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SG11202108502UA - Polynucleotides - Google Patents

Polynucleotides

Info

Publication number
SG11202108502UA
SG11202108502UA SG11202108502UA SG11202108502UA SG11202108502UA SG 11202108502U A SG11202108502U A SG 11202108502UA SG 11202108502U A SG11202108502U A SG 11202108502UA SG 11202108502U A SG11202108502U A SG 11202108502UA SG 11202108502U A SG11202108502U A SG 11202108502UA
Authority
SG
Singapore
Prior art keywords
polynucleotides
Prior art date
Application number
SG11202108502UA
Inventor
Amit Nathwani
Jenny Mcintosh
Romuald Corbau
Azadeh Kia
Carlos Miranda
Original Assignee
Freeline Therapeutics Ltd
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
Priority claimed from GBGB1901512.2A external-priority patent/GB201901512D0/en
Priority claimed from GBGB1917910.0A external-priority patent/GB201917910D0/en
Application filed by Freeline Therapeutics Ltd filed Critical Freeline Therapeutics Ltd
Publication of SG11202108502UA publication Critical patent/SG11202108502UA/en

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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • C12N15/52Genes encoding for enzymes or proenzymes
    • 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
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (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)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
SG11202108502UA 2019-02-04 2020-02-04 Polynucleotides SG11202108502UA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1901512.2A GB201901512D0 (en) 2019-02-04 2019-02-04 Polynucleotides
GBGB1917910.0A GB201917910D0 (en) 2019-12-06 2019-12-06 Polynucleotides
PCT/GB2020/050251 WO2020161483A1 (en) 2019-02-04 2020-02-04 Polynucleotides

Publications (1)

Publication Number Publication Date
SG11202108502UA true SG11202108502UA (en) 2021-09-29

Family

ID=69591668

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202108502UA SG11202108502UA (en) 2019-02-04 2020-02-04 Polynucleotides

Country Status (19)

Country Link
US (1) US20220154159A1 (en)
EP (2) EP4299116A3 (en)
JP (1) JP7600124B2 (en)
KR (1) KR20210148101A (en)
CN (1) CN113795575A (en)
AU (1) AU2020217894A1 (en)
BR (1) BR112021015312A2 (en)
CA (1) CA3128875A1 (en)
DK (1) DK3850089T5 (en)
ES (1) ES2968922T3 (en)
FI (1) FI3850089T3 (en)
HR (1) HRP20240184T1 (en)
HU (1) HUE065347T2 (en)
IL (1) IL285350B1 (en)
MX (1) MX2021009305A (en)
PL (1) PL3850089T3 (en)
PT (1) PT3850089T (en)
SG (1) SG11202108502UA (en)
WO (1) WO2020161483A1 (en)

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001036603A2 (en) * 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
EP1319082B1 (en) * 2000-09-18 2005-11-16 Genzyme Corporation Expression vectors containing hybrid ubiquitin promoters
US20040009151A1 (en) * 2002-04-04 2004-01-15 Kay Mark A. Methods for delivering recombinant adeno-associated virus virions to the liver of a mammal
US20050265988A1 (en) * 2004-03-18 2005-12-01 Byung-Kwon Choi Glycosylated glucocerebrosidase expression in fungal hosts
CA3069431A1 (en) 2007-04-03 2008-10-09 Oxyrane Uk Limited Glycosylation of molecules
JP5291093B2 (en) 2007-05-16 2013-09-18 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド Treatment of synucleinopathy
EP2365796A4 (en) * 2008-11-07 2015-04-29 Childrens Hosp Medical Center FUSOGENIC PROPERTIES OF SAPOSIN C AND RELATED PROTEINS AND PEPTIDES FOR APPLICATION TO TRANSMEMBRANE DRUG DELIVERY SYSTEMS
KR20180108886A (en) 2010-03-23 2018-10-04 인트렉손 코포레이션 Vectors Conditionally Expressing Therapeutic Proteins, Host Cells Comprising the Vectors, and Uses Thereof
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
US10648001B2 (en) * 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
ES2745566T3 (en) * 2013-06-11 2020-03-02 Portage Pharmaceuticals Ltd Structure, Manufacture, and Uses of Peptides that Can Penetrate Human-derived Cells Conjugated to Specific Biologically Active Charging Peptides
CA2927366A1 (en) * 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
US10335466B2 (en) * 2014-11-05 2019-07-02 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of parkinson's disease
CA2974050A1 (en) * 2015-03-17 2016-09-22 Greenovation Biotech Gmbh Glycosylated lysosomal proteins, method of production and uses
WO2017048495A1 (en) * 2015-09-14 2017-03-23 Children's Hospital Medical Center Methods and compositions for treatment of gaucher disease via modulation of c5a receptor
CA3039673A1 (en) * 2016-10-20 2018-04-26 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
AU2018264581B2 (en) 2017-05-11 2022-05-19 Zentrum Für Forschungsförderung In Der Pädiatrie Gmbh Concept for the treatment of monogenetic disorders
US11597917B2 (en) * 2017-07-06 2023-03-07 The Medical College Of Wisconsin, Inc. In vitro and in vivo enrichment strategy targeting lymphocytes derived from vector transduced HSCs for therapy of disorders

Also Published As

Publication number Publication date
DK3850089T3 (en) 2024-02-12
CA3128875A1 (en) 2020-08-13
IL285350B1 (en) 2025-03-01
US20220154159A1 (en) 2022-05-19
JP2022520179A (en) 2022-03-29
PT3850089T (en) 2024-01-24
CN113795575A (en) 2021-12-14
ES2968922T3 (en) 2024-05-14
EP4299116A2 (en) 2024-01-03
WO2020161483A1 (en) 2020-08-13
JP7600124B2 (en) 2024-12-16
MX2021009305A (en) 2021-11-04
HRP20240184T1 (en) 2024-04-26
BR112021015312A2 (en) 2021-11-09
FI3850089T3 (en) 2024-02-14
EP3850089B1 (en) 2023-12-13
KR20210148101A (en) 2021-12-07
EP4299116A3 (en) 2024-06-19
HUE065347T2 (en) 2024-05-28
PL3850089T3 (en) 2024-05-06
DK3850089T5 (en) 2024-08-19
AU2020217894A1 (en) 2021-08-26
EP3850089A1 (en) 2021-07-21
IL285350A (en) 2021-09-30

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