BR112016025819B1 - Vetores de vírus adeno-associados para o tratamento de doenças de depósito lisossômico - Google Patents

Vetores de vírus adeno-associados para o tratamento de doenças de depósito lisossômico Download PDF

Info

Publication number
BR112016025819B1
BR112016025819B1 BR112016025819-3A BR112016025819A BR112016025819B1 BR 112016025819 B1 BR112016025819 B1 BR 112016025819B1 BR 112016025819 A BR112016025819 A BR 112016025819A BR 112016025819 B1 BR112016025819 B1 BR 112016025819B1
Authority
BR
Brazil
Prior art keywords
cag
seq
alpha
aav9
vectors
Prior art date
Application number
BR112016025819-3A
Other languages
English (en)
Portuguese (pt)
Other versions
BR112016025819A2 (pt
Inventor
Maria Fàtima Bosch Tubert
Virginia AREBA HAURIGOT
Albert RIBERA SANCHEZ
Original Assignee
Universitat Autònoma De Barcelona
Esteve Pharmaceuticals, S.A.
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 Universitat Autònoma De Barcelona, Esteve Pharmaceuticals, S.A. filed Critical Universitat Autònoma De Barcelona
Publication of BR112016025819A2 publication Critical patent/BR112016025819A2/pt
Publication of BR112016025819B1 publication Critical patent/BR112016025819B1/pt

Links

Images

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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary 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
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • 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
    • 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
    • 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
    • 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)
    • C12N9/2474—Hyaluronoglucosaminidase (3.2.1.35), i.e. hyaluronidase
    • 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)
    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation
    • C12N2840/105—Vectors comprising a special translation-regulating system regulates levels of translation enhancing translation

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Rheumatology (AREA)
BR112016025819-3A 2014-05-14 2015-05-13 Vetores de vírus adeno-associados para o tratamento de doenças de depósito lisossômico BR112016025819B1 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP14382171 2014-05-14
EP14382171.8 2014-05-14
PCT/EP2015/060604 WO2015173308A1 (en) 2014-05-14 2015-05-13 Adenoassociated virus vectors for the treatment of lysosomal storage disorders

Publications (2)

Publication Number Publication Date
BR112016025819A2 BR112016025819A2 (pt) 2018-07-03
BR112016025819B1 true BR112016025819B1 (pt) 2022-08-16

Family

ID=50771225

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112016025819-3A BR112016025819B1 (pt) 2014-05-14 2015-05-13 Vetores de vírus adeno-associados para o tratamento de doenças de depósito lisossômico

Country Status (22)

Country Link
US (1) US11149285B2 (pt-PT)
EP (1) EP3143146B1 (pt-PT)
JP (1) JP6634073B2 (pt-PT)
KR (1) KR102446169B1 (pt-PT)
CN (1) CN107002095B (pt-PT)
AR (1) AR100419A1 (pt-PT)
AU (1) AU2015261519B2 (pt-PT)
BR (1) BR112016025819B1 (pt-PT)
CA (1) CA2947468C (pt-PT)
ES (1) ES2823948T3 (pt-PT)
IL (1) IL248538B (pt-PT)
MX (1) MX2016014773A (pt-PT)
MY (1) MY183472A (pt-PT)
NZ (1) NZ726316A (pt-PT)
PH (1) PH12016502248A1 (pt-PT)
PT (1) PT3143146T (pt-PT)
RU (1) RU2718248C2 (pt-PT)
SA (1) SA516380290B1 (pt-PT)
SG (1) SG11201609314XA (pt-PT)
TW (1) TWI701332B (pt-PT)
UA (1) UA123985C2 (pt-PT)
WO (1) WO2015173308A1 (pt-PT)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3128549A1 (en) 2010-07-12 2012-01-19 Universitat Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
WO2015017256A1 (en) 2013-07-29 2015-02-05 Danisco Us Inc. Variant enzymes
EP3194430A1 (en) 2014-09-16 2017-07-26 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
EP3015135B1 (en) 2014-10-29 2020-09-09 Noxell Corporation Hair colouration with a cationic or anionic coloured polymer, method and kit thereof
EP3015134B1 (en) 2014-10-29 2020-08-26 Noxell Corporation Hair colouration with a cationic coloured polymer, method and kit thereof
ES2824457T3 (es) 2014-12-05 2021-05-12 UNIV AUTòNOMA DE BARCELONA Vectores virales para el tratamiento de la diabetes
EP3978614A3 (en) 2015-01-07 2022-07-27 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
EP3485005A1 (en) * 2016-07-12 2019-05-22 The University Of Manchester Gene therapy
CN109863243B (zh) * 2016-09-30 2023-07-28 埃斯蒂文制药股份有限公司 用于治疗粘多糖症的腺相关病毒载体
EP3717652A4 (en) * 2017-11-30 2021-11-24 The Trustees of the University of Pennsylvania GENE THERAPY FOR MUCOPOLY SACCHARIDOSIS IIIB
EP3784695B1 (en) * 2018-04-27 2023-08-09 The Medical College of Wisconsin, Inc. Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders
KR20210027266A (ko) * 2018-05-30 2021-03-10 에스테베 파마슈티칼스 에스에이 뮤코다당질축적증 ｉｖ ａ형의 치료를 위한 아데노관련 바이러스 벡터
WO2020099548A1 (en) * 2018-11-15 2020-05-22 Esteve Pharmaceuticals, S.A. Animal model of mucopolysaccharidoses type iva
HUE060952T2 (hu) * 2018-12-18 2023-05-28 Neuway Pharma Gmbh Újszerû gyógyszer-adagolási rendszer és annak elõállítására szolgáló módszer
WO2020127678A1 (en) * 2018-12-20 2020-06-25 Esteve Pharmaceuticals, S.A. Recombinant vectors for the long term treatment of mucchopolysacharidosis
WO2021231491A1 (en) * 2020-05-11 2021-11-18 University Of Florida Research Foundation, Incorporated Disease correction by delivery of aav8 vectors expressing codon optimized naglu
CN111718947B (zh) * 2020-06-18 2022-08-23 舒泰神（北京）生物制药股份有限公司 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途
US20230323326A1 (en) * 2020-08-28 2023-10-12 Jcr Pharmaceuticals Co., Ltd. Mutant of alpha-N-Acetylglucosaminidase

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8105575B2 (en) * 2006-10-10 2012-01-31 Viromed Co., Ltd. Expression vectors with improved safety
EP2394667A1 (en) * 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
WO2012012742A2 (en) 2010-07-22 2012-01-26 Shire Human Genetic Therapies, Inc. Crystal structure of human alpha-n-acetylglucosaminidase
US20130039888A1 (en) * 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
JP6329483B2 (ja) * 2011-10-12 2018-05-23 シナジェバ・バイオファーマ・コーポレイションＳｙｎａｇｅｖａ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐ． 組換えヒトｎａｇｌｕタンパク質およびその利用
CN102618578A (zh) * 2012-03-16 2012-08-01 中国人民解放军第四军医大学 用于促进汉坦病毒融合蛋白g2s0.7有效提呈的重组腺病毒高表达载体
DE18200782T1 (de) * 2012-04-02 2021-10-21 Modernatx, Inc. Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen
DK3137497T3 (da) * 2014-05-02 2021-07-12 Genzyme Corp Aav-vektorer til retinal- og cns-genterapi
JP2020530307A (ja) * 2017-06-30 2020-10-22 インティマ・バイオサイエンス，インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
CN109762831B (zh) * 2019-01-24 2021-04-27 北京瑞希罕见病基因治疗技术研究所 用于治疗3a型粘多糖贮积症的基因药物构建体

Also Published As

Publication number Publication date
BR112016025819A2 (pt) 2018-07-03
TW201625793A (zh) 2016-07-16
AU2015261519A1 (en) 2016-11-24
RU2016149206A3 (pt-PT) 2019-04-25
AR100419A1 (es) 2016-10-05
US20170088859A1 (en) 2017-03-30
IL248538A0 (en) 2016-12-29
UA123985C2 (uk) 2021-07-07
PT3143146T (pt) 2020-08-27
EP3143146B1 (en) 2020-05-20
IL248538B (en) 2021-01-31
KR20170026358A (ko) 2017-03-08
MY183472A (en) 2021-02-19
SG11201609314XA (en) 2016-12-29
SA516380290B1 (ar) 2021-05-17
PH12016502248A1 (en) 2017-02-27
NZ726316A (en) 2023-07-28
CN107002095B (zh) 2021-03-02
CN107002095A (zh) 2017-08-01
RU2016149206A (ru) 2018-06-14
AU2015261519B2 (en) 2021-08-05
ES2823948T3 (es) 2021-05-10
CA2947468A1 (en) 2015-11-19
KR102446169B1 (ko) 2022-09-22
AU2015261519A2 (en) 2017-02-02
JP6634073B2 (ja) 2020-01-22
TWI701332B (zh) 2020-08-11
JP2017523802A (ja) 2017-08-24
RU2718248C2 (ru) 2020-03-31
US11149285B2 (en) 2021-10-19
WO2015173308A1 (en) 2015-11-19
MX2016014773A (es) 2017-05-04
CA2947468C (en) 2022-10-25
EP3143146A1 (en) 2017-03-22

Similar Documents

Publication Publication Date Title
US11149285B2 (en) Adenoassociated virus vectors for the treatment of lysosomal storage disorders
JP6312771B2 (ja) 神経代謝性疾患についての遺伝子治療
Motas et al. CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)
US10617771B2 (en) Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
US9279132B2 (en) Vectors and sequences for the treatment of diseases
US20180110877A1 (en) DUAL AAV VECTOR SYSTEM FOR CRISPR/Cas9 MEDIATED CORRECTION OF HUMAN DISEASE
US11338046B2 (en) Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
Motas Gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (hunter syndrome)
TW202338086A (zh) 有用於治療異染性白質失養症之組成物

Legal Events

Date Code Title Description
B25D Requested change of name of applicant approved

Owner name: UNIVERSITAT AUTONOMA DE BARCELONA (ES) ; ESTEVE PHARMACEUTICALS, S.A. (ES)

Owner name: UNIVERSITAT AUTONOMA DE BARCELONA (ES) ; ESTEVE PH

B25G Requested change of headquarter approved

Owner name: UNIVERSITAT AUTONOMA DE BARCELONA (ES) ; ESTEVE PHARMACEUTICALS, S.A. (ES)

Owner name: UNIVERSITAT AUTONOMA DE BARCELONA (ES) ; ESTEVE PH

B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]

Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI NO 10196/2001, QUE MODIFICOU A LEI NO 9279/96, A CONCESSAO DA PATENTE ESTA CONDICIONADA A ANUENCIA PREVIA DA ANVISA. CONSIDERANDO A APROVACAO DOS TERMOS DO PARECER NO 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL NO 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVIDENCIAS CABIVEIS.

B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B06A Patent application procedure suspended [chapter 6.1 patent gazette]
B09A Decision: intention to grant [chapter 9.1 patent gazette]
B16A Patent or certificate of addition of invention granted [chapter 16.1 patent gazette]

Free format text: PRAZO DE VALIDADE: 20 (VINTE) ANOS CONTADOS A PARTIR DE 13/05/2015, OBSERVADAS AS CONDICOES LEGAIS