IL269551A - Vectors and preparations for the treatment of hemoglobin diseases - Google Patents
Vectors and preparations for the treatment of hemoglobin diseasesInfo
- Publication number
- IL269551A IL269551A IL26955119A IL26955119A IL269551A IL 269551 A IL269551 A IL 269551A IL 26955119 A IL26955119 A IL 26955119A IL 26955119 A IL26955119 A IL 26955119A IL 269551 A IL269551 A IL 269551A
- Authority
- IL
- Israel
- Prior art keywords
- vectors
- compositions
- treating hemoglobinopathies
- hemoglobinopathies
- treating
- Prior art date
Links
- 208000034737 hemoglobinopathy Diseases 0.000 title 1
- 208000018337 inherited hemoglobinopathy Diseases 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 239000013598 vector Substances 0.000 title 1
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
- 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
-
- 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
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- A61P7/06—Antianaemics
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15045—Special targeting system for viral vectors
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15071—Demonstrated in vivo effect
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16045—Special targeting system for viral vectors
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16071—Demonstrated in vivo effect
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Hematology (AREA)
- Cell Biology (AREA)
- Developmental Biology & Embryology (AREA)
- Immunology (AREA)
- Dermatology (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762478375P | 2017-03-29 | 2017-03-29 | |
| US201762489149P | 2017-04-24 | 2017-04-24 | |
| PCT/US2018/025165 WO2018183692A1 (en) | 2017-03-29 | 2018-03-29 | Vectors and compositions for treating hemoglobinopathies |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL269551A true IL269551A (en) | 2019-11-28 |
Family
ID=63677097
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL26955119A IL269551A (en) | 2017-03-29 | 2019-09-23 | Vectors and preparations for the treatment of hemoglobin diseases |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20220259594A1 (https=) |
| EP (1) | EP3600448A4 (https=) |
| JP (2) | JP7228523B2 (https=) |
| CN (1) | CN110582305A (https=) |
| AU (1) | AU2018243293A1 (https=) |
| BR (1) | BR112019020322A2 (https=) |
| CA (1) | CA3057862A1 (https=) |
| IL (1) | IL269551A (https=) |
| SG (1) | SG11201908844YA (https=) |
| WO (1) | WO2018183692A1 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11008561B2 (en) | 2014-06-30 | 2021-05-18 | Bioverativ Therapeutics Inc. | Optimized factor IX gene |
| JP7228523B2 (ja) * | 2017-03-29 | 2023-02-24 | ブルーバード バイオ， インコーポレイテッド | ヘモグロビン異常症を治療するためのベクターおよび組成物 |
| US20220160788A1 (en) * | 2019-03-22 | 2022-05-26 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Bifunctional vectors allowing bcl11a silencing and expression of an anti-sickling hbb and uses thereof for gene therapy of b-hemoglobinopathies |
| EP3980549A4 (en) * | 2019-06-05 | 2023-07-26 | Orchard Therapeutics (Europe) Limited | COMPOSITIONS AND METHODS FOR MODIFYING EUKARYOT CELLS |
| KR20220097891A (ko) * | 2019-09-30 | 2022-07-08 | 바이오버라티브 테라퓨틱스 인크. | 렌티바이러스 벡터 제형 |
| CN117384967A (zh) * | 2020-04-24 | 2024-01-12 | 康霖生物科技（杭州）有限公司 | 一种核酸构建体 |
| US20240350664A1 (en) * | 2020-11-23 | 2024-10-24 | Intas Pharmaceuticals Ltd. | Gene therapy based administration of lentivirus vector for treating hemoglobinopathies |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8383604B2 (en) * | 2008-09-15 | 2013-02-26 | Children's Medical Center Corporation | Modulation of BCL11A for treatment of hemoglobinopathies |
| ES2769270T3 (es) * | 2011-09-30 | 2020-06-25 | Bluebird Bio Inc | Compuestos para transducción viral mejorada |
| WO2014093965A1 (en) * | 2012-12-14 | 2014-06-19 | Case Western Reserve University | Genomic rna packaging enhancer element |
| MX375592B (es) * | 2014-04-25 | 2025-03-06 | The Children´S Medical Center Corp | Composiciones y su uso para tratar hemoglobinopatias. |
| PL3134432T3 (pl) * | 2014-04-25 | 2020-10-19 | Bluebird Bio, Inc. | Promotor mnd chimeryczne receptory antygenowe |
| US20170049819A1 (en) * | 2014-04-25 | 2017-02-23 | Bluebird Bio, Inc. | Kappa/lambda chimeric antigen receptors |
| EP3620521B1 (en) | 2014-09-04 | 2024-05-29 | Memorial Sloan-Kettering Cancer Center | Globin gene therapy for treating hemoglobinopathies |
| HRP20191873T1 (hr) * | 2014-12-12 | 2020-01-24 | Bluebird Bio, Inc. | Kimerni antigenski receptori |
| LT3277807T (lt) | 2015-03-31 | 2020-01-10 | Glycotope Gmbh | Eukariotiniai ekspresijos vektoriai, apimantys globino genų klasterių reguliavimo elementus |
| WO2017040529A1 (en) | 2015-08-31 | 2017-03-09 | Bluebird Bio, Inc. | Anti-sialyl tn chimeric antigen receptors |
| JP7228523B2 (ja) * | 2017-03-29 | 2023-02-24 | ブルーバード バイオ， インコーポレイテッド | ヘモグロビン異常症を治療するためのベクターおよび組成物 |
| US11261441B2 (en) * | 2017-03-29 | 2022-03-01 | Bluebird Bio, Inc. | Vectors and compositions for treating hemoglobinopathies |
-
2018
- 2018-03-29 JP JP2019553350A patent/JP7228523B2/ja active Active
- 2018-03-29 AU AU2018243293A patent/AU2018243293A1/en not_active Abandoned
- 2018-03-29 SG SG11201908844Y patent/SG11201908844YA/en unknown
- 2018-03-29 WO PCT/US2018/025165 patent/WO2018183692A1/en not_active Ceased
- 2018-03-29 EP EP18775163.1A patent/EP3600448A4/en active Pending
- 2018-03-29 CA CA3057862A patent/CA3057862A1/en active Pending
- 2018-03-29 BR BR112019020322-2A patent/BR112019020322A2/pt not_active Application Discontinuation
- 2018-03-29 CN CN201880027694.4A patent/CN110582305A/zh active Pending
-
2019
- 2019-09-23 IL IL26955119A patent/IL269551A/en unknown
-
2022
- 2022-01-28 US US17/587,999 patent/US20220259594A1/en active Pending
-
2023
- 2023-01-18 JP JP2023005869A patent/JP2023033551A/ja not_active Withdrawn
Also Published As
| Publication number | Publication date |
|---|---|
| EP3600448A4 (en) | 2021-01-27 |
| JP2023033551A (ja) | 2023-03-10 |
| WO2018183692A8 (en) | 2019-05-02 |
| SG11201908844YA (en) | 2019-10-30 |
| AU2018243293A1 (en) | 2019-10-31 |
| CA3057862A1 (en) | 2018-10-04 |
| JP7228523B2 (ja) | 2023-02-24 |
| CN110582305A (zh) | 2019-12-17 |
| BR112019020322A2 (pt) | 2020-04-28 |
| WO2018183692A1 (en) | 2018-10-04 |
| EP3600448A1 (en) | 2020-02-05 |
| JP2020511991A (ja) | 2020-04-23 |
| US20220259594A1 (en) | 2022-08-18 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL268406A (en) | Preparations and methods for treating hemoglobin diseases | |
| IL261714A (en) | Crispr/cas-related methods and preparations for treating diseases in the hemoglobin cell | |
| IL260257A (en) | Preparations and methods for the treatment of hemoglobinopathies | |
| IL269620A (en) | Preparations and methods for the treatment of phenylketonuria | |
| IL267247B (en) | Compositions and methods for treating cancer | |
| IL266053A (en) | Preparations and methods for the treatment of ezh2-mediated cancer | |
| IL269551A (en) | Vectors and preparations for the treatment of hemoglobin diseases | |
| SI3580561T1 (sl) | Metode, osnovane na hla, in njihove sestave ter uporabe | |
| IL269637A (en) | Preparations and methods for the treatment of synovial diseases | |
| IL274837A (en) | Methods and preparations for the treatment of cancer | |
| IL271275B1 (en) | Preparations containing croons and their uses | |
| GB201807325D0 (en) | Compositions and methods | |
| IL263460A (en) | Preparations and methods for the treatment of cardiovascular diseases | |
| ZA201805490B (en) | Methods and compositions for treating hyperhidrosis | |
| IL262830A (en) | Preparations and methods for the treatment of spinal muscular atrophy | |
| IL325803A (en) | Materials and methods for cancer treatment | |
| PT3691649T (pt) | Composições e métodos para tratamento de feridas | |
| IL274524A (en) | Preparations and methods for aquaculture | |
| ZA202001880B (en) | Compositions and methods for treating brain injury | |
| ZA201908603B (en) | Hair compositions for damage treatment | |
| PL3402575T4 (pl) | Sposoby i kompozycje do traktowania zniszczonych włosów | |
| ZA202004533B (en) | Skin-brightening compositions and methods | |
| IL291266A (en) | Preparations and methods for treating infections | |
| SG10202013112YA (en) | Zinc-gamma-pga compositions and methods for treating cancer | |
| IL271923A (en) | Methods and compositions for treating addictions |