EA201691891A1 - Генная терапия для лечения пигментного ретинита - Google Patents
Генная терапия для лечения пигментного ретинитаInfo
- Publication number
- EA201691891A1 EA201691891A1 EA201691891A EA201691891A EA201691891A1 EA 201691891 A1 EA201691891 A1 EA 201691891A1 EA 201691891 A EA201691891 A EA 201691891A EA 201691891 A EA201691891 A EA 201691891A EA 201691891 A1 EA201691891 A1 EA 201691891A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- treatment
- gene therapy
- pigment retinitis
- retinitis
- pigment
- Prior art date
Links
- 206010038910 Retinitis Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 239000000049 pigment Substances 0.000 title 1
- 239000002245 particle Substances 0.000 abstract 3
- 230000003612 virological effect Effects 0.000 abstract 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 208000007014 Retinitis pigmentosa Diseases 0.000 abstract 1
- 210000000234 capsid Anatomy 0.000 abstract 1
- 238000002347 injection Methods 0.000 abstract 1
- 239000007924 injection Substances 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 108091089534 miR-708 stem-loop Proteins 0.000 abstract 1
Classifications
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- 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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- 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
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- 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
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- 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
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- 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
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- 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
- A61K9/0048—Eye, e.g. artificial tears
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
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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
- C12N15/63—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
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
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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
- 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
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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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Ophthalmology & Optometry (AREA)
- Virology (AREA)
- Immunology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Marine Sciences & Fisheries (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Dermatology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
В данном документе предусматриваются способы лечения пигментного ретинита с использованием частиц AAV, кодирующих miR-708. В одном из аспектов вирусные частицы вводят в глаз человека, например, с помощью субретинальной инъекции. Рассматриваются вирусные частицы, содержащие капсиды AAV5 или их мутанты.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201461969027P | 2014-03-21 | 2014-03-21 | |
PCT/US2015/021896 WO2015143418A2 (en) | 2014-03-21 | 2015-03-20 | Gene therapy for retinitis pigmentosa |
Publications (1)
Publication Number | Publication Date |
---|---|
EA201691891A1 true EA201691891A1 (ru) | 2017-01-30 |
Family
ID=52829343
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EA201691891A EA201691891A1 (ru) | 2014-03-21 | 2015-03-20 | Генная терапия для лечения пигментного ретинита |
Country Status (36)
Country | Link |
---|---|
US (3) | US10383953B2 (ru) |
EP (3) | EP3119437B1 (ru) |
JP (3) | JP6669664B2 (ru) |
KR (2) | KR20160127832A (ru) |
CN (2) | CN106456660B (ru) |
AR (1) | AR099837A1 (ru) |
AU (1) | AU2015230942B2 (ru) |
CA (1) | CA2943185A1 (ru) |
CL (1) | CL2016002333A1 (ru) |
CR (1) | CR20160480A (ru) |
DK (2) | DK3119437T3 (ru) |
DO (1) | DOP2016000237A (ru) |
EA (1) | EA201691891A1 (ru) |
EC (1) | ECSP16083000A (ru) |
ES (2) | ES2957840T3 (ru) |
FI (1) | FI3628334T3 (ru) |
HR (2) | HRP20231077T1 (ru) |
HU (2) | HUE046454T2 (ru) |
IL (3) | IL247543B (ru) |
LT (2) | LT3119437T (ru) |
MA (1) | MA39390B2 (ru) |
MX (2) | MX2016012201A (ru) |
MY (1) | MY190726A (ru) |
NZ (1) | NZ724622A (ru) |
PE (1) | PE20161252A1 (ru) |
PH (1) | PH12016501684A1 (ru) |
PL (2) | PL3628334T3 (ru) |
PT (2) | PT3628334T (ru) |
RS (2) | RS59634B1 (ru) |
SG (3) | SG11201607005UA (ru) |
SI (2) | SI3119437T1 (ru) |
TW (2) | TWI780401B (ru) |
UA (1) | UA120050C2 (ru) |
UY (1) | UY36044A (ru) |
WO (1) | WO2015143418A2 (ru) |
ZA (1) | ZA201605924B (ru) |
Families Citing this family (18)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
UA120050C2 (uk) * | 2014-03-21 | 2019-09-25 | Джензайм Корпорейшн | Генна терапія для лікування пігментного ретиніту |
EP3256588A2 (en) | 2015-02-10 | 2017-12-20 | Genzyme Corporation | VARIANT RNAi |
CN108474001B (zh) * | 2015-12-03 | 2022-04-08 | 弗里德里克·米谢尔生物医学研究所 | SynP160,用于基因在视杆光感受器中特异性表达的启动子 |
CN108472390B (zh) | 2015-12-03 | 2022-04-15 | 弗里德里克·米谢尔生物医学研究所 | SynP162,用于基因在视杆光感受器中特异性表达的启动子 |
JP6966442B2 (ja) * | 2015-12-04 | 2021-11-17 | ソルボンヌ・ユニヴェルシテSorbonne Universite | プロモーター及びその使用 |
WO2017151823A1 (en) * | 2016-03-01 | 2017-09-08 | University Of Florida Research Foundation, Inc. | Aav vectors for treatment of dominant retinitis pigmentosa |
CN109890424A (zh) * | 2016-07-05 | 2019-06-14 | 约翰霍普金斯大学 | 用于治疗视网膜变性的基于crispr/cas9的组合物和方法 |
US20200030458A1 (en) * | 2017-03-21 | 2020-01-30 | Stylianos Michalakis | Gene Therapy for the Treatment of CNGB1-linked Retinitis Pigmentosa |
CA3076191A1 (en) * | 2017-09-22 | 2019-03-28 | Genzyme Corporation | Variant rnai |
MX2020003351A (es) * | 2017-09-27 | 2020-10-12 | Sigilon Therapeutics Inc | Metodos, composiciones y elementos implantables que comprenden celulas activas. |
CN107961380B (zh) * | 2017-11-23 | 2020-05-05 | 清华大学 | 试剂在制备药物中的用途、筛选药物的方法以及药物组合物 |
EP3768327A4 (en) * | 2018-03-23 | 2022-04-13 | The Trustees of Columbia University in the City of New York | GENE EDIT FOR AUTOSOMAL DOMINANT DISEASES |
WO2019232517A1 (en) * | 2018-06-01 | 2019-12-05 | University Of Florida Research Foundation, Incorporated | Compositions and methods for treatment of dominant retinitis pigmentosa |
CN111518813B (zh) * | 2019-02-03 | 2023-04-28 | 武汉纽福斯生物科技有限公司 | 视紫红质的编码序列、其表达载体构建及其应用 |
SG11202109554XA (en) * | 2019-03-04 | 2021-09-29 | Univ Pennsylvania | Neuroprotective gene therapy targeting the akt pathway |
BR112021018354A2 (pt) * | 2019-03-21 | 2021-11-23 | Ptc Therapeutics Inc | Vetor e método para tratar síndrome de angelman |
KR102396200B1 (ko) * | 2020-07-24 | 2022-05-12 | 알지노믹스 주식회사 | 로돕신 전사체에 특이적인 트랜스-스플라이싱 라이보자임 및 이의 용도 |
TWI832531B (zh) * | 2022-11-02 | 2024-02-11 | 慈濟學校財團法人慈濟大學 | Rip1抑制劑或mlkl抑制劑用於治療或預防遺傳性視網膜失養症的用途 |
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US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
US6995006B2 (en) | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
JP4860886B2 (ja) | 2000-06-01 | 2012-01-25 | ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 二本鎖パルボウイルスベクター |
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EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
US8257969B2 (en) | 2007-04-12 | 2012-09-04 | The Provost Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
US20090214478A1 (en) * | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
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JP5884152B2 (ja) | 2011-07-29 | 2016-03-15 | シャープ株式会社 | 基地局、端末、通信システムおよび通信方法 |
ITRM20110685A1 (it) * | 2011-12-23 | 2013-06-24 | Internat Ct For Genetic En Gineering And | Microrna per la rigenerazione cardiaca attraverso l induzione della proliferazione dei miociti cardiaci |
AU2013221212B2 (en) * | 2012-02-17 | 2018-08-09 | The Children's Hospital Of Philadelphia | AAV vector compositions and methods for gene transfer to cells, organs and tissues |
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GEP20217251B (en) | 2012-05-25 | 2021-04-26 | Charpentier Emmanuelle De Emanuel | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
CN105120901A (zh) * | 2012-07-11 | 2015-12-02 | 宾夕法尼亚大学托管会 | Rpgr x染色体连锁视网膜退化的aav介导的基因治疗 |
WO2014066498A1 (en) * | 2012-10-23 | 2014-05-01 | Vivek Mittal | Treatment of metastatic breast cancer |
TW201605470A (zh) | 2013-10-04 | 2016-02-16 | 默沙東藥廠 | 葡萄糖反應性胰島素結合物 |
UA120050C2 (uk) | 2014-03-21 | 2019-09-25 | Джензайм Корпорейшн | Генна терапія для лікування пігментного ретиніту |
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2015
- 2015-03-20 UA UAA201610589A patent/UA120050C2/uk unknown
- 2015-03-20 KR KR1020167028893A patent/KR20160127832A/ko not_active Application Discontinuation
- 2015-03-20 US US15/127,757 patent/US10383953B2/en active Active
- 2015-03-20 SG SG11201607005UA patent/SG11201607005UA/en unknown
- 2015-03-20 HR HRP20231077TT patent/HRP20231077T1/hr unknown
- 2015-03-20 PE PE2016001643A patent/PE20161252A1/es unknown
- 2015-03-20 EP EP15716232.2A patent/EP3119437B1/en active Active
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