EP3952923A4 - Thérapies géniques pour troubles lysosomaux - Google Patents

Thérapies géniques pour troubles lysosomaux Download PDF

Info

Publication number: EP3952923A4
Authority: EP; European Patent Office
Prior art keywords: gene therapies; lysosomal disorders; lysosomal; disorders; therapies
Prior art date: 2019-04-10
Legal status (The legal status 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 Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.): Withdrawn

Application number

EP20787456.1A

Other languages

German (de)

English (en)

Other versions

EP3952923A1 (fr

Inventor

Asa Abeliovich

Laura Heckman

Herve Rhinn

Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)

Prevail Therapeutics Inc

Original Assignee

Prevail Therapeutics 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.)

2019-04-10

Filing date

2020-04-10

Publication date

2023-10-18

2020-04-10 Application filed by Prevail Therapeutics Inc filed Critical Prevail Therapeutics Inc

2020-04-10 Priority claimed from PCT/US2020/027658 external-priority patent/WO2020210615A1/fr

2022-02-16 Publication of EP3952923A1 publication Critical patent/EP3952923A1/fr

2023-10-18 Publication of EP3952923A4 publication Critical patent/EP3952923A4/fr

Status Withdrawn legal-status Critical Current

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
- 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
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/475—Growth factors; Growth regulators
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-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
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-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
- C12N15/1137—Non-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 against enzymes
- 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)
- C12N9/2405—Glucanases
- C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
- C12N9/2411—Amylases
- C12N9/2414—Alpha-amylase (3.2.1.1.)
- 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)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2207/00—Modified animals
- A01K2207/20—Animals treated with compounds which are neither proteins nor nucleic acids
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
- A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- 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
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/31—Combination therapy
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted 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
- 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

Landscapes

Health & Medical Sciences (AREA)
Life Sciences & Earth Sciences (AREA)
Chemical & Material Sciences (AREA)
Genetics & Genomics (AREA)
Engineering & Computer Science (AREA)
Organic Chemistry (AREA)
Bioinformatics & Cheminformatics (AREA)
General Health & Medical Sciences (AREA)
Zoology (AREA)
Biomedical Technology (AREA)
Wood Science & Technology (AREA)
Biotechnology (AREA)
Molecular Biology (AREA)
General Engineering & Computer Science (AREA)
Medicinal Chemistry (AREA)
Biochemistry (AREA)
Animal Behavior & Ethology (AREA)
Veterinary Medicine (AREA)
Public Health (AREA)
Pharmacology & Pharmacy (AREA)
Biophysics (AREA)
Microbiology (AREA)
Epidemiology (AREA)
Physics & Mathematics (AREA)
Plant Pathology (AREA)
Neurology (AREA)
Neurosurgery (AREA)
Virology (AREA)
Proteomics, Peptides & Aminoacids (AREA)
Toxicology (AREA)
Gastroenterology & Hepatology (AREA)
Chemical Kinetics & Catalysis (AREA)
General Chemical & Material Sciences (AREA)
Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
Psychology (AREA)
Immunology (AREA)
Orthopedic Medicine & Surgery (AREA)
Dermatology (AREA)
Cell Biology (AREA)
Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

EP20787456.1A 2019-04-10 2020-04-10 Thérapies géniques pour troubles lysosomaux Withdrawn EP3952923A4 (fr)

Applications Claiming Priority (10)

Application Number	Priority Date	Filing Date	Title
US201962831846P	2019-04-10	2019-04-10
US201962831856P	2019-04-10	2019-04-10
US201962831840P	2019-04-10	2019-04-10
US201962832223P	2019-04-10	2019-04-10
US201962934450P	2019-11-12	2019-11-12
US201962954089P	2019-12-27	2019-12-27
US202062960471P	2020-01-13	2020-01-13
US202062988665P	2020-03-12	2020-03-12
US202062990246P	2020-03-16	2020-03-16
PCT/US2020/027658 WO2020210615A1 (fr)	2019-04-10	2020-04-10	Thérapies géniques pour troubles lysosomaux

Publications (2)

Publication Number	Publication Date
EP3952923A1 EP3952923A1 (fr)	2022-02-16
EP3952923A4 true EP3952923A4 (fr)	2023-10-18

Family

ID=79032950

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
EP20787456.1A Withdrawn EP3952923A4 (fr)	2019-04-10	2020-04-10	Thérapies géniques pour troubles lysosomaux

Country Status (6)

Country	Link
EP (1)	EP3952923A4 (fr)
JP (1)	JP2022527015A (fr)
KR (1)	KR20210150487A (fr)
CN (1)	CN114025806A (fr)
IL (1)	IL286582A (fr)
MX (1)	MX2021012467A (fr)

Citations (1)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
WO2019028306A2 (fr) *	2017-08-03	2019-02-07	Voyager Therapeutics, Inc.	Compositions et procédés permettant l'administration de virus adéno-associés

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
US10648001B2 (en) *	2012-07-11	2020-05-12	Sangamo Therapeutics, Inc.	Method of treating mucopolysaccharidosis type I or II
HK1222093A1 (zh) *	2013-05-15	2017-06-23	明尼苏达大学董事会	腺相关病毒介导的基因向中枢神经系统转移
EP3292206B8 (fr) *	2015-05-07	2022-02-09	Takeda Pharmaceutical Company Limited	Glucocérébrosidase therapie genique pour la maladie de parkinson

2020
- 2020-04-10 EP EP20787456.1A patent/EP3952923A4/fr not_active Withdrawn
- 2020-04-10 JP JP2021559976A patent/JP2022527015A/ja active Pending
- 2020-04-10 KR KR1020217036238A patent/KR20210150487A/ko not_active Withdrawn
- 2020-04-10 CN CN202080027713.0A patent/CN114025806A/zh not_active Withdrawn
- 2020-04-10 MX MX2021012467A patent/MX2021012467A/es unknown
2021
- 2021-09-22 IL IL286582A patent/IL286582A/en unknown

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
WO2019028306A2 (fr) *	2017-08-03	2019-02-07	Voyager Therapeutics, Inc.	Compositions et procédés permettant l'administration de virus adéno-associés

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
AGNIESZKA CIESIELSKA ET AL: "Cerebral Infusion of AAV9 Vector-encoding Non-self Proteins Can Elicit Cell-mediated Immune Responses", MOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPY, vol. 21, no. 1, 1 January 2013 (2013-01-01), US, pages 158 - 166, XP055700835, ISSN: 1525-0016, DOI: 10.1038/mt.2012.167 *
ANDREW E ARRANT ET AL: "Neurobiology of Disease Progranulin Gene Therapy Improves Lysosomal Dysfunction and Microglial Pathology Associated with Frontotemporal Dementia and Neuronal Ceroid Lipofuscinosis", 28 February 2018 (2018-02-28), XP055700833, Retrieved from the Internet <URL:https://www.jneurosci.org/content/early/2018/01/29/JNEUROSCI.3081-17.2018/tab-article-info?versioned=true> [retrieved on 20200603] *
BENJAMIN E. DEVERMAN ET AL: "Gene therapy for neurological disorders: progress and prospects", NATURE REVIEWS DRUG DISCOVERY, vol. 17, no. 9, 10 August 2018 (2018-08-10), GB, pages 641 - 659, XP055609922, ISSN: 1474-1776, DOI: 10.1038/nrd.2018.110 *
LLUIS SAMARANCH ET AL: "AAV9-mediated Expression of a Non-self Protein in Nonhuman Primate Central Nervous System Triggers Widespread Neuroinflammation Driven by Antigen-presenting Cell Transduction", MOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPY, vol. 22, no. 2, 1 February 2014 (2014-02-01), US, pages 329 - 337, XP055701101, ISSN: 1525-0016, DOI: 10.1038/mt.2013.266 *

Also Published As

Publication number	Publication date
MX2021012467A (es)	2021-12-10
CN114025806A (zh)	2022-02-08
KR20210150487A (ko)	2021-12-10
IL286582A (en)	2021-12-01
EP3952923A1 (fr)	2022-02-16
JP2022527015A (ja)	2022-05-27

Publication	Publication Date	Title
EP3692075A4 (fr)	2022-02-09	Thérapies géniques pour troubles lysosomaux
EP3692151A4 (fr)	2021-07-14	Thérapies géniques pour troubles lysosomaux
EP3692158A4 (fr)	2021-08-04	Thérapies géniques pour troubles lysosomiaux
IL286901A (en)	2021-10-31	Gene therapies for lysosomal disorders
EP3773572A4 (fr)	2022-01-05	Rimegepant pour troubles liés au cgrp
IL286903A (en)	2021-10-31	Gene therapies for lysosomal disorders
EP3525735A4 (fr)	2020-03-11	Ultrasons thérapeutiques pour troubles oculaires
EP3409771A4 (fr)	2019-10-23	Ensemble de couplage pour enzymes thérapeutiques
EP3701030A4 (fr)	2022-04-20	Thérapies géniques pour maladie neurodégénérative
IL286825A (en)	2021-10-31	Gene therapy for eye pathologies
EP3638316A4 (fr)	2021-03-24	Thérapie génique pour troubles oculaires
EP3423109A4 (fr)	2019-08-14	Thérapie pour démence frontotemporale
EP3442547A4 (fr)	2019-11-27	Thérapies à base de probiotiques pour troubles du développement et autres troubles neurologiques
EP4070215A4 (fr)	2022-11-23	Techniques pour fournir un apprentissage automatique fédéré sécurisé
EP3887396A4 (fr)	2022-09-07	Thérapies géniques pour maladie neurodégénérative
EP3801721A4 (fr)	2022-03-09	Ensembles interface pour inhalothérapie
EP4149452A4 (fr)	2024-05-01	Polythérapie de troubles hépatiques
EP3893988A4 (fr)	2022-09-07	Thérapie de neuromodulation pour troubles auto-immuns et inflammatoires
EP3717653A4 (fr)	2021-12-01	Thérapie génique pour la mucopolysaccharidose de type iiia
EP3781588A4 (fr)	2022-03-30	Biomarqueur sanguin pour troubles gastro-intestinaux éosinophiliques
EP3589738A4 (fr)	2021-01-06	Thérapie génique pour troubles oculaires
IL287186A (en)	2021-12-01	Monitoring gene therapy
EP2918290A4 (fr)	2016-05-18	Agent thérapeutique pour troubles kératoconjonctifs
EP3952923A4 (fr)	2023-10-18	Thérapies géniques pour troubles lysosomaux
IL275613A (en)	2020-08-31	Gene therapy for eosinophilic disorders

Legal Events

Date	Code	Title	Description
2020-10-16	STAA	Information on the status of an ep patent application or granted ep patent	Free format text: STATUS: THE INTERNATIONAL PUBLICATION HAS BEEN MADE
2022-01-14	PUAI	Public reference made under article 153(3) epc to a published international application that has entered the european phase	Free format text: ORIGINAL CODE: 0009012
2022-01-14	STAA	Information on the status of an ep patent application or granted ep patent	Free format text: STATUS: REQUEST FOR EXAMINATION WAS MADE
2022-02-16	17P	Request for examination filed	Effective date: 20211011
2022-02-16	AK	Designated contracting states	Kind code of ref document: A1 Designated state(s): AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
2022-07-20	DAV	Request for validation of the european patent (deleted)
2022-07-20	DAX	Request for extension of the european patent (deleted)
2023-07-12	P01	Opt-out of the competence of the unified patent court (upc) registered	Effective date: 20230530
2023-09-11	REG	Reference to a national code	Ref country code: DE Ref legal event code: R079 Free format text: PREVIOUS MAIN CLASS: A61K0048000000 Ipc: C07K0014475000
2023-10-18	A4	Supplementary search report drawn up and despatched	Effective date: 20230915
2023-10-18	RIC1	Information provided on ipc code assigned before grant	Ipc: C12N 15/861 20060101ALI20230911BHEP Ipc: A61K 48/00 20060101ALI20230911BHEP Ipc: C07K 14/475 20060101AFI20230911BHEP
2024-08-15	STAA	Information on the status of an ep patent application or granted ep patent	Free format text: STATUS: THE APPLICATION IS DEEMED TO BE WITHDRAWN
2024-09-18	18D	Application deemed to be withdrawn	Effective date: 20240416