| WO (1) | WO2015138739A2 (OSRAM) |
Families Citing this family (53)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013066438A2 (en) | 2011-07-22 | 2013-05-10 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| EP3456831B1 (en) | 2013-04-16 | 2021-07-14 | Regeneron Pharmaceuticals, Inc. | Targeted modification of rat genome |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| KR20230054509A (ko) | 2013-11-07 | 2023-04-24 | 에디타스 메디신, 인코포레이티드 | 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물 |
| MX388127B (es) | 2013-12-11 | 2025-03-19 | Regeneron Pharma | Metodos y composiciones para la modificacion dirigida de un genoma. |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| CA3176380A1 (en) | 2014-11-21 | 2016-05-26 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modification using paired guide rnas |
| WO2016089866A1 (en) * | 2014-12-01 | 2016-06-09 | President And Fellows Of Harvard College | Rna-guided systems for in vivo gene editing |
| EP3748004A1 (en) * | 2015-04-01 | 2020-12-09 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy |
| WO2017044649A1 (en) * | 2015-09-08 | 2017-03-16 | Precision Biosciences, Inc. | Treatment of retinitis pigmentosa using engineered meganucleases |
| AU2016333886B2 (en) * | 2015-10-05 | 2020-10-08 | Precision Biosciences, Inc. | Engineered meganucleases with recognition sequences found in the human T cell receptor alpha constant region gene |
| EP4108255A1 (en) | 2015-10-05 | 2022-12-28 | Precision Biosciences, Inc. | Genetically-modified cells comprising a modified human t cell receptor alpha constant region gene |
| US12043852B2 (en) | 2015-10-23 | 2024-07-23 | President And Fellows Of Harvard College | Evolved Cas9 proteins for gene editing |
| WO2017201476A1 (en) | 2016-05-20 | 2017-11-23 | Regeneron Pharmaceuticals, Inc. | Methods for breaking immunological tolerance using multiple guide rnas |
| KR20250103795A (ko) | 2016-08-03 | 2025-07-07 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| CN109804066A (zh) | 2016-08-09 | 2019-05-24 | 哈佛大学的校长及成员们 | 可编程cas9-重组酶融合蛋白及其用途 |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| AU2017342543B2 (en) | 2016-10-14 | 2024-06-27 | President And Fellows Of Harvard College | AAV delivery of nucleobase editors |
| CA3044531A1 (en) * | 2016-11-28 | 2018-05-31 | The Board Of Regents Of The University Of Texas System | Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| JOP20190166A1 (ar) * | 2017-01-05 | 2019-07-02 | Univ Texas | استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Cancer vaccine |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| KR102758434B1 (ko) * | 2017-03-30 | 2025-01-21 | 고쿠리츠 다이가쿠 호진 교토 다이가쿠 | 게놈 편집에 의한 엑손 스키핑 유도 방법 |
| WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
| AU2018292526A1 (en) | 2017-06-30 | 2020-01-16 | Precision Biosciences, Inc. | Genetically-modified T cells comprising a modified intron in the T cell receptor alpha gene |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| CA3082251A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| WO2019200122A1 (en) | 2018-04-12 | 2019-10-17 | Precision Biosciences, Inc. | Optimized engineered nucleases having specificity for the human t cell receptor alpha constant region gene |
| WO2019226953A1 (en) | 2018-05-23 | 2019-11-28 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| WO2020106633A1 (en) * | 2018-11-19 | 2020-05-28 | Altius Institute For Biomedical Sciences | Compositions and methods for detection of cleavage of genomic dna by a nuclease |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| DE112020001306T5 (de) | 2019-03-19 | 2022-01-27 | Massachusetts Institute Of Technology | Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| US20220195407A1 (en) | 2019-05-07 | 2022-06-23 | Precision Biosciences, Inc. | Optimization of engineered meganucleases for recognition sequences |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| CN115484975A (zh) * | 2020-04-06 | 2022-12-16 | 同源药物公司 | 用于ids基因转移的腺相关病毒组合物和其使用方法 |
| EP4143308A4 (en) * | 2020-04-27 | 2024-06-26 | The Trustees of The University of Pennsylvania | COMPOSITIONS AND METHODS FOR REDUCING NUCLEASE EXPRESSION AND OFF-TARGET ACTIVITY USING A PROMOTER WITH LOW TRANSCRIPTION ACTIVITY |
| JP2023525304A (ja) | 2020-05-08 | 2023-06-15 | ザ ブロード インスティテュート，インコーポレーテッド | 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物 |
| WO2021231259A1 (en) | 2020-05-11 | 2021-11-18 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| EP4244342A1 (en) * | 2020-11-12 | 2023-09-20 | Precision BioSciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
| EP4444886A1 (en) * | 2021-12-08 | 2024-10-16 | Mammoth Biosciences, Inc. | Systems and uses thereof for the treatment of dmd-associated diseases |
| JP7691595B1 (ja) | 2024-02-26 | 2025-06-12 | 株式会社ニチレイフーズ | 欠失した改変ゲノムｄｎａを含む細胞の作製方法、該細胞を含む生物体の作製方法 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1590468A2 (en) * | 2003-01-28 | 2005-11-02 | Cellectis | Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof |
| ES2626025T3 (es) | 2005-10-18 | 2017-07-21 | Precision Biosciences | Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas |
| EP2660317B1 (en) | 2007-10-31 | 2016-04-06 | Precision Biosciences, Inc. | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| WO2009101399A1 (en) * | 2008-02-12 | 2009-08-20 | Isis Innovation Limited | Treatment of muscular dystrophy using peptide nucleic acid ( pna) |
| DK2313498T3 (en) | 2008-07-14 | 2017-05-22 | Prec Biosciences Inc | RECOGNITION SEQUENCES FOR I-CREI-DERIVED MECHANUCLEASES AND APPLICATIONS THEREOF |
| WO2011036640A2 (en) * | 2009-09-24 | 2011-03-31 | Cellectis | Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations |
| SG169914A1 (en) * | 2009-09-29 | 2011-04-29 | Univ Singapore | A clinical method for genotyping large genes for mutations that potentially cause disease |
| US20130145487A1 (en) * | 2010-05-12 | 2013-06-06 | Cellectis | Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof |
| JP2013544082A (ja) * | 2010-10-27 | 2013-12-12 | セレクティス | 二本鎖破断−誘導変異誘発の効率を増大させる方法 |
| DK3489366T3 (da) | 2011-06-01 | 2020-02-24 | Prec Biosciences Inc | Fremgangsmåder og produkter til produktion af manipulerede mammale cellelinier med forstærkede transgener |
| EP2766483B1 (en) * | 2011-10-10 | 2022-03-23 | The Hospital For Sick Children | Methods and compositions for screening and treating developmental disorders |
| US8885683B2 (en) * | 2011-12-21 | 2014-11-11 | Canon Kabushiki Kaisha | Process for forming microstructure of nitride semiconductor, surface emitting laser using two-dimensional photonic crystal and production process thereof |
| WO2013163628A2 (en) * | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
| EP2684892A1 (en) * | 2012-07-13 | 2014-01-15 | Association Française contre les Myopathies | Compositions and methods for duchenne muscular dystrophy gene therapy |
| BR122019025681B1 (pt) * | 2012-11-01 | 2023-04-18 | Factor Bioscience Inc | Método para inserir uma sequência de ácido nucleico em uma localização segura de um genoma de uma célula |
| US8697359B1 (en) * | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| JP7085716B2 (ja) * | 2013-06-05 | 2022-06-17 | デューク ユニバーシティ | Ｒｎａガイド遺伝子編集及び遺伝子調節 |
| CA2878645C (en) | 2014-01-22 | 2017-02-21 | Alfa Wassermann, Inc. | Centrifugation systems with non-contact seal assemblies |
| ES3040945T3 (en) * | 2015-10-28 | 2025-11-06 | Vertex Pharma | Materials and methods for treatment of duchenne muscular dystrophy |
| EP4244342A1 (en) * | 2020-11-12 | 2023-09-20 | Precision BioSciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
-
2015
- 2015-03-12 ES ES15761320T patent/ES2821149T3/es active Active
- 2015-03-12 AU AU2015229299A patent/AU2015229299A1/en not_active Abandoned
- 2015-03-12 ES ES20189872T patent/ES3037550T3/es active Active
- 2015-03-12 JP JP2016556801A patent/JP2017512767A/ja active Pending
- 2015-03-12 WO PCT/US2015/020205 patent/WO2015138739A2/en not_active Ceased
- 2015-03-12 CA CA2942268A patent/CA2942268A1/en active Pending
- 2015-03-12 US US15/124,984 patent/US20170106055A1/en not_active Abandoned
- 2015-03-12 EP EP15761320.9A patent/EP3116533B1/en active Active
- 2015-03-12 EP EP20189872.3A patent/EP3858376B1/en active Active
- 2015-03-12 DK DK15761320.9T patent/DK3116533T3/da active
- 2015-03-12 EP EP25191909.8A patent/EP4659765A2/en active Pending
-
2019
- 2019-02-25 US US16/284,733 patent/US20190269762A1/en not_active Abandoned
- 2019-07-03 US US16/503,396 patent/US20190365870A1/en not_active Abandoned
- 2019-08-23 JP JP2019152614A patent/JP6832995B2/ja active Active
-
2020
- 2020-09-25 US US17/033,331 patent/US20210145940A1/en not_active Abandoned
- 2020-10-26 AU AU2020260387A patent/AU2020260387A1/en not_active Abandoned
-
2021
- 2021-02-02 JP JP2021014851A patent/JP2021088563A/ja active Pending
-
2022
- 2022-09-28 JP JP2022154637A patent/JP2023002555A/ja active Pending
-
2023
- 2023-10-19 US US18/490,484 patent/US20240156919A1/en not_active Abandoned
-
2024
- 2024-04-02 AU AU2024202080A patent/AU2024202080A1/en active Pending
- 2024-09-27 JP JP2024168117A patent/JP2025013784A/ja active Pending
-
2025
- 2025-01-17 US US19/030,187 patent/US20250152679A1/en active Pending
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