BR112019011635A2 - terapia genética para mucopolissacaridose, tipo i - Google Patents

terapia genética para mucopolissacaridose, tipo i

Info

Publication number
BR112019011635A2
BR112019011635A2 BR112019011635-4A BR112019011635A BR112019011635A2 BR 112019011635 A2 BR112019011635 A2 BR 112019011635A2 BR 112019011635 A BR112019011635 A BR 112019011635A BR 112019011635 A2 BR112019011635 A2 BR 112019011635A2
Authority
BR
Brazil
Prior art keywords
mucopolysaccharidosis
type
gene therapy
methods
provides compositions
Prior art date
Application number
BR112019011635-4A
Other languages
English (en)
Portuguese (pt)
Inventor
B Parsons Geoffrey
A Goss Kendrick
Original Assignee
Bluebird Bio Inc
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 Bluebird Bio Inc filed Critical Bluebird Bio Inc
Publication of BR112019011635A2 publication Critical patent/BR112019011635A2/pt

Links

Classifications

    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs 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
    • 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
    • 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
    • A61P27/16—Otologicals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Hospice & Palliative Care (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Obesity (AREA)
  • Psychiatry (AREA)
  • Ophthalmology & Optometry (AREA)
BR112019011635-4A 2016-12-06 2017-12-06 terapia genética para mucopolissacaridose, tipo i BR112019011635A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662430795P 2016-12-06 2016-12-06
PCT/US2017/064913 WO2018106807A1 (en) 2016-12-06 2017-12-06 Gene therapy for mucopolysaccharidosis, type i

Publications (1)

Publication Number Publication Date
BR112019011635A2 true BR112019011635A2 (pt) 2019-11-12

Family

ID=62491308

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112019011635-4A BR112019011635A2 (pt) 2016-12-06 2017-12-06 terapia genética para mucopolissacaridose, tipo i

Country Status (13)

Country Link
US (1) US20220339296A1 (enExample)
EP (1) EP3551750A4 (enExample)
JP (1) JP2019536484A (enExample)
KR (1) KR20190089988A (enExample)
CN (1) CN110214182A (enExample)
AU (1) AU2017370662A1 (enExample)
BR (1) BR112019011635A2 (enExample)
CA (1) CA3046079A1 (enExample)
IL (1) IL267057A (enExample)
MA (1) MA47847A (enExample)
MX (1) MX2019006552A (enExample)
RU (1) RU2019120721A (enExample)
WO (1) WO2018106807A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109641063B (zh) 2016-04-20 2022-11-15 能源环境和技术研究中心O.A., M.P. 用于增强pklr的基因表达的组合物和方法
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
JP7576754B2 (ja) 2018-04-11 2024-11-01 スペースクラフト セブン リミテッド ライアビリティ カンパニー 幹細胞移植のための組成物および方法
MX2021001292A (es) * 2018-07-30 2021-07-15 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Metodos para la modificacion genica de celulas hematopoyeticas.
CN115109790A (zh) * 2021-03-23 2022-09-27 北京据德医药科技有限公司 一种重组a-L-艾杜糖醛酸普酶及其制备方法
CA3222751A1 (en) * 2021-06-08 2022-12-15 Touchlight IP Limited Lentiviral vector
WO2024201066A1 (en) * 2023-03-31 2024-10-03 Dawn Therapeutics Limited Novel vector

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2872170B1 (fr) * 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse Lentivirus non interactif et non replicatif, preparation et utilisations
US20110294114A1 (en) * 2009-12-04 2011-12-01 Cincinnati Children's Hospital Medical Center Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells
AU2012267512B2 (en) * 2011-06-10 2014-11-20 Bluebird Bio, Inc. Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
US9695443B2 (en) * 2012-05-25 2017-07-04 Commissariat A L'energie Atomique Et Aux Energies Alternatives Vector for the selective silencing of a gene in astrocytes
WO2014201252A2 (en) * 2013-06-13 2014-12-18 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
EP3413896B1 (en) * 2016-02-12 2021-03-17 Bluebird Bio, Inc. Vcn enhancer compositions and methods of using the same

Also Published As

Publication number Publication date
EP3551750A4 (en) 2020-07-22
JP2019536484A (ja) 2019-12-19
CN110214182A (zh) 2019-09-06
CA3046079A1 (en) 2018-06-14
AU2017370662A1 (en) 2019-06-27
RU2019120721A (ru) 2021-01-11
MX2019006552A (es) 2019-10-15
US20220339296A1 (en) 2022-10-27
MA47847A (fr) 2020-01-29
EP3551750A1 (en) 2019-10-16
IL267057A (en) 2019-08-29
WO2018106807A1 (en) 2018-06-14
KR20190089988A (ko) 2019-07-31

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Legal Events

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B350 Update of information on the portal [chapter 15.35 patent gazette]
B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

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B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

Free format text: EM VIRTUDE DO ARQUIVAMENTO PUBLICADO NA RPI 2699 DE 27-09-2022 E CONSIDERANDO AUSENCIA DE MANIFESTACAO DENTRO DOS PRAZOS LEGAIS, INFORMO QUE CABE SER MANTIDO O ARQUIVAMENTO DO PEDIDO DE PATENTE, CONFORME O DISPOSTO NO ARTIGO 12, DA RESOLUCAO 113/2013.