AR122072A1 - Moduladores htt para tratar la enfermedad de huntington - Google Patents

Moduladores htt para tratar la enfermedad de huntington

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Publication number
AR122072A1
AR122072A1 ARP210101306A ARP210101306A AR122072A1 AR 122072 A1 AR122072 A1 AR 122072A1 AR P210101306 A ARP210101306 A AR P210101306A AR P210101306 A ARP210101306 A AR P210101306A AR 122072 A1 AR122072 A1 AR 122072A1
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Argentina
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alkyl
optionally substituted
heterocyclyl
halo
haloalkyl
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ARP210101306A
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English (en)
Inventor
Celia Dominguez
Elizabeth M Doherty
Longbin Liu
Matthew Lee
Mark Stuart Chambers
Karine Fabienne Malagu
Perla Breccia
Alan F Haughan
Huw D Vater
Andrew J Stott
William R K Esmieu
Stephen John Webster
De Pol Amanda J Van
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Chdi Foundation Inc
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Publication of AR122072A1 publication Critical patent/AR122072A1/es

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    • 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
    • A61K31/4965—Non-condensed pyrazines
    • A61K31/497—Non-condensed pyrazines containing further heterocyclic rings
    • 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
    • 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
    • A61K31/4985—Pyrazines or piperazines 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
    • 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
    • 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
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/506—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic 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
    • A61K31/53—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with three nitrogens as the only ring hetero atoms, e.g. chlorazanil, melamine
    • 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
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C07D403/04—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 directly linked by a ring-member-to-ring-member bond
    • 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
    • C07D413/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D413/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings
    • C07D413/04—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D413/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D413/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
    • 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
    • C07D487/04—Ortho-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
    • C07D487/10—Spiro-condensed 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
    • C07D498/04—Ortho-condensed systems
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Neurology (AREA)
  • Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Electrotherapy Devices (AREA)
  • Magnetic Treatment Devices (AREA)
  • Hydrogenated Pyridines (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)

Abstract

En el presente documento se proporcionan ciertos compuestos útiles como moduladores de HTT. Tales compuestos son útiles en el tratamiento de la enfermedad de Huntington. Reivindicación 1: Un compuesto de fórmula (1) o un análogo enriquecido isotópicamente, una sal, profármaco, tautómero, estereoisómero o una mezcla de estereoisómeros farmacéuticamente aceptables del mismo, en donde: X¹, X², X³, y X⁴ son CR⁴ o N, en donde al menos dos pero no más de tres de X¹, X², X³, y X⁴ son N; cada R⁴ es independientemente hidrógeno, halo, hidroxi, alquiloC₁₋₆, haloalquiloC₁₋₆, o alcoxiC₁₋₆; Y¹ es CR⁵ o N; R⁵ es hidrógeno, ciano, halo, hidroxi, alquiloC₁₋₆, haloalquiloC₁₋₆, alquiloC₁₋₆tio, alcoxiC₁₋₆, haloalcoxiC₁₋₆, heterociclilo, -NH₂, -NHR¹⁷, o -N(R¹⁷)₂, y opcionalmente sustituido en un átomo de nitrógeno disponible con alquiloC₁₋₆ o haloalquiloC₁₋₆; Y² está ausente, CR⁶ o N; R⁶ es hidrógeno, ciano, halo, hidroxi, alquiloC₁₋₆, haloalquiloC₁₋₆, alquiloC₁₋₆tio, alcoxiC₁₋₆, haloalcoxiC₁₋₆, heterociclilo, -NH₂, -NHR¹⁷, o -N(R¹⁷)₂, y opcionalmente sustituido en un átomo de nitrógeno disponible con alquiloC₁₋₆ o haloalquiloC₁₋₆; y Y³ es CR³ o N; R³ es hidrógeno, ciano, halo, hidroxi, alquiloC₁₋₆, haloalquiloC₁₋₆, alquiloC₁₋₆tio, alcoxiC₁₋₆, haloalcoxiC₁₋₆, heterociclilo, -NH₂, -NHR¹⁷, o -N(R¹⁷)₂, y opcionalmente sustituido en un átomo de nitrógeno disponible con alquiloC₁₋₆ o haloalquiloC₁₋₆; cada R¹⁷ es independientemente alquiloC₁₋₄, o dos R¹⁷ se unen, con cualquier átomo que intervenga, para formar un heterociclilo de 3 a 6 miembros; cada uno de Z¹ y Z² es C o N; anillo A y anillo B juntos forman un heteroarilo bicíclico de 9 ó 10 miembros que contiene 1 a 3 átomos de nitrógeno del anillo; Anillo B contiene 1 a 3 heteroátomos independientemente seleccionados de N, O, y S, y está opcionalmente sustituido en los átomos de carbono disponibles con 1 a 3 sustituyentes seleccionados independientemente de halo, hidroxi, alquiloC₁₋₆, haloalquiloC₁₋₆, alcoxiC₁₋₆, y haloalcoxiC₁₋₆, y opcionalmente sustituido en un átomo de nitrógeno disponible con alquiloC₁₋₆ o haloalquiloC₁₋₆; R¹ es -L¹-R¹¹, en donde L¹ es -O-, -S-, -S(O)-, -S(O)₂-, -N(R¹²)-, -alquilenoC₁₋₃-, -O-alquilenoC₁₋₃-, -N(R¹²)-alquilenoC₁₋₃-, o ausente, y R¹¹ es alquiniloC₂₋₆, cicloalquiloC₃₋₁₀, ariloC₆₋₁₀, heteroarilo, o heterociclilo, en donde R¹¹ está opcionalmente sustituido con 1 a 4 grupos R¹³; R¹² es hidrógeno o alquiloC₁₋₆; cada R¹³ se selecciona independientemente de halo, ciano, hidroxi, alquiloC₁₋₆ opcionalmente sustituido con R¹⁶, haloalquiloC₁₋₆, hidroxialquiloC₁₋₆, cicloalquiloC₃₋₁₀ opcionalmente sustituido con R¹⁶, cicloalquiloC₃₋₁₀-alquiloC₁₋₆ opcionalmente sustituido con R¹⁶, ariloC₆₋₁₀ opcionalmente sustituido con R¹⁶, ariloC₆₋₁₀-alquiloC₁₋₆ opcionalmente sustituido con R¹⁶, heteroarilo opcionalmente sustituido con R¹⁶, heteroaril-alquiloC₁₋₆ opcionalmente sustituido con R¹⁶, heterociclilo opcionalmente sustituido con R¹⁶, heterociclilo-alquiloC₁₋₆ opcionalmente sustituido con R¹⁶, OR¹⁴, -NH₂, -NHR¹⁴, -N(R¹⁴)₂, -alquilenoC₁₋₆-NH₂, -alquilenoC₁₋₆-NHR¹⁴, -alquilenoC₁₋₆-N(R¹⁴)₂, -C(O)R¹⁵, -C(O)OR¹⁵, -C(O)NHR¹⁵, -C(O)N(alquiloC₁₋₄)R¹⁵, -S(O)₂R¹⁵, -S(O)R¹⁵, -NHC(O)R¹⁵, -N(alquiloC₁₋₄)C(O)R¹⁵, -NHS(O)R¹⁵, -N(alquiloC₁₋₄)S(O)R¹⁵, -NHS(O)₂R¹⁵, y -N(alquiloC₁₋₄)S(O)₂R¹⁵; cada R¹⁴ se selecciona independientemente de alquiloC₁₋₆, cicloalquiloC₃₋₁₀, ariloC₆₋₁₀, heteroarilo, y heterociclilo; y cada R¹⁴ está opcionalmente sustituido con uno a seis halo, alquiloC₁₋₃, alcoxiC₁₋₃, cicloalquiloC₃₋₁₀, o -NHSO₂-aril-N(CH₃)₂; cada R¹⁵ es independientemente hidrógeno, -OH, alquiloC₁₋₆, haloalquiloC₁₋₆, cicloalquiloC₃₋₁₀, ariloC₆₋₁₀, heteroarilo, o heterociclilo; cada R¹⁶ es independientemente halo, ciano, hidroxi, -NH₂, -NHR²¹, -N(R²¹)₂, alquiloC₁₋₆, haloalquiloC₁₋₆, OR²¹, o cicloalquiloC₃₋₁₀; cada R²¹ se selecciona independientemente de alquiloC₁₋₆, cicloalquiloC₃₋₁₀, ariloC₆₋₁₀, heteroarilo, y heterociclilo, y cada R²¹ está opcionalmente sustituido con uno a seis halo o alcoxiC₁₋₃; y R² es hidrógeno o alquiloC₁₋₆.
ARP210101306A 2020-05-13 2021-05-12 Moduladores htt para tratar la enfermedad de huntington AR122072A1 (es)

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US (1) US11806346B2 (es)
EP (1) EP4149937A1 (es)
JP (1) JP2023532623A (es)
KR (1) KR20230028269A (es)
CN (1) CN116997548A (es)
AR (1) AR122072A1 (es)
AU (1) AU2021272972A1 (es)
BR (1) BR112022023025A2 (es)
CA (1) CA3182912A1 (es)
IL (1) IL298109A (es)
MX (1) MX2022013856A (es)
TW (1) TW202208358A (es)
WO (1) WO2021231571A1 (es)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3133100A1 (en) 2019-03-19 2020-09-24 Boehringer Ingelheim Animal Health USA Inc. Anthelmintic aza-benzothiophene and aza-benzofuran compounds
JP2023528822A (ja) 2020-05-29 2023-07-06 ベーリンガー インゲルハイム アニマル ヘルス ユーエスエイ インコーポレイテッド 駆虫性複素環式化合物
WO2023073641A1 (en) 2021-11-01 2023-05-04 Boehringer Ingelheim Vetmedica Gmbh Anthelmintic pyrrolopyridazine compounds
CN118510769A (zh) * 2021-11-17 2024-08-16 Chdi基金会股份有限公司 用于治疗亨廷顿病的htt调节剂
WO2023225244A1 (en) * 2022-05-20 2023-11-23 Biogen Ma Inc. Heterocyclic compounds for treating huntington's disease
WO2024025389A1 (ko) * 2022-07-29 2024-02-01 아이디언스 주식회사 프탈라지논 유도체의 중간체의 제조방법
WO2024026061A1 (en) * 2022-07-29 2024-02-01 Biogen Ma Inc. Compounds for treating huntington's disease

Family Cites Families (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2007524596A (ja) 2003-02-28 2007-08-30 トランスフォーム・ファーマシューティカルズ・インコーポレイテッド 共結晶医薬組成物
CN100418965C (zh) * 2003-08-15 2008-09-17 万有制药株式会社 咪唑并吡啶衍生物
PE20070589A1 (es) * 2005-10-04 2007-06-22 Aventis Pharma Inc Compuestos de pirimidina amida como inhibidores de pgds
EP2147914B1 (en) * 2007-04-24 2014-06-04 Shionogi&Co., Ltd. Aminodihydrothiazine derivatives substituted with cyclic groups
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
WO2014209841A2 (en) 2013-06-25 2014-12-31 F. Hoffmann-La Roche Ag Compounds for treating spinal muscular atrophy
EP3107905B1 (en) * 2014-02-20 2018-09-19 University of Notre Dame du Lac Selective matrix metalloproteinase inhibitors
KR102565544B1 (ko) 2014-06-25 2023-08-10 에프. 호프만-라 로슈 아게 척수성 근위축증을 치료하기 위한 이미다조[1,2-a]피라진-1일-벤즈아마이드 화합물
US10457662B2 (en) 2015-05-20 2019-10-29 The Regents Of The University Of Michigan Substituted amides for treating and preventing cancer
IL281633B (en) 2015-12-10 2022-07-01 Ptc Therapeutics Inc Methods for treating Huntington's disease
JP2018083799A (ja) 2016-11-15 2018-05-31 バイオエレクトロン テクノロジー コーポレイション ２−置換アミノ−ナフト［１，２−ｄ］イミダゾール−５−オン化合物またはその製薬学上許容される塩
EP3544435A4 (en) 2016-11-28 2020-11-04 PTC Therapeutics, Inc. RNA SPLICE MODULATION PROCESSES
EP3634953B1 (en) 2017-06-05 2024-01-03 PTC Therapeutics, Inc. Compounds for treating huntington's disease
CN111372611A (zh) 2017-06-14 2020-07-03 Ptc医疗公司 修饰rna剪接的方法
CA3067591A1 (en) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methods for treating huntington's disease
US11395822B2 (en) 2017-06-28 2022-07-26 Ptc Therapeutics, Inc. Methods for treating Huntington's disease
CN111499615B (zh) 2017-08-04 2024-02-02 斯基霍克疗法公司 用于调节剪接的方法和组合物
CA3094703A1 (en) 2018-03-27 2019-10-03 Ptc Therapeutics, Inc. Compounds for treating huntington's disease
US11685746B2 (en) 2018-06-27 2023-06-27 Ptc Therapeutics, Inc. Heteroaryl compounds for treating Huntington's disease
WO2020005873A1 (en) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Heterocyclic and heteroaryl compounds for treating huntington's disease
BR112020026534A2 (pt) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. Compostos de heteroarila para o tratamento da doença de huntington
EP3920916A4 (en) 2019-02-04 2022-09-28 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
EP3920917A4 (en) 2019-02-04 2022-09-28 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
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
EP3920910A4 (en) 2019-02-04 2022-11-09 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
EP3920915A4 (en) 2019-02-05 2022-10-05 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
KR20210135240A (ko) 2019-02-05 2021-11-12 스카이호크 테라퓨틱스, 인코포레이티드 스플라이싱을 조절하는 방법 및 조성물
CN114126613A (zh) 2019-02-05 2022-03-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
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
JP2022519312A (ja) 2019-02-05 2022-03-22 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
EP3921311A4 (en) 2019-02-06 2022-11-09 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATING SPLICE
EP3920928A4 (en) 2019-02-06 2022-09-28 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
PE20220139A1 (es) 2019-02-13 2022-01-27 Ptc Therapeutics Inc Compuestos de tioeno[3,2-b] piridin-7-amina para tratar la disautonomia familiar
EP3924050A1 (en) 2019-02-13 2021-12-22 PTC Therapeutics, Inc. Pyrrolo[2,3-d]pyrimidine compounds for treating familial dysautonomia
JP2022533120A (ja) 2019-05-13 2022-07-21 ピーティーシー セラピューティクス， インコーポレイテッド ハンチントン病を処置するための化合物
WO2021007378A1 (en) 2019-07-11 2021-01-14 Ptc Therapeutics, Inc. Compounds for use in treating huntington's disease
CN114650822A (zh) 2019-11-01 2022-06-21 诺华股份有限公司 剪接调节子用于减慢亨廷顿氏病进展的治疗的用途
AU2021228767A1 (en) 2020-02-28 2022-09-29 Remix Therapeutics Inc. Heterocyclic amides and their use for modulating splicing
US20230142338A1 (en) 2020-02-28 2023-05-11 Remix Therapeutics Inc. Thiophenyl derivatives useful for modulating nucleic acid splicing
CA3169667A1 (en) 2020-02-28 2021-09-02 Dominic Reynolds Pyridazine derivatives for modulating nucleic acid splicing
WO2021174167A1 (en) 2020-02-28 2021-09-02 Remix Therapeutics Inc. Compounds and methods for modulating splicing
KR20220167298A (ko) 2020-04-08 2022-12-20 레믹스 테라퓨틱스 인크. 스플라이싱을 조절하기 위한 화합물 및 방법
JP2023520925A (ja) 2020-04-08 2023-05-22 リミックス セラピューティクス インコーポレイテッド スプライシングを調節するための化合物及び方法
IL297044A (en) 2020-04-09 2022-12-01 Ptc Therapeutics Inc Compounds for the treatment of Huntington's disease

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US20220409615A1 (en) 2022-12-29
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