DK3289080T3 - Genterapi til autosomalt dominante sygdomme - Google Patents
Genterapi til autosomalt dominante sygdomme Download PDFInfo
- Publication number
- DK3289080T3 DK3289080T3 DK16787314.0T DK16787314T DK3289080T3 DK 3289080 T3 DK3289080 T3 DK 3289080T3 DK 16787314 T DK16787314 T DK 16787314T DK 3289080 T3 DK3289080 T3 DK 3289080T3
- Authority
- DK
- Denmark
- Prior art keywords
- autosomal dominant
- dominant diseases
- generation therapy
- therapy
- generation
- Prior art date
Links
Classifications
-
- 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
-
- 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
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- 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
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- 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
-
- 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
-
- 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
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
-
- 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
- C12N2320/34—Allele or polymorphism specific uses
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Ophthalmology & Optometry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Dermatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562154966P | 2015-04-30 | 2015-04-30 | |
PCT/US2016/030475 WO2016176690A2 (en) | 2015-04-30 | 2016-05-02 | Gene therapy for autosomal dominant diseases |
Publications (1)
Publication Number | Publication Date |
---|---|
DK3289080T3 true DK3289080T3 (da) | 2021-11-08 |
Family
ID=57198841
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DK16787314.0T DK3289080T3 (da) | 2015-04-30 | 2016-05-02 | Genterapi til autosomalt dominante sygdomme |
Country Status (7)
Country | Link |
---|---|
US (1) | US20190275168A1 (da) |
EP (2) | EP3289080B1 (da) |
DK (1) | DK3289080T3 (da) |
ES (1) | ES2898917T3 (da) |
HK (1) | HK1251615A1 (da) |
PT (1) | PT3289080T (da) |
WO (1) | WO2016176690A2 (da) |
Families Citing this family (48)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP2734621B1 (en) | 2011-07-22 | 2019-09-04 | President and Fellows of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
US9163284B2 (en) | 2013-08-09 | 2015-10-20 | 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 |
US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
PL3221457T3 (pl) | 2014-11-21 | 2019-09-30 | Regeneron Pharmaceuticals, Inc. | Sposoby i kompozycje do celowanej modyfikacji genetycznej z zastosowaniem sparowanych przewodników RNA |
JP7067793B2 (ja) | 2015-10-23 | 2022-05-16 | プレジデント アンド フェローズ オブ ハーバード カレッジ | 核酸塩基編集因子およびその使用 |
CA3014671A1 (en) | 2016-03-01 | 2017-09-08 | University Of Florida Research Foundation, Inc. | Aav vectors for treatment of dominant retinitis pigmentosa |
US20200080108A1 (en) * | 2016-07-05 | 2020-03-12 | The Johns Hopkins University | Crispr/cas9-based compositions and methods for treating retinal degenerations |
JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | アデノシン核酸塩基編集因子およびそれらの使用 |
AU2017308889B2 (en) | 2016-08-09 | 2023-11-09 | President And Fellows Of Harvard College | Programmable Cas9-recombinase fusion proteins and uses thereof |
US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
IT201600102542A1 (it) * | 2016-10-12 | 2018-04-12 | Univ Degli Studi Di Trento | Plasmide e sistema lentivirale contenente un circuito autolimitante della Cas9 che ne incrementa la sicurezza. |
KR20240007715A (ko) | 2016-10-14 | 2024-01-16 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
WO2018085644A1 (en) * | 2016-11-03 | 2018-05-11 | Youhealth Biotech, Limited | Methods and compositions for cellular reprogramming |
WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
EP4317447A3 (en) * | 2017-02-15 | 2024-05-01 | 2seventy bio, Inc. | Donor repair templates multiplex genome editing |
US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
EP3592777A1 (en) | 2017-03-10 | 2020-01-15 | President and Fellows of Harvard College | Cytosine to guanine base editor |
KR20190130613A (ko) | 2017-03-23 | 2019-11-22 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
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 |
EP3634451A4 (en) * | 2017-05-15 | 2021-03-24 | The Trustees of Columbia University in the City of New York | REPROGRAMMATION OF METABOLISM BY VHL INHIBITION FOR THE TREATMENT OF NEURODEGENERENCE |
JP2020534795A (ja) | 2017-07-28 | 2020-12-03 | プレジデント アンド フェローズ オブ ハーバード カレッジ | ファージによって支援される連続的進化(pace)を用いて塩基編集因子を進化させるための方法および組成物 |
EP3661523A4 (en) * | 2017-08-03 | 2021-08-04 | University of Iowa Research Foundation | TREATMENT METHODS FOR GENETIC HEARING LOSS |
CA3074466A1 (en) * | 2017-08-28 | 2019-03-07 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating cone-rod retinal dystrophy |
WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
WO2019066549A2 (ko) * | 2017-09-29 | 2019-04-04 | 주식회사 툴젠 | 망막 기능장애 질환 치료를 위한 유전자 조작 |
WO2019079347A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | USES OF BASIC EDITORS ADENOSINE |
CN111886340A (zh) * | 2017-11-15 | 2020-11-03 | 密歇根大学董事会 | 包括rdh12编码区的病毒载体和治疗视网膜营养不良的方法 |
WO2019102381A1 (en) | 2017-11-21 | 2019-05-31 | Casebia Therapeutics Llp | Materials and methods for treatment of autosomal dominant retinitis pigmentosa |
CN108034709B (zh) * | 2017-12-18 | 2021-05-07 | 青岛大学 | Guca1a基因在制备检测视锥细胞营养不良制品中的应用 |
JP2021519067A (ja) * | 2018-03-23 | 2021-08-10 | ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク | 常染色体優性疾患のための遺伝子編集 |
EP3784292A4 (en) * | 2018-04-27 | 2022-01-19 | Seattle Children's Hospital (DBA Seattle Children's Research Institute) | THERAPEUTIC GENOME EDITING IN HYPER-IGM SEX-LINKED SYNDROME |
US20210254012A1 (en) * | 2018-06-09 | 2021-08-19 | Arizona Board Of Regents On Behalf Of Arizona State University | Next generation designer liver organoids and their methods of preparation and use |
CN110857440B (zh) * | 2018-08-23 | 2021-02-19 | 武汉纽福斯生物科技有限公司 | 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用 |
CN113348247A (zh) * | 2018-10-15 | 2021-09-03 | 泰莱托恩基金会 | 基因组编辑方法与构建体 |
CN109456995A (zh) * | 2018-11-08 | 2019-03-12 | 杜以军 | 基因敲除质粒、细胞系及制备方法和应用 |
CN113631710A (zh) * | 2019-02-25 | 2021-11-09 | 爱迪塔斯医药公司 | 用于治疗rho相关的常染色体显性视网膜色素变性(adrp)的crispr/rna指导的核酸酶相关方法和组合物 |
EP3942040A1 (en) | 2019-03-19 | 2022-01-26 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
EP3997227A1 (en) * | 2019-07-10 | 2022-05-18 | Locanabio, Inc. | Rna-targeting knockdown and replacement compositions and methods for use |
CN110923333B (zh) * | 2019-12-11 | 2020-09-29 | 湖北省农业科学院畜牧兽医研究所 | 山羊zbp1基因第一内含子内与产羔数关联的单倍型标记及其应用 |
IL295673A (en) * | 2020-02-28 | 2022-10-01 | Univ Pennsylvania | Treatment of autosomal dominant strophin diseases and methods of their evaluation |
CN111484981B (zh) * | 2020-04-26 | 2021-02-02 | 四川省人民医院 | 一种用于构建视网膜新生血管疾病模型的方法和应用 |
WO2021226558A1 (en) | 2020-05-08 | 2021-11-11 | The Broad Institute, Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
WO2022221741A1 (en) * | 2021-04-16 | 2022-10-20 | Editas Medicine, Inc. | Crispr/rna-guided nuclease-related methods and compositions for treating rho-associated autosomal-dominant retinitis pigmentosa (adrp) |
Family Cites Families (8)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease |
DE69432628T8 (de) | 1993-02-12 | 2004-07-08 | The Johns-Hopkins University | FUNKTIONELLE DOMÄNEN DER RESTRIKTIONSENDONUKLEASEN AUS i-(FLAVOBAKTERIUM OKEANOKOITES)(FOKI) |
US8551970B2 (en) * | 1996-04-02 | 2013-10-08 | Optigen Patents Limited | Genetic suppression and replacement |
US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
ES2527997T5 (es) | 2009-12-10 | 2018-05-17 | Regents Of The University Of Minnesota | Modificación del ADN inducida por el efector TAL |
GB201004475D0 (en) * | 2010-03-17 | 2010-05-05 | Isis Innovation | Gene silencing |
DK3011031T3 (da) * | 2013-06-17 | 2020-12-21 | Broad Inst Inc | Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi |
KR20230122182A (ko) * | 2013-07-09 | 2023-08-22 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 멀티플렉스 rna-가이드된 게놈 조작 |
-
2016
- 2016-05-02 EP EP16787314.0A patent/EP3289080B1/en active Active
- 2016-05-02 ES ES16787314T patent/ES2898917T3/es active Active
- 2016-05-02 US US15/569,920 patent/US20190275168A1/en active Pending
- 2016-05-02 DK DK16787314.0T patent/DK3289080T3/da active
- 2016-05-02 PT PT167873140T patent/PT3289080T/pt unknown
- 2016-05-02 EP EP21186523.3A patent/EP4008780A1/en active Pending
- 2016-05-02 WO PCT/US2016/030475 patent/WO2016176690A2/en unknown
-
2018
- 2018-08-30 HK HK18111142.4A patent/HK1251615A1/zh unknown
Also Published As
Publication number | Publication date |
---|---|
US20190275168A1 (en) | 2019-09-12 |
ES2898917T3 (es) | 2022-03-09 |
EP3289080B1 (en) | 2021-08-25 |
HK1251615A1 (zh) | 2019-02-01 |
EP3289080A4 (en) | 2018-10-03 |
PT3289080T (pt) | 2021-11-19 |
WO2016176690A3 (en) | 2016-12-01 |
WO2016176690A2 (en) | 2016-11-03 |
EP3289080A2 (en) | 2018-03-07 |
EP4008780A1 (en) | 2022-06-08 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
DK3289080T3 (da) | Genterapi til autosomalt dominante sygdomme | |
DK3328880T3 (da) | Terapeutiske midler | |
DK3211071T3 (da) | Fremstillingsfremgangsmåde til retinalt væv | |
DK3356313T3 (da) | 2-perfluorhexyloktan til oftalmisk indgivelse | |
DK3107563T3 (da) | Glycomålrettede terapeutiske midler | |
DK3253382T3 (da) | Farmaceutiske sammensætninger til kombinationsterapi | |
DK3072835T3 (da) | Fremgangsmåde til fremføring | |
DK3262066T4 (da) | Genterapi | |
BR112016021682A2 (pt) | cenicriviroc para o tratamento da fibrose | |
MA39483A (fr) | Agents thérapeutiques cibles | |
BR112018005409A2 (pt) | terapêutica de glicodirecionamento. | |
DK3265087T3 (da) | Fremgangsmåde til behandling med tradipitant | |
DK3310432T3 (da) | Indretning til effektiv ikke-invasiv to-trinsneurostimulation | |
DK3393478T3 (da) | Kombinationsterapi | |
DK3386997T3 (da) | Monomaleimid-funktionaliserede platinforbindelser til cancerterapi | |
DK3408265T3 (da) | Terapeutiske forbindelser | |
DK3294769T3 (da) | Behandling for multipelt myelom (mm) | |
DK3329004T3 (da) | Terapeutiske oligonukleotider | |
DK3261679T3 (da) | Genterapi til synsforbedring | |
FI20145996A (fi) | Pilarikenkä | |
ITUB20156862A1 (it) | Composizioni per il trattamento della psoriasi. | |
TWD174592S (zh) | 按摩器 | |
TWD174591S (zh) | 按摩器 | |
ES1144311Y (es) | Calzado con propiedades terapeuticas | |
FR3036281B1 (fr) | Fauteuil roulant |