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
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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
-
- 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)
- Zoology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Cell Biology (AREA)
- Immunology (AREA)
- Hematology (AREA)
- Developmental Biology & Embryology (AREA)
- Dermatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (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 (enExample) |
| EP (1) | EP3600448A4 (enExample) |
| JP (2) | JP7228523B2 (enExample) |
| CN (1) | CN110582305A (enExample) |
| AU (1) | AU2018243293A1 (enExample) |
| BR (1) | BR112019020322A2 (enExample) |
| CA (1) | CA3057862A1 (enExample) |
| IL (1) | IL269551A (enExample) |
| SG (1) | SG11201908844YA (enExample) |
| WO (1) | WO2018183692A1 (enExample) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3160478A4 (en) | 2014-06-30 | 2018-05-16 | 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 |
| JP2022534798A (ja) * | 2019-06-05 | 2022-08-03 | オーチャード セラピューティクス（ヨーロッパ）リミテッド | 真核細胞を修飾するための組成物及び方法 |
| CN115279896A (zh) * | 2019-09-30 | 2022-11-01 | 比奥维拉迪维治疗股份有限公司 | 慢病毒载体配制品 |
| 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 |
| EP4222271A4 (en) * | 2020-11-23 | 2025-07-30 | Intas Pharmaceuticals Ltd | LENTIVIRUS VECTOR GENE THERAPY-BASED DELIVERY FOR THE TREATMENT OF HEMOGLOBINOPATHIES |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2334794T3 (en) * | 2008-09-15 | 2017-02-20 | Children's Medical Center Corp | MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES |
| UA114796C2 (uk) * | 2011-09-30 | 2017-08-10 | Блубьод Байо, Інк. | Спосіб підвищення ефективності трансдукції |
| WO2014093965A1 (en) * | 2012-12-14 | 2014-06-19 | Case Western Reserve University | Genomic rna packaging enhancer element |
| EP3134434A4 (en) * | 2014-04-25 | 2017-10-25 | Bluebird Bio, Inc. | Kappa/lambda chimeric antigen receptors |
| JP6514717B2 (ja) * | 2014-04-25 | 2019-05-15 | ザ チルドレンズ メディカル センター コーポレーション | 異常ヘモグロビン症を治療するための組成物および方法 |
| EP3134432B1 (en) * | 2014-04-25 | 2019-12-25 | Bluebird Bio, Inc. | Mnd promoter chimeric antigen receptors |
| ES2746529T3 (es) * | 2014-09-04 | 2020-03-06 | Memorial Sloan Kettering Cancer Center | Terapia génica de globina para tratar hemoglobinopatías |
| LT3230321T (lt) * | 2014-12-12 | 2019-12-10 | Bluebird Bio Inc | Bcma chimeriniai antigeno receptoriai |
| LT3277807T (lt) * | 2015-03-31 | 2020-01-10 | Glycotope Gmbh | Eukariotiniai ekspresijos vektoriai, apimantys globino genų klasterių reguliavimo elementus |
| ES2911015T3 (es) * | 2015-08-31 | 2022-05-17 | Helixmith Co Ltd | Receptores quiméricos de antígeno anti-sialil Tn |
| US11261441B2 (en) * | 2017-03-29 | 2022-03-01 | Bluebird Bio, Inc. | Vectors and compositions for treating hemoglobinopathies |
| 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 |
-
2018
- 2018-03-29 JP JP2019553350A patent/JP7228523B2/ja active Active
- 2018-03-29 BR BR112019020322-2A patent/BR112019020322A2/pt not_active Application Discontinuation
- 2018-03-29 CA CA3057862A patent/CA3057862A1/en active Pending
- 2018-03-29 AU AU2018243293A patent/AU2018243293A1/en not_active Abandoned
- 2018-03-29 WO PCT/US2018/025165 patent/WO2018183692A1/en not_active Ceased
- 2018-03-29 SG SG11201908844Y patent/SG11201908844YA/en unknown
- 2018-03-29 CN CN201880027694.4A patent/CN110582305A/zh active Pending
- 2018-03-29 EP EP18775163.1A patent/EP3600448A4/en 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 |
|---|---|
| WO2018183692A1 (en) | 2018-10-04 |
| BR112019020322A2 (pt) | 2020-04-28 |
| AU2018243293A1 (en) | 2019-10-31 |
| SG11201908844YA (en) | 2019-10-30 |
| EP3600448A4 (en) | 2021-01-27 |
| US20220259594A1 (en) | 2022-08-18 |
| JP7228523B2 (ja) | 2023-02-24 |
| CA3057862A1 (en) | 2018-10-04 |
| EP3600448A1 (en) | 2020-02-05 |
| JP2023033551A (ja) | 2023-03-10 |
| WO2018183692A8 (en) | 2019-05-02 |
| CN110582305A (zh) | 2019-12-17 |
| JP2020511991A (ja) | 2020-04-23 |
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