MA47847A - Thérapie génique pour traiter la mucopolysaccharidose de type i - Google Patents

Thérapie génique pour traiter la mucopolysaccharidose de type i

Info

Publication number
MA47847A
MA47847A MA047847A MA47847A MA47847A MA 47847 A MA47847 A MA 47847A MA 047847 A MA047847 A MA 047847A MA 47847 A MA47847 A MA 47847A MA 47847 A MA47847 A MA 47847A
Authority
MA
Morocco
Prior art keywords
gene therapy
mucopolysaccharidosis type
treat mucopolysaccharidosis
treat
type
Prior art date
Application number
MA047847A
Other languages
English (en)
Inventor
Kendrick A Goss
Geoffrey B Parsons
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 MA47847A publication Critical patent/MA47847A/fr

Links

Classifications

    • 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
    • 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
    • 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)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Ophthalmology & Optometry (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Diabetes (AREA)
MA047847A 2016-12-06 2017-12-06 Thérapie génique pour traiter la mucopolysaccharidose de type i MA47847A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662430795P 2016-12-06 2016-12-06

Publications (1)

Publication Number Publication Date
MA47847A true MA47847A (fr) 2020-01-29

Family

ID=62491308

Family Applications (1)

Application Number Title Priority Date Filing Date
MA047847A MA47847A (fr) 2016-12-06 2017-12-06 Thérapie génique pour traiter la mucopolysaccharidose de type i

Country Status (13)

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

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7045362B2 (ja) 2016-04-20 2022-03-31 セントロ デ インベスティガシオンス エネルジェチカス メディオアンビエンタゥス イェ テクノロジカス オー．エイ． エム．ピー． Ｐｋｌｒの遺伝子発現増強のための組成物および方法
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
KR102726267B1 (ko) 2017-10-16 2024-11-11 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 피루베이트 키나제 결핍증을 치료하기 위한 pklr 전달용 렌티바이러스 벡터
IL280465B2 (en) * 2018-07-30 2025-03-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Methods for gene modification of hematopoietic cells
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 (fr) * 2021-06-08 2022-12-15 Touchlight IP Limited Vecteur lentiviral
WO2024201066A1 (fr) * 2023-03-31 2024-10-03 Dawn Therapeutics Limited Nouveau vecteur

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
CN103717240A (zh) * 2011-06-10 2014-04-09 蓝鸟生物公司 用于肾上腺脑白质营养不良症和肾上腺脊髓神经病的基因治疗载体
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
US20160122727A1 (en) * 2013-06-13 2016-05-05 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
WO2017139561A1 (fr) * 2016-02-12 2017-08-17 Bluebird Bio, Inc. Compositions d'augmentation de vcn et ses procédés d'utilisation

Also Published As

Publication number Publication date
BR112019011635A2 (pt) 2019-11-12
CA3046079A1 (fr) 2018-06-14
IL267057A (en) 2019-08-29
RU2019120721A (ru) 2021-01-11
KR20190089988A (ko) 2019-07-31
AU2017370662A1 (en) 2019-06-27
EP3551750A1 (fr) 2019-10-16
WO2018106807A1 (fr) 2018-06-14
EP3551750A4 (fr) 2020-07-22
MX2019006552A (es) 2019-10-15
JP2019536484A (ja) 2019-12-19
US20220339296A1 (en) 2022-10-27
CN110214182A (zh) 2019-09-06

Similar Documents

Publication Publication Date Title
MA43968A (fr) Thérapie génique pour traiter la mucopolysaccharidose de type i
MA44874A (fr) Thérapie génique pour traiter la mucopolysaccharidose de type ii
IL271085A (en) Bacteria for the treatment of disorders
MA50016A (fr) Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i
IL257486A (en) Methods and compositions for treating a proprotein convertase subtilisin kexin (pcsk9) gene-associated disorder
PL3265053T3 (pl) Sposoby leczenia skóry
DK3262066T4 (da) Genterapi
HRP20190165T1 (hr) Genska terapija za fabryjevu bolest
LT3283527T (lt) Kompleksinė terapija vėžio gydymui
MA47847A (fr) Thérapie génique pour traiter la mucopolysaccharidose de type i
SMT202400506T1 (it) Terapia genica
IL259762B (en) Complement activity modulators
IL256206A (en) Mct4 inhibitors for treating disease
MA43570A (fr) Thérapie génique pour traiter l'hypercholestérolémie familiale
EP3717653A4 (fr) Thérapie génique pour la mucopolysaccharidose de type iiia
HUE056613T2 (hu) Komplementaktivitás modulátorai
PL3612237T3 (pl) Terapia genowa
IL257764B (en) Methods for treatment of diseases
IL255581B (en) Methods of treating epstein-barr virus-associated lymphoproliferative disorders by t cell therapy
IL269544A (en) Optimization of enzyme replacement therapy for treatment of homocystinuria
DK3302478T3 (da) Pac-1 kombinations behandling
MA47173A (fr) Thérapie génique pour traiter la mucopolysaccharidose de type ii
PL3261679T3 (pl) Terapia genowa dla poprawy wzroku