MY201938A - Methods and compositions for modulating splicing - Google Patents

Methods and compositions for modulating splicing

Info

Publication number
MY201938A
MY201938A MYPI2020000611A MYPI2020000611A MY201938A MY 201938 A MY201938 A MY 201938A MY PI2020000611 A MYPI2020000611 A MY PI2020000611A MY PI2020000611 A MYPI2020000611 A MY PI2020000611A MY 201938 A MY201938 A MY 201938A
Authority
MY
Malaysia
Prior art keywords
methods
compositions
splicing
modulating splicing
mrna
Prior art date
Application number
MYPI2020000611A
Other languages
English (en)
Inventor
Michael LUZZIO
Kathleen Mccarthy
William Haney
Original Assignee
Skyhawk 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.)
Filing date
Publication date
Application filed by Skyhawk Therapeutics Inc filed Critical Skyhawk Therapeutics Inc
Publication of MY201938A publication Critical patent/MY201938A/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D401/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
    • C07D401/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50—Pyridazines; Hydrogenated pyridazines
    • A61K31/501—Pyridazines; Hydrogenated pyridazines not condensed and containing further heterocyclic rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings
    • C07D403/10—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings linked by a carbon chain containing aromatic rings
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5383—1,4-Oxazines, e.g. morpholine ortho- or peri-condensed with heterocyclic ring systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • 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
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/04—Antineoplastic agents specific for metastasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D209/00—Heterocyclic compounds containing five-membered rings, condensed with other rings, with one nitrogen atom as the only ring hetero atom
    • C07D209/02—Heterocyclic compounds containing five-membered rings, condensed with other rings, with one nitrogen atom as the only ring hetero atom condensed with one carbocyclic ring
    • C07D209/52—Heterocyclic compounds containing five-membered rings, condensed with other rings, with one nitrogen atom as the only ring hetero atom condensed with one carbocyclic ring condensed with a ring other than six-membered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing three or more hetero rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D451/00—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof
    • C07D451/02—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D451/00—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof
    • C07D451/02—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof
    • C07D451/04—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof with hetero atoms directly attached in position 3 of the 8-azabicyclo [3.2.1] octane or in position 7 of the 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D451/00—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof
    • C07D451/02—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof
    • C07D451/04—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing not further condensed 8-azabicyclo [3.2.1] octane or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane; Cyclic acetals thereof with hetero atoms directly attached in position 3 of the 8-azabicyclo [3.2.1] octane or in position 7 of the 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring system
    • C07D451/06—Oxygen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D451/00—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof
    • C07D451/14—Heterocyclic compounds containing 8-azabicyclo [3.2.1] octane, 9-azabicyclo [3.3.1] nonane, or 3-oxa-9-azatricyclo [3.3.1.0<2,4>] nonane ring systems, e.g. tropane or granatane alkaloids, scopolamine; Cyclic acetals thereof containing 9-azabicyclo [3.3.1] nonane ring systems, e.g. granatane, 2-aza-adamantane; Cyclic acetals thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/08—Bridged systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/10—Spiro-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/08—Bridged systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
    • C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
    • C07D491/08—Bridged systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D498/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D498/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D498/08—Bridged systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified A

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Genetics & Genomics (AREA)
  • Hematology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Oncology (AREA)
  • Obesity (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Urology & Nephrology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
MYPI2020000611A 2017-08-04 2018-08-03 Methods and compositions for modulating splicing MY201938A (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762541202P 2017-08-04 2017-08-04
US201762562927P 2017-09-25 2017-09-25
US201762562948P 2017-09-25 2017-09-25
PCT/US2018/045282 WO2019028440A1 (en) 2017-08-04 2018-08-03 METHODS AND COMPOSITIONS FOR MODULATING SPLICING

Publications (1)

Publication Number Publication Date
MY201938A true MY201938A (en) 2024-03-25

Family

ID=65233135

Family Applications (1)

Application Number Title Priority Date Filing Date
MYPI2020000611A MY201938A (en) 2017-08-04 2018-08-03 Methods and compositions for modulating splicing

Country Status (15)

Country Link
US (8) US11162101B2 (OSRAM)
EP (2) EP3661509A4 (OSRAM)
JP (2) JP7312749B2 (OSRAM)
KR (2) KR102636384B1 (OSRAM)
CN (2) CN111194215B (OSRAM)
AU (2) AU2018309187C1 (OSRAM)
CA (1) CA3072205A1 (OSRAM)
GB (2) GB2579747B8 (OSRAM)
IL (2) IL273741B (OSRAM)
MX (2) MX2020001425A (OSRAM)
MY (1) MY201938A (OSRAM)
RU (1) RU2020105929A (OSRAM)
SG (2) SG11202000980QA (OSRAM)
WO (1) WO2019028440A1 (OSRAM)
ZA (2) ZA202000695B (OSRAM)

Families Citing this family (70)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102488323B1 (ko) 2015-12-10 2023-01-12 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 또는 개선을 위한 조성물
JP2019535789A (ja) 2016-11-28 2019-12-12 ピーティーシー セラピューティクス，インコーポレーテッド Ｒｎａスプライシングを調節する方法
KR20200033249A (ko) 2017-06-05 2020-03-27 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 화합물
JP2020523365A (ja) 2017-06-14 2020-08-06 ピーティーシー セラピューティクス，インコーポレーテッド Ｒｎａスプライシングを改変する方法
MX2019015578A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
CN111194215B (zh) 2017-08-04 2024-03-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
US12103926B2 (en) 2018-03-27 2024-10-01 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
WO2019199972A1 (en) 2018-04-10 2019-10-17 Skyhawk Therapeutics, Inc. Compounds for the treatment of cancer
WO2020005877A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
AU2019294478B2 (en) 2018-06-27 2023-03-23 Ptc Therapeutics, Inc. Heterocyclic and heteroaryl compounds for treating Huntington's disease
EA202190581A1 (ru) 2018-08-20 2021-07-13 Рогкон, Инк. Антисмысловые олигонуклеотиды, нацеленные на scn2a, для лечения энцефалопатии scn1a
US20220090087A1 (en) * 2019-01-23 2022-03-24 The Florey Institute Of Neuroscience And Mental Health Antisense oligonucleotides targeting scn2a retained introns
CN113692402A (zh) * 2019-02-04 2021-11-23 斯基霍克疗法公司 用于调节剪接的方法和组合物
WO2020163409A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
WO2020163541A1 (en) * 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP2022523148A (ja) * 2019-02-05 2022-04-21 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
EP3920919A4 (en) * 2019-02-05 2023-03-29 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
WO2020163405A1 (en) 2019-02-05 2020-08-13 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
EP3921311A4 (en) * 2019-02-06 2022-11-09 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
JP7603595B2 (ja) 2019-02-06 2024-12-20 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
CN113840602A (zh) * 2019-03-15 2021-12-24 斯基霍克疗法公司 用于校正异常剪接的组合物和方法
CA3132936A1 (en) 2019-04-09 2020-10-15 Maria Luisa PINEDA Cancer-specific molecules and methods of use thereof
CN109988163A (zh) * 2019-04-15 2019-07-09 宁夏医科大学 一种异丙基托品醇异构体的制备分离方法
UY38687A (es) 2019-05-17 2023-05-15 Novartis Ag Inhibidores del inflamasoma nlrp3, composiciones, combinaciones de los mismos y métodos para su uso
US11129829B2 (en) * 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
WO2021014428A1 (en) 2019-07-25 2021-01-28 Novartis Ag Regulatable expression systems
WO2021071982A1 (en) * 2019-10-08 2021-04-15 Skyhawk Therapeutics, Inc. Compounds for modulating splicing
MX2022005254A (es) 2019-11-01 2022-06-29 Novartis Ag El uso de un modulador de empalme para un tratamiento que retrasa la progresion de la enfermedad de huntington.
CA3169697A1 (en) 2020-02-28 2021-09-02 Dominic Reynolds Thiophenyl derivatives useful for modulating nucleic acid splicing
BR112022017089A2 (pt) * 2020-02-28 2022-11-16 Remix Therapeutics Inc Derivados de piridazina para modular o splicing de ácido nucleico
EP4110459A1 (en) * 2020-02-28 2023-01-04 Remix Therapeutics Inc. Heterocyclic amides and their use for modulating splicing
WO2021174167A1 (en) 2020-02-28 2021-09-02 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2021207554A1 (en) 2020-04-08 2021-10-14 Remix Therapeutics Inc. Compounds and methods for modulating splicing
EP4132935A1 (en) 2020-04-08 2023-02-15 Remix Therapeutics Inc. Compounds and methods for modulating splicing
MX2022013856A (es) 2020-05-13 2023-04-05 Chdi Foundation Inc Moduladores htt para tratar la enfermedad de huntington.
JP2023532331A (ja) 2020-07-02 2023-07-27 リミックス セラピューティクス インコーポレイテッド 核酸をスプライシングするため及び増殖性疾患を処置するための修飾因子としての５－［５－（ピペリジン－４－イル）チエノ［３，２－ｃ］ピラゾール－２－イル］インダゾール誘導体及び関連する化合物並びにスプライシングを調節するための化合物及び方法
AU2021299515A1 (en) 2020-07-02 2023-02-02 Remix Therapeutics Inc. 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
WO2022032007A2 (en) * 2020-08-05 2022-02-10 Skyhawk Therapeutics, Inc. Process for the preparation of intermediates useful in the preparation of compounds that modulate splicing
CN116171153B (zh) * 2020-08-05 2025-11-21 斯基霍克疗法公司 用于调节剪接的组合物
WO2022031998A2 (en) * 2020-08-05 2022-02-10 Skyhawk Therapeutics, Inc. Process for the preparation of intermediates useful in the preparation of compounds that modulate splicing
CN114113621A (zh) * 2020-08-25 2022-03-01 张曼 尿液鸟嘌呤核苷酸结合蛋白亚基α-13及其多肽片段在烧伤中的应用
WO2022060944A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
WO2022060951A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
WO2022060943A1 (en) * 2020-09-16 2022-03-24 Skyhawk Therapeutics, Inc. Compositions for modulating splicing
US20240018522A1 (en) * 2020-10-26 2024-01-18 Remix Therapeutics Inc. Oligonucleotides useful for modulation of splicing
WO2022109022A1 (en) * 2020-11-17 2022-05-27 Skyhawk Therapeutics, Inc. Rna-targeting splicing modifiers for treatment of mda5-associated conditions and diseases
EP4395891A1 (en) 2021-08-30 2024-07-10 Remix Therapeutics Inc. Compounds and methods for modulating splicing
PE20251687A1 (es) 2021-08-30 2025-07-02 Remix Therapeutics Inc Compuestos y metodos para modular splicing
EP4396177A1 (en) 2021-08-30 2024-07-10 Remix Therapeutics Inc. Compounds and methods for modulating splicing
US20240400584A1 (en) 2021-08-30 2024-12-05 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2023034836A1 (en) 2021-08-30 2023-03-09 Remix Therapeutics Inc. Compounds and methods for modulating splicing
EP4416156A1 (en) 2021-10-13 2024-08-21 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
EP4416141A1 (en) 2021-10-13 2024-08-21 Remix Therapeutics Inc. Compounds and methods for modulating nucleic acid splicing
JP2024540477A (ja) 2021-11-17 2024-10-31 シーエイチディーアイ ファウンデーション，インコーポレーテッド ハンチントン病を治療するためのｈｔｔモジュレータとしてのｎ－（２ｈ－インダゾール－５－イル）ピラジン－２－カルボキサミド誘導体及び類似化合物
CN118786117A (zh) * 2021-11-18 2024-10-15 亚根达医疗公司 杂环酰胺及其使用方法
CN114225051B (zh) * 2021-12-16 2024-05-10 中国人民解放军空军军医大学 一种用于治疗银屑病的药物及其应用
US20250092065A1 (en) 2022-01-05 2025-03-20 Remix Therapeutics Inc. 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and related compounds as modulators for splicing nucleic acids and for the treatment of proliferative diseases
TW202346305A (zh) 2022-01-05 2023-12-01 美商雷密克斯醫療公司 用於調節剪切之化合物及方法
WO2023133229A2 (en) 2022-01-05 2023-07-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
CN114350813B (zh) * 2022-03-17 2022-08-02 广州达安临床检验中心有限公司 Tmem236基因在制备用于诊断直肠癌新辅助放疗抵抗标记物中的用途
CN114592006A (zh) * 2022-04-29 2022-06-07 昆明理工大学 Memo1基因的新用途
KR102470669B1 (ko) * 2022-06-03 2022-11-25 충남대학교 산학협력단 스플라이싱-스위치 올리고뉴클레오티드를 유효성분으로 포함하는 폴리프로모 1 유전자의 스플라이싱 조절용 조성물
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
UY40374A (es) 2022-08-03 2024-02-15 Novartis Ag Inhibidores de inflamasoma nlrp3
TW202432551A (zh) * 2022-10-26 2024-08-16 日商安斯泰來製藥股份有限公司 稠環嗒嗪衍生物
WO2024173719A1 (en) * 2023-02-15 2024-08-22 Ascidian Therapeutics, Inc. Htt trans-splicing molecules
WO2025247338A1 (zh) * 2024-05-30 2025-12-04 纽欧申医药(上海)有限公司 哒嗪环类化合物、其药物组合物及其应用
CN119569728B (zh) * 2024-12-02 2025-09-30 重庆飞因科生物科技有限公司 吡啶并吡咯类衍生物及其在制备富亮氨酸重复激酶2抑制剂中的应用

Family Cites Families (133)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4873192A (en) 1987-02-17 1989-10-10 The United States Of America As Represented By The Department Of Health And Human Services Process for site specific mutagenesis without phenotypic selection
NZ227684A (en) 1988-01-30 1991-07-26 Merck Sharp & Dohme Pyrazine, pyridazine, and pyrimidine derivatives and pharmaceutical compositions
GB9005737D0 (en) 1990-03-14 1990-05-09 Beecham Group Plc Novel compounds
FR2676444B1 (fr) 1991-05-16 1995-03-10 Sanofi Elf Nouveaux derives d'amino-3 pyridazines actifs sur le systeme nerveux central, procede de preparation et compositions pharmaceutiques en contenant.
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
JP4291143B2 (ja) 2001-08-31 2009-07-08 ジェン−プロウブ インコーポレイテッド 分析物ポリヌクレオチドを定量するためのアフィニティーシフトしたプローブ
US7135484B2 (en) 2002-08-14 2006-11-14 Abbott Laboratories Azabicyclic compounds are central nervous system active agents
DE60326894D1 (de) 2002-09-30 2009-05-07 Neurosearch As 1,4-Diazabicycloalkanderivate, deren Herstellung und Verwendung
US8014953B2 (en) 2003-08-08 2011-09-06 The Regents Of The University Of California RNA surveillance among curated proteins
US7399765B2 (en) 2003-09-19 2008-07-15 Abbott Laboratories Substituted diazabicycloalkane derivatives
US7160876B2 (en) 2003-12-22 2007-01-09 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
US7309699B2 (en) 2003-12-22 2007-12-18 Abbott Laboratories 3-Quinuclidinyl amino-substituted biaryl derivatives
US7655657B2 (en) 2003-12-22 2010-02-02 Abbott Laboratories Fused bicycloheterocycle substituted quinuclidine derivatives
US7309487B2 (en) 2004-02-09 2007-12-18 George Inana Methods and compositions for detecting and treating retinal diseases
PE20060437A1 (es) 2004-06-18 2006-06-08 Novartis Ag COMPUESTOS AZA-BICICLONONANOS COMO LIGANDOS COLINERGICOS DE nAChR
GB0415746D0 (en) 2004-07-14 2004-08-18 Novartis Ag Organic compounds
EP2380889B1 (en) 2004-10-07 2013-06-26 Merck Sharp & Dohme Corp. Thiazolyl MGLUR5 antagonists and methods for their use
MX2007004551A (es) 2004-10-18 2007-05-23 Amgen Inc Compuestos tiadiazol y metodos de uso.
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
WO2006081230A2 (en) 2005-01-26 2006-08-03 Schering Corporation 3-(indazol-5-yl)-(1,2, 4) triazine derivatives and related compounds as protein kinase inhibitors for the treatment of cancer
US20070078135A1 (en) 2005-04-18 2007-04-05 Neurogen Corporation Substituted heteroaryl CB1 antagonists
US7598052B2 (en) 2005-10-11 2009-10-06 The Regents Of The University Of Michigan Expression profile of thyroid cancer
US8603457B2 (en) 2005-12-02 2013-12-10 University Of Rochester Nonsense suppression and genetic codon alteration by targeted modification
US7834178B2 (en) 2006-03-01 2010-11-16 Bristol-Myers Squibb Company Triazine 11-beta hydroxysteroid dehydrogenase type 1 inhibitors
NZ573735A (en) 2006-05-19 2011-10-28 Abbott Lab Cns active fused bicycloheterocycle substituted azabicyclic alkane derivatives
ATE518862T1 (de) 2006-05-30 2011-08-15 Neurosearch As Neue 1,4-diaza-bicycloä3.2.2ünonyl-oxadiazolyl- derivate und deren medizinische verwendung
US8314119B2 (en) 2006-11-06 2012-11-20 Abbvie Inc. Azaadamantane derivatives and methods of use
US8343941B2 (en) 2007-03-30 2013-01-01 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
US8283116B1 (en) 2007-06-22 2012-10-09 Ptc Therapeutics, Inc. Methods of screening for compounds for treating spinal muscular atrophy using SMN mRNA translation regulation
DE102007032507A1 (de) * 2007-07-12 2009-04-02 Merck Patent Gmbh Pyridazinonderivate
HUE028662T2 (en) 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
EP2280010B1 (en) 2007-11-21 2012-12-19 Abbott Laboratories Biaryl substituted azabicyclic alkane derivatives as nicotinic acetylcholine receptor activity modulators
JP5177219B2 (ja) 2008-03-18 2013-04-03 コニカミノルタホールディングス株式会社 電気化学的表示素子
WO2009151546A2 (en) 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
US8133724B2 (en) 2008-09-17 2012-03-13 University Of Maryland, Baltimore Human androgen receptor alternative splice variants as biomarkers and therapeutic targets
ES2567047T3 (es) 2008-12-23 2016-04-19 Abbvie Inc. Derivados de pirimidina anti-virales
CN102482677B (zh) 2009-03-16 2017-10-17 库尔纳公司 通过抑制nrf2的天然反义转录物治疗核因子(红细胞衍生2)‑样2(nrf2)相关疾病
CN102625840A (zh) 2009-04-10 2012-08-01 肌肉学研究协会 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法
SI3305302T1 (sl) 2009-06-17 2018-12-31 Biogen Ma Inc. Sestave in metode za modulacijo združevanja smn2 pri subjektu
TWI558398B (zh) 2009-09-22 2016-11-21 諾華公司 菸鹼乙醯膽鹼受體α７活化劑之用途
EP2305679A1 (en) 2009-09-28 2011-04-06 GenKyoTex SA Pyrazoline dione derivatives as nadph oxidase inhibitors
AR079724A1 (es) * 2010-01-20 2012-02-15 Abbott Lab Metodos para el tratamiento del dolor
HUE028640T2 (en) 2010-02-05 2016-12-28 Heptares Therapeutics Ltd 1,2,4-triazin-4-amine derivative
AR081626A1 (es) 2010-04-23 2012-10-10 Cytokinetics Inc Compuestos amino-piridazinicos, composiciones farmaceuticas que los contienen y uso de los mismos para tratar trastornos musculares cardiacos y esqueleticos
US9133123B2 (en) 2010-04-23 2015-09-15 Cytokinetics, Inc. Certain amino-pyridines and amino-triazines, compositions thereof, and methods for their use
AR081331A1 (es) 2010-04-23 2012-08-08 Cytokinetics Inc Amino- pirimidinas composiciones de las mismas y metodos para el uso de los mismos
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
JP6069661B2 (ja) 2010-06-24 2017-02-01 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 種々のアミロイドβペプチドアロフォームレベルの調節における組成物及びその使用
KR101764952B1 (ko) 2010-07-29 2017-08-03 리겔 파마슈티칼스, 인크. Ａｍｐｋ-활성화 헤테로시클릭 화합물 및 그의 사용 방법
US20130071369A1 (en) 2011-03-14 2013-03-21 Nse Products, Inc. Oral Formulations For Promoting Cellular Purification
US9464065B2 (en) 2011-03-24 2016-10-11 The Scripps Research Institute Compounds and methods for inducing chondrogenesis
WO2013019938A1 (en) 2011-08-02 2013-02-07 The Brigham And Women's Hospital, Inc. Pyridazine derivatives as eaat2 activators
US10252984B2 (en) 2011-10-27 2019-04-09 Mayo Foundation For Medical Education And Research Inhibiting G protein coupled receptor 6 kinase polypeptides
CA2861609C (en) 2011-12-30 2021-02-16 Ptc Therapeutics, Inc. Compounds for treating spinal muscular atrophy
CA2862084C (en) 2012-01-26 2021-05-11 Ptc Therapeutics, Inc. 1h-isochromen-1-one derivatives and compositions thereof for treating spinal muscular atrophy
WO2013117693A1 (en) 2012-02-10 2013-08-15 Glaxosmithkline Intellectual Property Development Limited Pde4 inhibitor for treating huntington's disease
PL2812004T3 (pl) 2012-02-10 2019-01-31 Ptc Therapeutics, Inc. Związki do leczenia rdzeniowego zaniku mięśni
EA029155B1 (ru) 2012-03-01 2018-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Соединения для лечения спинальной мышечной атрофии
MX358514B (es) 2012-03-23 2018-08-24 Ptc Therapeutics Inc Compuestos para tratar la atrofia muscular espinal.
US8729263B2 (en) * 2012-08-13 2014-05-20 Novartis Ag 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions
AU2013204200B2 (en) 2012-10-11 2016-10-20 Brandeis University Treatment of amyotrophic lateral sclerosis
WO2014062686A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for modulating c9orf72 expression
JP2016508159A (ja) * 2013-01-15 2016-03-17 ノバルティス アーゲー アルファ７ニコチン性アセチルコリン受容体アゴニストの使用
CN105263492B (zh) * 2013-01-15 2018-04-10 诺华有限公司 α7烟碱型乙酰胆碱受体激动剂在治疗发作性睡病中的应用
KR101879920B1 (ko) * 2013-01-15 2018-07-18 노파르티스 아게 알파 7 니코틴성 아세틸콜린 수용체 작용물질의 용도
ITMI20130063A1 (it) * 2013-01-17 2014-07-18 Valier Alberto Dispositivo manuale per la raccolta dal suolo di frutti o altri oggetti
US9040712B2 (en) 2013-01-23 2015-05-26 Novartis Ag Thiadiazole analogs thereof and methods for treating SMN-deficiency-related-conditions
KR102314290B1 (ko) 2013-06-25 2021-10-21 에프. 호프만-라 로슈 아게 척수성 근위축증을 치료하기 위한 화합물
MX383686B (es) 2013-07-31 2025-03-14 Novartis Ag Derivados de piridazina 1,4-disustituida, y su uso para el tratamiento de afecciones relacionadas con la deficiencia de smn.
RU2016109324A (ru) 2013-08-19 2017-09-26 Ф. Хоффманн-Ля Рош Аг Способ скрининга
CA2922567A1 (en) 2013-08-26 2015-03-05 Purdue Pharma L.P. Azaspiro[4.5]decane derivatives and use thereof
WO2015048792A1 (en) 2013-09-30 2015-04-02 The Regents Of The University Of California Identification of structurally similar small molecules that enhance therapeutic exon skipping
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
ES2850999T3 (es) 2014-03-14 2021-09-02 Raqualia Pharma Inc Derivados de azaespiro como antagonistas de TRPM8
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
AU2015272127A1 (en) 2014-06-10 2017-01-19 Erasmus University Medical Center Rotterdam Methods for characterizing alternatively or aberrantly spliced mRNA isoforms
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
WO2016037039A1 (en) 2014-09-04 2016-03-10 Rutgers, The State University Of New Jersey Compositions for increasing survival of motor neuron protein (smn) levels in target cells and methods of use thereof for the treatment of spinal muscular atrophy
US20170334977A1 (en) 2014-10-20 2017-11-23 The Brigham And Women's Hospital, Inc. Targeting Apolipoprotein E (APOE) in Neurologic Disease
US10053697B1 (en) 2015-01-09 2018-08-21 The Board Of Trustees Of The Leland Stanford Junior University Programmable alternative splicing devices and uses thereof
CA2973873A1 (en) 2015-01-20 2016-07-28 Merial, Inc. Anthelmintic compounds, compositions and method of using thereof
EP3053577A1 (en) 2015-02-09 2016-08-10 F. Hoffmann-La Roche AG Compounds for the treatment of cancer
JP6884102B2 (ja) 2015-02-09 2021-06-09 エフ・ホフマン−ラ・ロシュ・アクチェンゲゼルシャフト がんの治療のための化合物
US10668171B2 (en) * 2015-05-30 2020-06-02 Ptc Therapeutics, Inc. Methods for modulating RNA splicing
CN107922396B (zh) 2015-07-20 2022-08-05 建新公司 集落刺激因子-1受体(csf-1r)抑制剂
WO2017041036A1 (en) 2015-09-02 2017-03-09 Pathways Bioscience, Llc Compositions and methods for treatment or prevention of oral mucositis
RU2018112749A (ru) 2015-09-24 2019-10-24 ГлаксоСмитКлайн Интеллекчуал Проперти (N 2) Лимитед Модуляторы индоламин-2,3-диоксигеназы
WO2017062751A1 (en) 2015-10-08 2017-04-13 The Regents Of The University Of California Compounds and methods for promoting stress resistance
KR102422625B1 (ko) 2015-10-09 2022-07-20 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
EP3183347A4 (en) * 2015-10-17 2018-04-18 Lifesplice Pharma LLC Splice modulating oligonucleotides and methods of use thereof
WO2017080967A1 (en) 2015-11-12 2017-05-18 F. Hoffmann-La Roche Ag Compositions for treating spinal muscular atrophy
US20180344680A1 (en) 2015-12-08 2018-12-06 The Arizona Board Of Regents On Behalf Of The University Of Arizona Compositions and methods for treatment, amelioration, and prevention of diabetes-related skin ulcers
KR102488323B1 (ko) * 2015-12-10 2023-01-12 피티씨 테라퓨틱스, 인크. 헌팅턴병 치료 또는 개선을 위한 조성물
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
JP2019505579A (ja) 2015-12-23 2019-02-28 ムーンショット ファーマ エルエルシー 免疫応答を誘導するための方法
US10787433B2 (en) 2016-01-15 2020-09-29 The Brigham And Women's Hospital, Inc. Pyridazine derivatives as EAAT2 activators
EP3411080A4 (en) 2016-02-01 2019-08-14 Arrakis Therapeutics, Inc. COMPOUNDS AND METHODS FOR TREATING RNA-MEDIATED DISEASES
MX2018010109A (es) 2016-02-23 2018-12-17 Univ Indiana Res & Tech Corp Terapias de combinacion para tratamiento de atrofia muscular espinal.
EP3423158B1 (en) 2016-02-24 2023-11-15 The Rockefeller University Embryonic cell-based therapeutic candidate screening systems, models for huntington's disease and uses thereof
US11066456B2 (en) 2016-02-25 2021-07-20 Washington University Compositions comprising TREM2 and methods of use thereof
US20170268066A1 (en) 2016-03-15 2017-09-21 Chalmers Ventures Ab Cancer biomarkers
CN108779093B (zh) 2016-03-29 2021-03-09 默克专利股份公司 作为丙酮酸脱氢酶激酶的抑制剂的n1-(3,3,3-三氟-2-羟基-2-甲基丙酰基)-哌啶衍生物
CA3019847A1 (en) 2016-04-04 2017-10-12 University Of Florida Research Foundation, Incorporated Manipulation of eif3 to modulate repeat associated non-atg (ran) translation
US10577360B2 (en) 2016-04-12 2020-03-03 Esteve Pharmaceuticals, S.A. Arylamide derivatives having multimodal activity against pain
LT3368069T (lt) 2016-06-13 2020-12-10 Scholar Rock, Inc. Miostatino inhibitorių naudojimas, ir kombinuotas gydymas
MX2018016038A (es) * 2016-07-01 2019-05-13 Arrakis Therapeutics Inc Compuestos y metodos para modular la funcion del acido ribonucleico (arn).
JP2019535789A (ja) 2016-11-28 2019-12-12 ピーティーシー セラピューティクス，インコーポレーテッド Ｒｎａスプライシングを調節する方法
CN114432318A (zh) 2017-02-20 2022-05-06 国立大学法人京都大学 用于起因于剪接异常的遗传性疾病的药物组合物及治疗方法
EP3628055A4 (en) 2017-05-01 2021-06-09 Thomas Jefferson University Systems-level analysis of tcga cancers reveals disease trna fragmentation patterns and associations with messenger rnas and repeat
JP2020523365A (ja) * 2017-06-14 2020-08-06 ピーティーシー セラピューティクス，インコーポレーテッド Ｒｎａスプライシングを改変する方法
MX2019015580A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
MX2019015578A (es) 2017-06-28 2020-07-28 Ptc Therapeutics Inc Metodos para tratar la enfermedad de huntington.
CN111194215B (zh) 2017-08-04 2024-03-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
WO2019057123A1 (zh) 2017-09-20 2019-03-28 杭州英创医药科技有限公司 作为ido抑制剂和/或ido-hdac双重抑制剂的多环化合物
CN111212834B (zh) 2017-10-12 2024-01-19 锐新医药公司 作为变构shp2抑制剂的吡啶、吡嗪和三嗪化合物
JP7381476B2 (ja) 2017-10-24 2023-11-15 サンガモ セラピューティクス， インコーポレイテッド 希少疾患の処置のための方法および組成物
EP3479845A1 (en) 2017-11-06 2019-05-08 Stalicla S.A. Challenge test for diagnosing subtype of autism spectrum disease
US10905707B2 (en) 2017-11-10 2021-02-02 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders
US12103926B2 (en) 2018-03-27 2024-10-01 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
WO2019199972A1 (en) 2018-04-10 2019-10-17 Skyhawk Therapeutics, Inc. Compounds for the treatment of cancer
WO2020005877A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heteroaryl compounds for treating huntington's disease
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
AU2019294478B2 (en) 2018-06-27 2023-03-23 Ptc Therapeutics, Inc. Heterocyclic and heteroaryl compounds for treating Huntington's disease
MX2020014116A (es) 2018-06-27 2021-06-15 Reborna Biosciences Inc Agente profilactico o terapeutico para atrofia muscular espinal.
WO2020028814A1 (en) 2018-08-03 2020-02-06 H. Lee Moffitt Cancer Center And Research Institute, Inc. Allosteric inhibitor of wee1 kinase
TW202028183A (zh) 2018-10-10 2020-08-01 大陸商江蘇豪森藥業集團有限公司 含氮雜芳類衍生物調節劑、其製備方法和應用
US11129829B2 (en) * 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
EP4110459A1 (en) 2020-02-28 2023-01-04 Remix Therapeutics Inc. Heterocyclic amides and their use for modulating splicing
WO2021174167A1 (en) 2020-02-28 2021-09-02 Remix Therapeutics Inc. Compounds and methods for modulating splicing
BR112022017089A2 (pt) 2020-02-28 2022-11-16 Remix Therapeutics Inc Derivados de piridazina para modular o splicing de ácido nucleico
CA3169697A1 (en) 2020-02-28 2021-09-02 Dominic Reynolds Thiophenyl derivatives useful for modulating nucleic acid splicing
EP4132935A1 (en) 2020-04-08 2023-02-15 Remix Therapeutics Inc. Compounds and methods for modulating splicing
WO2021207554A1 (en) 2020-04-08 2021-10-14 Remix Therapeutics Inc. Compounds and methods for modulating splicing

Also Published As

Publication number Publication date
KR20200037844A (ko) 2020-04-09
KR102636384B1 (ko) 2024-02-14
JP7135026B2 (ja) 2022-09-12
US11008572B2 (en) 2021-05-18
JP2020169171A (ja) 2020-10-15
JP7312749B2 (ja) 2023-07-21
AU2018309187B2 (en) 2021-04-01
CN111194215B (zh) 2024-03-01
GB2579747A (en) 2020-07-01
EP3689863A1 (en) 2020-08-05
US20200224199A1 (en) 2020-07-16
GB202003042D0 (en) 2020-04-15
GB2587457B (en) 2022-06-01
CA3072205A1 (en) 2019-02-07
GB2579747B8 (en) 2022-10-05
MX2020001425A (es) 2020-08-06
US20220403383A1 (en) 2022-12-22
WO2019028440A1 (en) 2019-02-07
IL272413A (en) 2020-03-31
KR20200038545A (ko) 2020-04-13
US20200247799A1 (en) 2020-08-06
US11162101B2 (en) 2021-11-02
EP3661509A1 (en) 2020-06-10
GB2579747A8 (en) 2022-10-05
RU2020105929A (ru) 2021-09-06
AU2020200699C1 (en) 2021-08-12
CN111499615B (zh) 2024-02-02
AU2018309187A1 (en) 2020-02-20
US11326165B1 (en) 2022-05-10
ZA202000695B (en) 2022-07-27
SG10202002990XA (en) 2020-05-28
BR122020005073A2 (pt) 2020-10-13
US11603531B1 (en) 2023-03-14
AU2020200699A1 (en) 2020-02-20
CN111499615A (zh) 2020-08-07
US11434489B1 (en) 2022-09-06
US20200277289A1 (en) 2020-09-03
CN111194215A (zh) 2020-05-22
US11021708B2 (en) 2021-06-01
BR112020002270A2 (pt) 2020-07-28
ZA202004932B (en) 2022-12-21
US11091475B2 (en) 2021-08-17
AU2018309187C1 (en) 2023-09-07
IL273741A (en) 2020-05-31
SG11202000980QA (en) 2020-02-27
GB2587457A (en) 2021-03-31
MX2020005748A (es) 2020-08-20
IL273741B (en) 2022-09-01
GB2579747B (en) 2022-06-08
IL272413B (en) 2022-09-01
JP2020530487A (ja) 2020-10-22
US20210171519A1 (en) 2021-06-10
EP3661509A4 (en) 2021-01-13
AU2020200699B2 (en) 2021-03-25
US20230059865A1 (en) 2023-02-23
KR102636383B1 (ko) 2024-02-14
GB202008364D0 (en) 2020-07-15

Similar Documents

Publication Publication Date Title
MX2020005748A (es) Metodos y composiciones para modular el empalme.
PH12020550523A1 (en) Modulators of the integrated stress pathway
PH12020550524A1 (en) Modulators of the integrated stress pathway
EA201992497A1 (ru) МОДУЛЯТОРЫ K-Ras
AU2019268063A1 (en) Compositions for modulating sod-1 expression
MX2023009561A (es) Moduladores de profarmacos de la via de estres integrada.
MX2021013193A (es) Cicloalquilos sustituidos como moduladores de la vía integrada del estrés.
PH12018502329A1 (en) Modulators of the integrated stress pathway
AU2017261336A1 (en) Modulators of the integrated stress pathway
AU2017260374A1 (en) Modulators of the integrated stress pathway
EP4534092A3 (en) Compositions and methods for modulating pkk expression
EP3978610A3 (en) Compositions for modulating ataxin 2 expression
MX2018016038A (es) Compuestos y metodos para modular la funcion del acido ribonucleico (arn).
PH12019502646A1 (en) Pyrazole magl inhibitors
MX2017012738A (es) Inhibidores de indolamina 2.3-dioxigenasa (ido) para el tratamiento de cancer.
MX2015011374A (es) Inhibidores de indolamina 2,3-dioxigenasa (ido).
EP4616910A3 (en) Pcna inhibitors
EP4347605A4 (en) COMPOUNDS, COMPOSITIONS AND METHODS FOR MODULATING FERROPTOSIS AND TREATING EXCITOTOXIC DISORDERS
MX2018004674A (es) Moduladores de interacción de sestrina-gator2 y sus usos.
MX2017015923A (es) Inhibidores de indolamina-2,3-dioxigenasa (ido).
MX2020012990A (es) Formulaciones de tegavivint y compuestos relacionados.
ZA202310843B (en) Chromene derivatives as inhibitors of tcr-nck interaction
MX2023001558A (es) Composiciones para modular el corte y empalme.
PH12017501601A1 (en) Use of isolated fractions of mastic gum for treating optic neuropathy
MX2019009199A (es) Composiciones y métodos para modular ppp2r1a.