MX2024012094A - Metodos y compuestos para modular la enfermedad de huntington - Google Patents

Metodos y compuestos para modular la enfermedad de huntington

Info

Publication number
MX2024012094A
MX2024012094A MX2024012094A MX2024012094A MX2024012094A MX 2024012094 A MX2024012094 A MX 2024012094A MX 2024012094 A MX2024012094 A MX 2024012094A MX 2024012094 A MX2024012094 A MX 2024012094A MX 2024012094 A MX2024012094 A MX 2024012094A
Authority
MX
Mexico
Prior art keywords
disease
methods
compounds
huntington
modulating
Prior art date
Application number
MX2024012094A
Other languages
English (en)
Spanish (es)
Inventor
Chengzhi Zhang
Abhijit Bhat
Jarod Waybright
Hannah Nourie
Fei Yang
Original Assignee
Design 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 Design Therapeutics Inc filed Critical Design Therapeutics Inc
Publication of MX2024012094A publication Critical patent/MX2024012094A/es

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D495/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
    • C07D495/12—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms in which the condensed system contains three hetero rings
    • C07D495/14—Ortho-condensed systems
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/545—Heterocyclic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/55—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/595—Polyamides, e.g. nylon
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • 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
    • 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
    • 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
    • C07D495/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
    • C07D495/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
    • C07D495/04—Ortho-condensed systems

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychiatry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MX2024012094A 2022-04-01 2024-09-30 Metodos y compuestos para modular la enfermedad de huntington MX2024012094A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263326625P 2022-04-01 2022-04-01
US202363482670P 2023-02-01 2023-02-01
PCT/US2023/017201 WO2023192642A2 (en) 2022-04-01 2023-03-31 Methods and compounds for modulating huntington's disease

Publications (1)

Publication Number Publication Date
MX2024012094A true MX2024012094A (es) 2024-11-08

Family

ID=88203356

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024012094A MX2024012094A (es) 2022-04-01 2024-09-30 Metodos y compuestos para modular la enfermedad de huntington

Country Status (11)

Country Link
US (2) US20240058460A1 (https=)
EP (1) EP4504339A2 (https=)
JP (1) JP2025512877A (https=)
KR (1) KR20250016096A (https=)
CN (1) CN119584961A (https=)
AU (1) AU2023241739A1 (https=)
CA (1) CA3247116A1 (https=)
IL (1) IL315965A (https=)
MX (1) MX2024012094A (https=)
TW (1) TW202404642A (https=)
WO (1) WO2023192642A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025122764A1 (en) * 2023-12-08 2025-06-12 Design Therapeutics, Inc. Methods and compounds for modulating inherited genetic diseases
WO2026015815A1 (en) * 2024-07-12 2026-01-15 Design Therapeutics, Inc. Methods of treating haploinsufficiency disorders

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10517877B2 (en) * 2016-03-30 2019-12-31 Wisconsin Alumni Research Foundation Compounds and methods for modulating frataxin expression
EP4234549A1 (en) * 2018-05-22 2023-08-30 Design Therapeutics, Inc. Methods and compounds for the treatment of genetic disease
CN121045206A (zh) * 2020-02-03 2025-12-02 设计治疗公司 用于治疗遗传疾病的方法和化合物
WO2022126000A1 (en) * 2020-12-11 2022-06-16 Design Therapeutics, Inc. Methods and compounds for modulating myotonic dystrophy 1

Also Published As

Publication number Publication date
US20240042046A1 (en) 2024-02-08
EP4504339A2 (en) 2025-02-12
AU2023241739A1 (en) 2024-11-07
TW202404642A (zh) 2024-02-01
US20240058460A1 (en) 2024-02-22
IL315965A (en) 2024-11-01
CN119584961A (zh) 2025-03-07
KR20250016096A (ko) 2025-02-03
JP2025512877A (ja) 2025-04-22
WO2023192642A2 (en) 2023-10-05
CA3247116A1 (en) 2023-10-05
WO2023192642A3 (en) 2023-11-30

Similar Documents

Publication Publication Date Title
MX2024012094A (es) Metodos y compuestos para modular la enfermedad de huntington
CU24743B1 (es) Composiciones inmunogénicas contra el coronavirus
AR110148A1 (es) Aparato y método para codificar o decodificar una señal multicanal usando una ganancia lateral y una ganancia residual
CO6141479A2 (es) Proceso para producir poxvirus y composiciones de poxvirus
AR069020A1 (es) Composicion para el cuidado del cabello
BR112022025653A2 (pt) Vacina autoamplificante de rna sars-cov-2
EA200601760A1 (ru) Тетрагидроиндазольные модуляторы каннабиноидов
MX344973B (es) Combinaciones antitumorales que contienen anticuerpos que reconocen especificamente la cd38 y la citarabina.
BRPI0509819A (pt) integração de marca d' água
UY26815A1 (es) Métodos para tratar enfermedades reumáticas al usar una molécula ctla4 soluble
CY1117681T1 (el) Ρυθμιστες 2,3-διοξυγενασης ινδολοαμινης και μεθοδοι χρησης αυτων
BR0200350A (pt) Bactéria produtora de l-aminoácido pertencente ao gênero escherichia, e, método produzir l-aminoácido
NO20063244L (no) Florfenikolprolegemiddel som har forbedret vannloselighet
AR054207A1 (es) Metodo para agotar corrientes de procesamiento de vinaza diluida
DE602004029075D1 (de) Verwendung von knotenholzextrakten
AR114790A1 (es) Producción de hidrocarburos
BR0103282A (pt) Processo para a preparação de polìmero de vinilpinolidona e produto resultante
MX2021014594A (es) Procedimiento de una etapa para producir un oleogel proteico.
CY1112992T1 (el) Μεσο καλλιεργειας χωρις ορο για την παρασκευη ανασυνδυασμενων γοναδοτροπινων
CO6290699A2 (es) Termoestabilizacion de proteinas
DE60311990D1 (de) Verfahren zur herstellung eines virus inaktivierten thrombin präparates
MX2023006846A (es) Microorganismo que expresa una proteina derivada de shewanella oneidensis y metodo para producir l-aminoacido usando el mismo.
WO2021257816A3 (en) Modified caveolin-1 peptides for the treatment of pathogen-induced lung injury
WO2023133284A3 (en) Compounds and methods for treating friedreich's ataxia
AR096355A1 (es) Composición odontológica de papaína en gel para el tratamiento atraumático de caries y método para prepararla