[go: up one dir, main page]

MX2017010369A - Arni variante. - Google Patents

Arni variante.

Info

Publication number
MX2017010369A
MX2017010369A MX2017010369A MX2017010369A MX2017010369A MX 2017010369 A MX2017010369 A MX 2017010369A MX 2017010369 A MX2017010369 A MX 2017010369A MX 2017010369 A MX2017010369 A MX 2017010369A MX 2017010369 A MX2017010369 A MX 2017010369A
Authority
MX
Mexico
Prior art keywords
rnai
guide sequence
recombinant
disease
further provided
Prior art date
Application number
MX2017010369A
Other languages
English (en)
Inventor
Pablo Sardi Sergio
O´Riordan Catherine
M Stanek Lisa
Palermo Adam
Richards Brenda
Song Antonius
Original Assignee
Genzyme Corp
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of MX2017010369A publication Critical patent/MX2017010369A/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/08Antiepileptics; Anticonvulsants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/533Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10022New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10041Use of virus, viral particle or viral elements as a vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10041Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Vascular Medicine (AREA)
  • Pain & Pain Management (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Hospice & Palliative Care (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)

Abstract

En la presente descripción se proporcionan moléculas de ARNi que incluyen una primera cadena que contiene una secuencia guía y una segunda cadena que comprende una secuencia no guía en donde la secuencia no guía contiene una protuberancia opuesta a la región semilla de las secuencias guía; p. ej., opuesta a la secuencia de escisión. En algunos aspectos, la invención proporciona ARNi para el tratamiento de la enfermedad de Huntington. Adicionalmente se proporcionan en la presente descripción casetes de expresión, vectores (p. ej., vectores de rAAV, adenovirales recombinantes, lentivirales recombinantes, y de HSV recombinantes), células, partículas virales y composiciones farmacéuticas que contienen el ARNi. Asimismo se proporcionan adicionalmente en la presente descripción métodos y kits relacionados con el uso del ARNi, por ejemplo, para el tratamiento de la enfermedad de Huntington.
MX2017010369A 2015-02-10 2016-02-09 Arni variante. MX2017010369A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562114578P 2015-02-10 2015-02-10
PCT/US2016/017207 WO2016130589A2 (en) 2015-02-10 2016-02-09 VARIANT RNAi

Publications (1)

Publication Number Publication Date
MX2017010369A true MX2017010369A (es) 2017-12-14

Family

ID=56614905

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017010369A MX2017010369A (es) 2015-02-10 2016-02-09 Arni variante.
MX2022013005A MX2022013005A (es) 2015-02-10 2017-08-10 Arni variante.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2022013005A MX2022013005A (es) 2015-02-10 2017-08-10 Arni variante.

Country Status (25)

Country Link
US (4) US10450563B2 (es)
EP (1) EP3256588A2 (es)
JP (2) JP2018506304A (es)
KR (1) KR102670852B1 (es)
CN (1) CN107438671B (es)
AR (1) AR103646A1 (es)
AU (3) AU2016219396B2 (es)
CA (1) CA2976075A1 (es)
CL (1) CL2017002027A1 (es)
CO (1) CO2017009083A2 (es)
CR (1) CR20170406A (es)
EA (1) EA201791805A1 (es)
EC (1) ECSP17059343A (es)
HK (1) HK1247641A1 (es)
IL (1) IL253893B (es)
MA (1) MA40819B1 (es)
MX (2) MX2017010369A (es)
MY (2) MY194175A (es)
PE (1) PE20171382A1 (es)
PH (1) PH12017501432A1 (es)
SG (3) SG11201706444TA (es)
TN (1) TN2017000354A1 (es)
TW (1) TWI781079B (es)
UY (1) UY36554A (es)
WO (1) WO2016130589A2 (es)

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2616051T3 (es) 2008-12-02 2017-06-09 Wave Life Sciences Japan, Inc. Método para la síntesis de ácidos nucleicos modificados en el átomo de fósforo
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
RU2014105311A (ru) 2011-07-19 2015-08-27 Уэйв Лайф Сайенсес Пте. Лтд. Способы синтеза функционализованных нуклеиновых кислот
CA2879066C (en) 2012-07-13 2019-08-13 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant
EP3812370A1 (en) 2012-07-13 2021-04-28 Wave Life Sciences Ltd. Asymmetric auxiliary group
US9982257B2 (en) 2012-07-13 2018-05-29 Wave Life Sciences Ltd. Chiral control
US10322173B2 (en) 2014-01-15 2019-06-18 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
RU2016133035A (ru) 2014-01-16 2018-02-21 Уэйв Лайф Сайенсес Лтд. Хиральный дизайн
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
RU2020108189A (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
KR102670852B1 (ko) 2015-02-10 2024-05-29 젠자임 코포레이션 변이형 RNAi
US20160319278A1 (en) 2015-04-03 2016-11-03 University Of Massachusetts Fully stabilized asymmetric sirna
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US10633653B2 (en) 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
EP3366774A4 (en) * 2015-10-23 2019-03-27 Rena Therapeutics Inc. NUCLEIC ACID COMPLEX WITH AT LEAST ONE WOOD STRUCTURE
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
EP3619308A4 (en) 2017-05-05 2021-01-27 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT FOR HUNTINGTON'S MORBUS
JP7406793B2 (ja) 2017-06-23 2023-12-28 ユニバーシティー オブ マサチューセッツ 2テイル自己デリバリー型siRNAおよび関連方法
MY205041A (en) * 2017-09-22 2024-09-29 Genzyme Corp Variant rnai
US20200377887A1 (en) * 2017-09-22 2020-12-03 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
MX388998B (es) * 2017-12-06 2025-03-11 Ovid Therapeutics Inc Uso de mir101 o mir128 en el tratamiento de trastornos convulsivos.
EP3768386A4 (en) 2018-03-23 2022-04-13 University of Massachusetts GENE THERAPY FOR THE TREATMENT OF BONE DISORDERS
TW202012628A (zh) 2018-05-15 2020-04-01 美商航海家醫療公司 用於帕金森氏症治療的組成物及方法
CN112770812A (zh) 2018-07-24 2021-05-07 沃雅戈治疗公司 产生基因治疗制剂的系统和方法
KR20210062627A (ko) * 2018-08-03 2021-05-31 젠자임 코포레이션 알파-시뉴클레인에 대한 변이형 RNAi
WO2020041769A1 (en) 2018-08-23 2020-02-27 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
CN113194982B (zh) * 2018-10-12 2025-04-01 建新公司 通过肝导向基因替代疗法治疗严重pku的改良的人pah的产生
BR112021013109A2 (pt) * 2019-01-09 2021-10-13 Universidade De Coimbra Rna de fita dupla e seus usos.
EP3911747A4 (en) 2019-01-18 2023-05-24 University Of Massachusetts DYNAMIC PHARMACOKINETICS-MODIFYING ANCHORS
EP3983077A4 (en) * 2019-06-17 2023-12-20 Alnylam Pharmaceuticals, Inc. DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM
KR20220047989A (ko) 2019-08-09 2022-04-19 유니버시티 오브 매사추세츠 Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
CA3176204A1 (en) * 2020-04-20 2021-10-28 Anastasia Khvorova Oligonucleotides for msh3 modulation
EP4442830A4 (en) 2021-11-29 2025-04-30 Shanghai Regenelead Therapies Co., Ltd. AADC/GDNF polynucleotide and use thereof in the treatment of Parkinson's disease
CA3244192A1 (en) 2022-02-21 2025-01-22 Shanghai Regenelead Therapies Co Ltd VEGF-BINDING MOLECULE AND ITS PHARMACEUTICAL USE
WO2024187106A2 (en) * 2023-03-09 2024-09-12 Leal Therapeutics, Inc. Compositions and methods for modulating grin2a

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2264482A1 (en) 1996-09-06 1998-03-12 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
JP4827353B2 (ja) 1999-08-09 2011-11-30 ターゲティッド ジェネティクス コーポレイション 鎖内塩基対を形成するような配列の設計による、組換えウイルスベクターからの一本鎖の異種ヌクレオチド配列の発現の増大
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
ES2455126T3 (es) 2001-11-13 2014-04-14 The Trustees Of The University Of Pennsylvania Secuencias Cy.5 de virus adeno-asociados (AAV), vectores que las contienen y uso de las mismas.
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
AU2003282877B9 (en) 2002-09-25 2011-05-12 University Of Massachusetts In Vivo gene silencing by chemically modified and stable siRNA
WO2004101787A1 (ja) * 2003-05-14 2004-11-25 Japan Science And Technology Agency ハンチンチン遺伝子の発現抑制
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
US7902352B2 (en) * 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
WO2008134646A2 (en) 2007-04-26 2008-11-06 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
ES2549122T3 (es) * 2007-05-31 2015-10-23 University Of Iowa Research Foundation Reducción de toxicidad por interferencia de ARN desviada de su diana
EP2240783A2 (en) * 2008-02-04 2010-10-20 Galapagos N.V. Molecular targets and compounds, methods to identify the same, useful in the treatment of neurodegenerative diseases.
WO2009100502A1 (en) 2008-02-14 2009-08-20 Monash University Immunostimulatory sirna molecules
FR2929292A1 (fr) 2008-03-28 2009-10-02 Exonhit Therapeutics S A Sa Procede et methodes de diagnostic de la maladie d'alzheimer
US8796238B2 (en) * 2009-09-17 2014-08-05 Sigma-Aldrich Co. Llc Short RNA mimetics
JP5963743B2 (ja) 2010-04-23 2016-08-03 ユニバーシティ オブ マサチューセッツ Cnsターゲティングaavベクターおよびその使用方法
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
ES2724800T3 (es) 2011-02-17 2019-09-16 Univ Pennsylvania Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9
KR102084539B1 (ko) 2012-02-29 2020-03-04 상가모 테라퓨틱스, 인코포레이티드 헌팅턴병을 치료하기 위한 방법 및 조성물
US9550989B2 (en) * 2013-10-03 2017-01-24 Washington University Rational design of microRNA-siRNA chimeras for multi-functional target suppression
SG10201912968WA (en) 2014-03-21 2020-02-27 Genzyme Corp Gene therapy for retinitis pigmentosa
HRP20211024T4 (hr) 2014-05-02 2025-06-20 Genzyme Corporation Aav vektori za gensku terapiju mrežnice i cns-a
KR102636633B1 (ko) 2014-12-24 2024-02-13 유니큐어 아이피 비.브이. 헌팅턴 유전자 억제를 유도하는 RNAi
KR102670852B1 (ko) 2015-02-10 2024-05-29 젠자임 코포레이션 변이형 RNAi
MY205041A (en) 2017-09-22 2024-09-29 Genzyme Corp Variant rnai

Also Published As

Publication number Publication date
MY181458A (en) 2020-12-22
US10760079B2 (en) 2020-09-01
JP2021118740A (ja) 2021-08-12
CO2017009083A2 (es) 2018-01-31
WO2016130589A3 (en) 2016-09-15
TW201704470A (zh) 2017-02-01
US20210047641A1 (en) 2021-02-18
US20180023082A1 (en) 2018-01-25
ECSP17059343A (es) 2019-03-29
US20240182896A1 (en) 2024-06-06
CN107438671B (zh) 2021-05-25
NZ772770A (en) 2024-10-25
AU2016219396A1 (en) 2017-09-28
CA2976075A1 (en) 2016-08-18
PE20171382A1 (es) 2017-09-15
MX2022013005A (es) 2022-11-09
JP2018506304A (ja) 2018-03-08
AR103646A1 (es) 2017-05-24
KR20170110149A (ko) 2017-10-10
MY194175A (en) 2022-11-17
IL253893A0 (en) 2017-10-31
KR102670852B1 (ko) 2024-05-29
NZ735289A (en) 2024-07-26
US11781137B2 (en) 2023-10-10
IL253893B (en) 2021-08-31
MA40819A1 (fr) 2018-07-31
UY36554A (es) 2016-09-30
BR112017017028A2 (pt) 2018-04-10
US20200109401A1 (en) 2020-04-09
TWI781079B (zh) 2022-10-21
CN107438671A (zh) 2017-12-05
SG10201907396XA (en) 2019-09-27
CL2017002027A1 (es) 2018-04-20
AU2025205501A1 (en) 2025-08-07
JP7526710B2 (ja) 2024-08-01
SG10201912942YA (en) 2020-02-27
EA201791805A1 (ru) 2018-05-31
HK1247641A1 (zh) 2018-09-28
US10450563B2 (en) 2019-10-22
MA40819B1 (fr) 2020-04-30
SG11201706444TA (en) 2017-09-28
EP3256588A2 (en) 2017-12-20
TN2017000354A1 (en) 2019-01-16
AU2022204274B2 (en) 2025-04-17
PH12017501432A1 (en) 2018-01-15
CR20170406A (es) 2017-11-14
WO2016130589A2 (en) 2016-08-18
AU2022204274A1 (en) 2022-07-07
AU2016219396B2 (en) 2022-03-17

Similar Documents

Publication Publication Date Title
MX2022013005A (es) Arni variante.
MX2025008949A (es) Arni variante
MX2018014152A (es) Composiciones y métodos para tratar la enfermedad de huntington.
BR112017004349A2 (pt) terapia com gene de globina para o tratamento de hemoglobinopatias
SA519401379B1 (ar) علاج فيروس مرتبط بغدة لمرض هنتنجتون
MX2019013172A (es) Composiciones y metodos para tratar la enfermedad de huntington.
PH12019550076A1 (en) SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
BR112017005892A2 (pt) variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma
EP4393495A3 (en) Programmed cell death 1 ligand 1 (pd-l1) irna compositions and methods of use thereof
EA201500802A1 (ru) Способы профилактики и/или лечения кардиомиопатии и устранения симптомов кардиомиопатии, фармацевтическая композиция, векторы
SG10201901482XA (en) Oncolytic adenovirus encoding a b7 protein
UY36106A (es) Vectores aav para terapia génica retiniana y del snc.
BR112017005235A2 (pt) vetores virais adeno-associados para o tratamento de glaucoma de miocilina (myoc)
EA201890861A1 (ru) Композиции, содержащие лентивирусные векторы, экспрессирующие il-12, и способы их применения
MX2021005980A (es) Adenovirus y metodos para usar adenovirus.
BR112016010342A2 (pt) sequência de rna isolada ou sintética, sequência de dna, vetor que permite a expressão de uma sequência de rna, célula que compreende uma sequência de rna, composição que compreende uma sequência de rna, utilização de uma sequência de rna, camundongo transgênico, método que permite avaliar a eficácia cosmética de um composto de teste, e, método que permite avaliar a eficácia terapêutica de um composto de teste
DOP2017000185A (es) Arni variante
RU2014145910A (ru) Способы воздействия на экспрессию гена APOE