PL3717646T3 - Leczenie antysensowe zespołu angelmana - Google Patents

Leczenie antysensowe zespołu angelmana

Info

Publication number
PL3717646T3
PL3717646T3 PL18883102.8T PL18883102T PL3717646T3 PL 3717646 T3 PL3717646 T3 PL 3717646T3 PL 18883102 T PL18883102 T PL 18883102T PL 3717646 T3 PL3717646 T3 PL 3717646T3
Authority
PL
Poland
Prior art keywords
angelman syndrome
antisense treatment
antisense
treatment
angelman
Prior art date
Application number
PL18883102.8T
Other languages
English (en)
Inventor
Scott Victor DINDOT
Original Assignee
The Texas A&M University System
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 The Texas A&M University System filed Critical The Texas A&M University System
Publication of PL3717646T3 publication Critical patent/PL3717646T3/pl

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
    • C12N15/11—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • 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
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
PL18883102.8T 2017-12-01 2018-11-30 Leczenie antysensowe zespołu angelmana PL3717646T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762593431P 2017-12-01 2017-12-01
US201862676034P 2018-05-24 2018-05-24
PCT/US2018/063416 WO2019109001A1 (en) 2017-12-01 2018-11-30 Angelman syndrome antisense treatment

Publications (1)

Publication Number Publication Date
PL3717646T3 true PL3717646T3 (pl) 2025-10-27

Family

ID=66664231

Family Applications (1)

Application Number Title Priority Date Filing Date
PL18883102.8T PL3717646T3 (pl) 2017-12-01 2018-11-30 Leczenie antysensowe zespołu angelmana

Country Status (27)

Country Link
US (5) US11198869B2 (pl)
EP (2) EP3717646B1 (pl)
JP (4) JP7297320B2 (pl)
KR (2) KR102723989B1 (pl)
CN (2) CN111433361B (pl)
AU (1) AU2018375807B2 (pl)
BR (1) BR112020009431A2 (pl)
CA (1) CA3079755A1 (pl)
CL (1) CL2020001344A1 (pl)
CO (1) CO2020006361A2 (pl)
DK (1) DK3717646T3 (pl)
ES (1) ES3032315T3 (pl)
FI (1) FI3717646T3 (pl)
HR (1) HRP20250804T1 (pl)
HU (1) HUE072645T2 (pl)
IL (1) IL274146B2 (pl)
LT (1) LT3717646T (pl)
MX (2) MX2020005680A (pl)
PE (1) PE20211238A1 (pl)
PL (1) PL3717646T3 (pl)
PT (1) PT3717646T (pl)
RS (1) RS66986B1 (pl)
RU (1) RU2020121752A (pl)
SG (1) SG11202003596WA (pl)
SI (1) SI3717646T1 (pl)
SM (1) SMT202500334T1 (pl)
WO (1) WO2019109001A1 (pl)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HK1258434A1 (zh) 2015-11-12 2019-11-08 F. Hoffmann-La Roche Ag 来自灵长类物种的标准化神经元细胞测定试验
EP3758714A4 (en) * 2018-02-27 2021-12-01 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR TREATMENT OF ANGELMAN'S SYNDROME
TW202536179A (zh) 2019-03-29 2025-09-16 美商Ionis製藥公司 用於調節ube3a-ats之化合物及方法
TW202216996A (zh) * 2020-06-29 2022-05-01 美商Ionis製藥公司 調節ｐｌｐ１之化合物及方法
CA3197664A1 (en) * 2020-10-06 2022-04-14 University Of Maryland, Baltimore Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides
CN119384504A (zh) * 2022-03-07 2025-01-28 康涅狄格大学 靶向SNORD115位置以恢复快乐木偶综合征中父系UBE3A基因表达的shRNA
KR20250107262A (ko) 2023-01-31 2025-07-11 후지필름 가부시키가이샤 경화성 조성물, 경화물, 광학 재료, 회절 광학 소자 및 화합물
WO2025100980A1 (ko) * 2023-11-08 2025-05-15 한국과학기술원 Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도
WO2025217466A1 (en) * 2024-04-11 2025-10-16 Ultragenyx Pharmaceutical Inc. Dosing regimens for antisense treatment of angelman syndrome
CN121992041A (zh) * 2024-11-06 2026-05-08 中国科学院脑科学与智能技术卓越创新中心 靶向ube3a等位基因建立天使综合征动物模型的方法和应用

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2823959B2 (ja) 1991-10-24 1998-11-11 アイシス・ファーマシューティカルス・インコーポレーテッド 改良された取り込みおよびその他の性質を持つ誘導化オリゴヌクレオチド
CN100523215C (zh) * 2000-12-01 2009-08-05 马普科技促进协会 介导rna干涉的小rna分子
EP1799859B1 (en) * 2004-09-17 2014-07-02 Isis Pharmaceuticals, Inc. Enhanced antisense oligonucleotides
EP1676834A1 (en) 2004-12-30 2006-07-05 Sanofi-Aventis Deutschland GmbH Fused bicyclic carboxamide derivates for use as CXCR2 inhibitors in the treatment of inflammation
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
US8178503B2 (en) * 2006-03-03 2012-05-15 International Business Machines Corporation Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes
US20080299659A1 (en) * 2007-03-02 2008-12-04 Nastech Pharmaceutical Company Inc. Nucleic acid compounds for inhibiting apob gene expression and uses thereof
US8580756B2 (en) 2007-03-22 2013-11-12 Santaris Pharma A/S Short oligomer antagonist compounds for the modulation of target mRNA
US9290534B2 (en) 2008-04-04 2016-03-22 Ionis Pharmaceuticals, Inc. Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside
BRPI0923225A2 (pt) 2008-12-02 2016-10-04 Chiralgen Ltd metodo para sintese de acidos nucleicos modificados no atomo de fosforo
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
RU2013150331A (ru) 2011-04-20 2015-05-27 Рош Гликарт Аг СПОСОБ И УСТРОЙСТВА ДЛЯ рН-ЗАВИСИМОГО ПРОХОЖДЕНИЯ ГЕМАТОЭНЦЕФАЛИЧЕСКОГО БАРЬЕРА
US10023861B2 (en) 2011-08-29 2018-07-17 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
CN104540946A (zh) * 2012-05-16 2015-04-22 Rana医疗有限公司 用于调节utrn表达的组合物和方法
WO2013173637A1 (en) * 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
ES2809199T3 (es) * 2012-06-25 2021-03-03 Ionis Pharmaceuticals Inc Modulación de la expresión de UBE3A-ATS
DK2872147T3 (da) 2012-07-13 2023-02-20 Wave Life Sciences Ltd Fremgangsmåde til fremstilling af kirale oligonukleotider
EP3094728B1 (en) 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Chiral design
EP3221329A1 (en) 2014-11-19 2017-09-27 Roche Innovation Center Copenhagen A/S Lna gapmer oligonucleotides comprising chiral phosphorothioate linkages
WO2016086104A1 (en) 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
DK3234131T3 (da) 2014-12-16 2020-06-29 Roche Innovation Ct Copenhagen As Fremgangsmåde til screening af kiral toksicitet
CA2975525A1 (en) * 2015-02-04 2016-08-11 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
EP3286318A2 (en) * 2015-04-22 2018-02-28 Mina Therapeutics Limited Sarna compositions and methods of use
HK1258434A1 (zh) 2015-11-12 2019-11-08 F. Hoffmann-La Roche Ag 来自灵长类物种的标准化神经元细胞测定试验
WO2017194498A1 (en) 2016-05-12 2017-11-16 Roche Innovation Center Copenhagen A/S Enhanced coupling of stereodefined oxazaphospholidine phosphoramidite monomers to nucleoside or oligonucleotide
JP6636673B2 (ja) 2016-10-04 2020-01-29 メルク・シャープ・アンド・ドーム・コーポレーションＭｅｒｃｋ Ｓｈａｒｐ ＆ Ｄｏｈｍｅ Ｃｏｒｐ． ＳＴＩＮＧアゴニストとしてのベンゾ［ｂ］チオフェン化合物
KR20190135494A (ko) 2017-03-29 2019-12-06 로슈 이노베이션 센터 코펜하겐 에이/에스 입체한정된 포스포로티오에이트 올리고뉴클레오티드의 제조를 위한 직교 보호기
JP2020524718A (ja) 2017-06-22 2020-08-20 キュラデブ・ファーマ・リミテッドＣｕｒａｄｅｖ Ｐｈａｒｍａ Ｌｉｍｉｔｅｄ ヒトｓｔｉｎｇの複素環式小分子調節因子

Also Published As

Publication number Publication date
CN118291458A (zh) 2024-07-05
KR102967878B1 (ko) 2026-05-26
US11739328B2 (en) 2023-08-29
JP7601441B2 (ja) 2024-12-17
JP2021505129A (ja) 2021-02-18
RU2020121752A (ru) 2021-12-30
JP7897667B1 (ja) 2026-07-30
JP2025041632A (ja) 2025-03-26
KR20200094152A (ko) 2020-08-06
US20230112629A1 (en) 2023-04-13
CL2020001344A1 (es) 2020-09-21
MX2025009778A (es) 2025-09-02
AU2018375807B2 (en) 2025-04-10
EP3717646B1 (en) 2025-04-23
CN111433361B (zh) 2024-03-29
HRP20250804T1 (hr) 2025-09-12
SMT202500334T1 (it) 2025-11-10
PT3717646T (pt) 2025-07-28
SI3717646T1 (sl) 2025-08-29
ES3032315T3 (en) 2025-07-17
PE20211238A1 (es) 2021-07-09
US20230332152A1 (en) 2023-10-19
US12180480B2 (en) 2024-12-31
US11198869B2 (en) 2021-12-14
US20200370046A1 (en) 2020-11-26
FI3717646T3 (fi) 2025-07-17
BR112020009431A2 (pt) 2020-10-13
CO2020006361A2 (es) 2020-08-10
WO2019109001A1 (en) 2019-06-06
IL274146B1 (en) 2024-07-01
EP4328306A3 (en) 2025-05-21
HUE072645T2 (hu) 2025-12-28
EP4328306A2 (en) 2024-02-28
AU2018375807A1 (en) 2020-05-07
JP7863362B2 (ja) 2026-05-21
JP2023123517A (ja) 2023-09-05
KR20240161203A (ko) 2024-11-12
SG11202003596WA (en) 2020-05-28
LT3717646T (lt) 2025-06-10
US11685920B2 (en) 2023-06-27
EP3717646A4 (en) 2021-12-01
RS66986B1 (sr) 2025-07-31
IL274146A (en) 2020-06-30
JP7297320B2 (ja) 2023-06-26
KR102723989B1 (ko) 2024-11-01
IL274146B2 (en) 2024-11-01
US20220090075A1 (en) 2022-03-24
US20250066786A1 (en) 2025-02-27
CN111433361A (zh) 2020-07-17
DK3717646T3 (en) 2025-05-19
EP3717646A1 (en) 2020-10-07
MX2020005680A (es) 2020-08-20
CA3079755A1 (en) 2019-06-06

Similar Documents

Publication Publication Date Title
IL274146A (en) Antisense therapy in Engelmann syndrome
IL253730A0 (en) Antisense tau oligomers and their uses
GB201904293D0 (en) Mass correction
PL3977996T3 (pl) Leczenie zespołu tourette’a
IL272098B1 (en) Dihydrooxadiazinones
GB201502137D0 (en) Treatment
IL273394A (en) RNAI variant
GB201522243D0 (en) Treatment
GB201820521D0 (en) n
GB201701986D0 (en) n
GB201517432D0 (en) Splint
GB201513826D0 (en) Pasteurisation
PL3393415T3 (pl) Bandaż
GB201512582D0 (en) Wound treatment
GB201508841D0 (en) Treatment
GB201503008D0 (en) Treatment
GB201813378D0 (en) n
GB201718985D0 (en) Treatment
GB2567679B (en) Curved devices
GB201713650D0 (en) Treatment
GB201711677D0 (en) Combination treatment
GB201706955D0 (en) Treatment
GB201704949D0 (en) Combination treatment
GB201716645D0 (en) Non-freid monoliths
GB201710601D0 (en) Redcloud BB2