CO2021002637A2 - Arni variante frente a alfa-sinucleína - Google Patents

Arni variante frente a alfa-sinucleína

Info

Publication number
CO2021002637A2
CO2021002637A2 CONC2021/0002637A CO2021002637A CO2021002637A2 CO 2021002637 A2 CO2021002637 A2 CO 2021002637A2 CO 2021002637 A CO2021002637 A CO 2021002637A CO 2021002637 A2 CO2021002637 A2 CO 2021002637A2
Authority
CO
Colombia
Prior art keywords
synuclein
rnai
arni
alpha
variant versus
Prior art date
Application number
CONC2021/0002637A
Other languages
English (en)
Spanish (es)
Inventor
Bradford Elmer
Brenda Richards
Martine Latta-Mahieu
Maria Carmen Obinu
Véronique Taupin
Véronique Blanchard
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 CO2021002637A2 publication Critical patent/CO2021002637A2/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
    • 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
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/38Vector systems having a special element relevant for transcription being a stuffer
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/42Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CONC2021/0002637A 2018-08-03 2021-02-26 Arni variante frente a alfa-sinucleína CO2021002637A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862714616P 2018-08-03 2018-08-03
PCT/US2019/044924 WO2020028816A1 (en) 2018-08-03 2019-08-02 Variant rnai against alpha-synuclein

Publications (1)

Publication Number Publication Date
CO2021002637A2 true CO2021002637A2 (es) 2021-07-30

Family

ID=67587976

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2021/0002637A CO2021002637A2 (es) 2018-08-03 2021-02-26 Arni variante frente a alfa-sinucleína

Country Status (16)

Country Link
US (1) US12365895B2 (enExample)
EP (1) EP3830265A1 (enExample)
JP (2) JP2021534814A (enExample)
KR (1) KR20210062627A (enExample)
CN (1) CN112805382A (enExample)
AR (1) AR114540A1 (enExample)
AU (1) AU2019314529B2 (enExample)
BR (1) BR112021001980A2 (enExample)
CA (1) CA3108526A1 (enExample)
CO (1) CO2021002637A2 (enExample)
IL (1) IL280581A (enExample)
MX (1) MX2021001395A (enExample)
PH (1) PH12021550254A1 (enExample)
SG (1) SG11202101072UA (enExample)
TW (2) TW202509215A (enExample)
WO (1) WO2020028816A1 (enExample)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3684932A1 (en) 2017-09-22 2020-07-29 Genzyme Corporation Variant rnai
PH12021550254A1 (en) 2018-08-03 2023-02-06 Genzyme Corp Variant rnai against alpha-synuclein
CN115777021A (zh) * 2020-03-18 2023-03-10 马萨诸塞大学 用于snca调节的寡核苷酸
WO2022072447A1 (en) * 2020-10-01 2022-04-07 Alnylam Pharmaceuticals, Inc. Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases
EP4359525A1 (en) * 2021-06-21 2024-05-01 uniQure biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
AU2023248463A1 (en) * 2022-04-06 2024-11-28 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
JP2025531512A (ja) * 2022-09-29 2025-09-19 アタランタ セラピューティクス,インコーポレーテッド アルファシヌクレイン核酸を標的化するsiRNA組成物及び方法
WO2024211480A2 (en) * 2023-04-05 2024-10-10 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2025160429A1 (en) * 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting snca
TW202529779A (zh) * 2024-01-30 2025-08-01 大陸商上海舶望製藥有限公司 用於抑制突觸核蛋白α(SNCA)基因表達的組合物和方法

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6989264B2 (en) 1997-09-05 2006-01-24 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
DE60039766D1 (de) 1999-08-09 2008-09-18 Targeted Genetics Corp Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
US20050137155A1 (en) 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
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
CA2915124C (en) 2001-11-13 2018-08-14 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
PT2164967E (pt) 2007-05-31 2015-10-27 Univ Iowa Res Found Redução da toxicidade não dirigida do arn de interferência
GB0816778D0 (en) 2008-09-12 2008-10-22 Isis Innovation Gene silencing
EP3514232A1 (en) 2010-04-23 2019-07-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
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
AU2011329777B2 (en) 2010-11-17 2016-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
JP6224459B2 (ja) 2011-02-17 2017-11-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組織特異性を改変し、aav9媒介遺伝子導入を改善するための組成物および方法
BR112014021104B1 (pt) 2012-02-29 2023-03-28 Sangamo Biosciences, Inc Proteína de fusão de ocorrência não natural compreendendo um domínio de ligação de dna de dedo de zinco manipulado que se liga a um gene htt, seu uso, método in vitro de modificação da expressão de um gene htt em uma célula, e método de geração de um sistema modelo para o estudo da doença de huntington
EP4600255A3 (en) 2014-05-02 2025-10-22 Genzyme Corporation Aav vectors for retinal and cns gene therapy
US10450563B2 (en) 2015-02-10 2019-10-22 Genzyme Corporation Variant RNAi
PH12021550254A1 (en) 2018-08-03 2023-02-06 Genzyme Corp Variant rnai against alpha-synuclein

Also Published As

Publication number Publication date
AU2019314529B2 (en) 2025-09-11
SG11202101072UA (en) 2021-03-30
MX2021001395A (es) 2021-08-11
CN112805382A (zh) 2021-05-14
US20210309999A1 (en) 2021-10-07
PH12021550254A1 (en) 2023-02-06
WO2020028816A1 (en) 2020-02-06
BR112021001980A2 (pt) 2021-05-04
JP2021534814A (ja) 2021-12-16
JP2024156715A (ja) 2024-11-06
US12365895B2 (en) 2025-07-22
AU2019314529A1 (en) 2021-03-25
AR114540A1 (es) 2020-09-16
IL280581A (en) 2021-03-25
TW202022116A (zh) 2020-06-16
KR20210062627A (ko) 2021-05-31
CA3108526A1 (en) 2020-02-06
TW202509215A (zh) 2025-03-01
EP3830265A1 (en) 2021-06-09

Similar Documents

Publication Publication Date Title
CO2021002637A2 (es) Arni variante frente a alfa-sinucleína
CL2024000823A1 (es) Composiciones y métodos de inhibición del masp-3 para tratar enfermedades y trastornos div 201900230
CL2020000991A1 (es) Proteínas trispecíficas y métodos de uso.
CL2017002027A1 (es) Arni variante.
UY37897A (es) ARNi VARIANTE
ECSP20030074A (es) Nuevos profármacos de catecolamina para uso en el tratamiento de la enfermedad de parkinson
BR112018074454A2 (pt) composições e métodos de uso de nintedanib para tratar doenças oculares com neovascularização anormal
BR112018014281A2 (pt) agentes, usos e métodos para o tratamento da sinucleinopatia
CL2018003438A1 (es) Anticuerpos a la alfa-sinucleína y usos de los mismos.
CL2019001749A1 (es) Uso de anticuerpos anti-esclerostina en el tratamiento de osteogenesis imperfecta.
MX2017005940A (es) Pyrazolo [1, 5-a] pirimidinas sustituidas y su uso en el tratamiento de trastornos médicos.
CO2019006979A2 (es) Tratamiento de enfermedades neurológicas
BR112019003579A2 (pt) Composições de ribosídeo de nicotinamida e pterostilbeno e métodos para o tratamento dedistúrbios neurodegenerativos
UY29796A1 (es) Nuevos compuestos para el tratamiento de trastornos neurológicos, psiquiátricos o del dolor
MX2020011826A (es) Composiciones de combinacion que comprenden compuestos de bisfluoroalquil-1,4-benzodiazepinona y metodos de uso de las mismas.
MX2021005822A (es) Derivados de bis-octahidrofenantreno carboxamida y conjugados de proteinas de los mismos para usarse como agonistas de lxr.
MX2024013040A (es) Pirfenidona enriquecida con deuterio y metodos para su uso
ZA202002626B (en) Use of riluzole prodrugs to treat ataxias
CL2016000019A1 (es) Compuestos derivados de dihidroxifenilo deuterado o una sal de los mismos; composición farmacéutica que los comprende y su uso para el tratamiento de trastornos mediados por neurotransmisores tales como hipotensión, trastornos del sueño, enfermedad de alzheimer y depresión.
MX2020009532A (es) Oligonucleotidos modificados para uso en el tratamiento de tauopatias.
CL2020002038A1 (es) Oligonucleótidos para modular la expresión de tmem106b.
UY37729A (es) NUEVOS COMPUESTOS QUE INHIBEN LA ACTIVIDAD DE Nav1.7
CL2021000930A1 (es) Piridazinas novedosas
MX2022004212A (es) Moduladores de atf6 y usos de los mismos.
CL2020002398A1 (es) Inhibidores de secreción de proteínas basados en triazaciclododecansulfonamida ("tcd")