CA3031785A1 - Bcl11a homing endonuclease variants, compositions, and methods of use - Google Patents
Bcl11a homing endonuclease variants, compositions, and methods of use Download PDFInfo
- Publication number
- CA3031785A1 CA3031785A1 CA3031785A CA3031785A CA3031785A1 CA 3031785 A1 CA3031785 A1 CA 3031785A1 CA 3031785 A CA3031785 A CA 3031785A CA 3031785 A CA3031785 A CA 3031785A CA 3031785 A1 CA3031785 A1 CA 3031785A1
- Authority
- CA
- Canada
- Prior art keywords
- beta
- polypeptide
- amino acid
- seq
- cell
- 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.)
- Abandoned
Links
Classifications
-
- 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]
-
- 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
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- 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
- 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
-
- 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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
-
- 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
-
- 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)
-
- 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)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- General Engineering & Computer Science (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Manufacturing & Machinery (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662366530P | 2016-07-25 | 2016-07-25 | |
| US62/366,530 | 2016-07-25 | ||
| US201662367465P | 2016-07-27 | 2016-07-27 | |
| US62/367,465 | 2016-07-27 | ||
| US201662375829P | 2016-08-16 | 2016-08-16 | |
| US62/375,829 | 2016-08-16 | ||
| US201662414273P | 2016-10-28 | 2016-10-28 | |
| US62/414,273 | 2016-10-28 | ||
| PCT/US2017/043726 WO2018022619A1 (en) | 2016-07-25 | 2017-07-25 | Bcl11a homing endonuclease variants, compositions, and methods of use |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3031785A1 true CA3031785A1 (en) | 2018-02-01 |
Family
ID=61017227
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3031785A Abandoned CA3031785A1 (en) | 2016-07-25 | 2017-07-25 | Bcl11a homing endonuclease variants, compositions, and methods of use |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20190184035A1 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3487994A4 (cg-RX-API-DMAC7.html) |
| JP (1) | JP2019525759A (cg-RX-API-DMAC7.html) |
| CN (1) | CN109689865A (cg-RX-API-DMAC7.html) |
| AU (1) | AU2017301609A1 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3031785A1 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2018022619A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10967298B2 (en) | 2012-03-15 | 2021-04-06 | Flodesign Sonics, Inc. | Driver and control for variable impedence load |
| US9950282B2 (en) | 2012-03-15 | 2018-04-24 | Flodesign Sonics, Inc. | Electronic configuration and control for acoustic standing wave generation |
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| US9458450B2 (en) | 2012-03-15 | 2016-10-04 | Flodesign Sonics, Inc. | Acoustophoretic separation technology using multi-dimensional standing waves |
| EP3092049A1 (en) | 2014-01-08 | 2016-11-16 | Flodesign Sonics Inc. | Acoustophoresis device with dual acoustophoretic chamber |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| US11021699B2 (en) | 2015-04-29 | 2021-06-01 | FioDesign Sonics, Inc. | Separation using angled acoustic waves |
| US11459540B2 (en) | 2015-07-28 | 2022-10-04 | Flodesign Sonics, Inc. | Expanded bed affinity selection |
| US11474085B2 (en) | 2015-07-28 | 2022-10-18 | Flodesign Sonics, Inc. | Expanded bed affinity selection |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| US11085035B2 (en) | 2016-05-03 | 2021-08-10 | Flodesign Sonics, Inc. | Therapeutic cell washing, concentration, and separation utilizing acoustophoresis |
| CA3035455A1 (en) | 2016-09-08 | 2018-03-15 | Bluebird Bio, Inc. | Pd-1 homing endonuclease variants, compositions, and methods of use |
| AU2017346683B2 (en) | 2016-10-17 | 2024-04-18 | Regeneron Pharmaceuticals, Inc. | TGFβR2 endonuclease variants, compositions, and methods of use |
| KR20190127655A (ko) | 2016-10-19 | 2019-11-13 | 프로디자인 소닉스, 인크. | 음향학에 의한 친화성 세포 추출 |
| TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| JP7191042B2 (ja) | 2017-05-25 | 2022-12-16 | ２セブンティ バイオ インコーポレイテッド | Ｃｂｌｂエンドヌクレアーゼバリアント、組成物、および使用方法 |
| JP2021507561A (ja) | 2017-12-14 | 2021-02-22 | フロデザイン ソニックス， インク．Ｆｌｏｄｅｓｉｇｎ Ｓｏｎｉｃｓ， Ｉｎｃ． | 音響トランスデューサドライバ及びコントローラ |
| EP3784790A4 (en) * | 2018-04-27 | 2022-02-09 | Seattle Children's Hospital d/b/a Seattle Children's Research Institute | BRUTON TYROSINE KINASE HOMING ENDONUCLEAR VARIANTS, COMPOSITIONS AND METHOD OF USE |
| WO2020072059A1 (en) | 2018-10-04 | 2020-04-09 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
| US12404500B2 (en) | 2018-12-10 | 2025-09-02 | Novo Nordisk A/S | Homing endonuclease variants |
| CA3137896A1 (en) * | 2019-04-24 | 2020-10-29 | Seattle Children's Hospital D/B/A Seattle Children's Research Institute | Wiskott-aldrich syndrome gene homing endonuclease variants, compositions, and methods of use |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025153530A1 (en) * | 2024-01-16 | 2025-07-24 | Novo Nordisk A/S | Albumin-targeted endonucleases, compositions, and methods of use |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009095742A1 (en) * | 2008-01-31 | 2009-08-06 | Cellectis | New i-crei derived single-chain meganuclease and uses thereof |
| EP2660317B1 (en) * | 2007-10-31 | 2016-04-06 | Precision Biosciences, Inc. | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| US20140148361A1 (en) * | 2010-06-07 | 2014-05-29 | Barry L. Stoddard | Generation and Expression of Engineered I-ONUI Endonuclease and Its Homologues and Uses Thereof |
| RU2018109507A (ru) * | 2012-02-24 | 2019-02-28 | Фред Хатчинсон Кэнсер Рисерч Сентер | Композиции и способы лечения гемоглобинопатии |
| AU2014273089B2 (en) * | 2013-05-31 | 2018-02-22 | Cellectis | A LAGLIDADG homing endonuclease cleaving the C-C Chemokine Receptor Type-5 (CCR5) gene and uses thereof |
| AU2014273091B2 (en) * | 2013-05-31 | 2019-12-12 | Cellectis | A LAGLIDADG homing endonuclease cleaving the T cell receptor alpha gene and uses thereof |
-
2017
- 2017-07-25 AU AU2017301609A patent/AU2017301609A1/en not_active Abandoned
- 2017-07-25 US US16/320,280 patent/US20190184035A1/en not_active Abandoned
- 2017-07-25 JP JP2019503701A patent/JP2019525759A/ja active Pending
- 2017-07-25 EP EP17835117.7A patent/EP3487994A4/en not_active Withdrawn
- 2017-07-25 WO PCT/US2017/043726 patent/WO2018022619A1/en not_active Ceased
- 2017-07-25 CA CA3031785A patent/CA3031785A1/en not_active Abandoned
- 2017-07-25 CN CN201780054710.4A patent/CN109689865A/zh active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CN109689865A (zh) | 2019-04-26 |
| US20190184035A1 (en) | 2019-06-20 |
| AU2017301609A1 (en) | 2019-02-21 |
| JP2019525759A (ja) | 2019-09-12 |
| EP3487994A1 (en) | 2019-05-29 |
| WO2018022619A1 (en) | 2018-02-01 |
| EP3487994A4 (en) | 2020-01-29 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FZDE | Discontinued |
Effective date: 20230126 |
|
| FZDE | Discontinued |
Effective date: 20230126 |