MX2023008153A - Metodos y compuestos para tratar la ataxia de friedreich. - Google Patents

Metodos y compuestos para tratar la ataxia de friedreich.

Info

Publication number
MX2023008153A
MX2023008153A MX2023008153A MX2023008153A MX2023008153A MX 2023008153 A MX2023008153 A MX 2023008153A MX 2023008153 A MX2023008153 A MX 2023008153A MX 2023008153 A MX2023008153 A MX 2023008153A MX 2023008153 A MX2023008153 A MX 2023008153A
Authority
MX
Mexico
Prior art keywords
terminus
compounds
methods
binding
ataxia
Prior art date
Application number
MX2023008153A
Other languages
English (en)
Inventor
Chengzhi Zhang
Pratik Shah
Aseem Ansari
Abhi Bhat
Sean Jeffries
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 MX2023008153A publication Critical patent/MX2023008153A/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D519/00Heterocyclic 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D471/00Heterocyclic 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
    • C07D471/02Heterocyclic 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
    • C07D471/04Ortho-condensed systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D401/00Heterocyclic 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
    • C07D401/14Heterocyclic 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02Heterocyclic 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
    • C07D487/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D495/00Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
    • C07D495/12Heterocyclic 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
    • C07D495/14Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D498/00Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D498/02Heterocyclic 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
    • C07D498/04Ortho-condensed systems

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Psychology (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Indole Compounds (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Photoreceptors In Electrophotography (AREA)
  • Medicinal Preparation (AREA)
  • Polyethers (AREA)

Abstract

La presente divulgación se refiere a compuestos y métodos para modular la expresión de fxn, y tratar enfermedades y afecciones en las que fxn desempeña un papel activo. El compuesto puede ser una molécula moduladora de la transcripción que tiene un primer extremo, un segundo extremo y una estructura oligomérica, en donde: a) el primer extremo comprende un resto de unión a ADN capaz de unirse de forma no covalente a una secuencia de repetición de trinucleótidos GAA; b) el segundo extremo comprende un resto de unión a proteína que se une a una molécula reguladora que modula la expresión de un gen que comprende la secuencia de repetición de nucleótidos GAA; y c) la estructura oligomérica comprende un enlazador entre la primera extremo y la segunda extremo.
MX2023008153A 2021-01-08 2022-01-07 Metodos y compuestos para tratar la ataxia de friedreich. MX2023008153A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163135427P 2021-01-08 2021-01-08
PCT/US2022/011560 WO2022150555A2 (en) 2021-01-08 2022-01-07 Methods and compounds for treating friedreich's ataxia

Publications (1)

Publication Number Publication Date
MX2023008153A true MX2023008153A (es) 2023-09-21

Family

ID=82358799

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023008153A MX2023008153A (es) 2021-01-08 2022-01-07 Metodos y compuestos para tratar la ataxia de friedreich.

Country Status (11)

Country Link
US (1) US20240124491A1 (es)
EP (1) EP4274583A2 (es)
JP (1) JP2024502469A (es)
CN (1) CN117241805A (es)
AR (1) AR124589A1 (es)
AU (1) AU2022206424A1 (es)
CA (1) CA3204523A1 (es)
IL (1) IL304322A (es)
MX (1) MX2023008153A (es)
TW (1) TW202241892A (es)
WO (1) WO2022150555A2 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2024008474A (es) * 2022-01-06 2024-07-12 Design Therapeutics Inc Compuestos y metodos para tratar la ataxia de friedreich.

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3019342A1 (en) * 2016-03-30 2017-10-05 Wisconsin Alumni Research Foundation Methods and compositions for modulating frataxin expression
US20210283265A1 (en) * 2018-04-20 2021-09-16 Design Therapeutics, Inc. Methods and compounds for the treatment of genetic disease

Also Published As

Publication number Publication date
WO2022150555A2 (en) 2022-07-14
WO2022150555A3 (en) 2022-12-22
EP4274583A2 (en) 2023-11-15
US20240124491A1 (en) 2024-04-18
AR124589A1 (es) 2023-04-12
CA3204523A1 (en) 2022-07-14
TW202241892A (zh) 2022-11-01
CN117241805A (zh) 2023-12-15
IL304322A (en) 2023-09-01
AU2022206424A1 (en) 2023-08-24
AU2022206424A9 (en) 2024-09-19
JP2024502469A (ja) 2024-01-19

Similar Documents

Publication Publication Date Title
EP4234553A3 (en) Methods and compounds for the treatment of genetic disease
EA202090034A1 (ru) Способы модификации сплайсинга рнк
ATE544473T1 (de) Chimäre moleküle zur modulation der genexpression
MX2023008153A (es) Metodos y compuestos para tratar la ataxia de friedreich.
CY1119614T1 (el) ΟΛΙΓΟΜΕΡΕΙΣ ΕΝΩΣΕΙΣ ΓΙΑ ΤΗ ΔΙΑΜΟΡΦΩΣΗ ΤΗΣ ΕΚΦΡΑΣΗΣ HIF-1α
EA201991309A1 (ru) Способы модуляции сплайсинга рнк
UY26815A1 (es) Métodos para tratar enfermedades reumáticas al usar una molécula ctla4 soluble
CY1105271T1 (el) Δεσμες λειτουργικης πρωτεϊνης
EA200600591A1 (ru) МОДУЛИРОВАНИЕ ЭКСПРЕССИИ eIF4E
ES2689746T3 (es) Anticuerpos monoclonales contra la claudina-18 para el tratamiento de cáncer
EP4234550A3 (en) Methods and compounds for the treatment of genetic disease
EP2189471A4 (en) PEPTIDE FOXM1 AND MEDICINAL AGENT COMPRISING SAME
DK1210357T3 (da) Oligonukleotid-N3' -P5' -thiophosphoramidater: syntese og anvendelse deraf
EA200401622A1 (ru) Способы лечения ангиогенеза, роста опухолей и метастазов
DE602005027399D1 (de) Für humane matriptase spezifische bindungsproteine
BRPI0414810A (pt) composições baseadas no vìrus seneca valley e métodos para tratar doença
CY1117666T1 (el) 4-((ΦΑΙΝΟΞΥΑΛΚΥΛΟ)ΘΕΙΟ)-ΦΑΙΝΟΞΥΟΞΙΚΑ ΟΞΕΑ ΓΙΑ ΧΡΗΣΗ ΣΤΗΝ ΘΕΡΑΠΕΥΤΙΚΗ ΑΓΩΓΗ PPARδ ΔΙΑΜΕΣΟΛΑΒΟΥΜΕΝΩΝ ΠΑΘΗΣΕΩΝ
ATE439856T1 (de) Verwendung von peptiden, die aus der b beta kette des humanen fibrinogens abgeleitet wurden, zur behandlung von schock
NO20044628L (no) Immunglobulindomene som inneholder celleoverflategjenkjennende molekyler
DE502004008819D1 (de) Medikament zur wachstumsinhibierung von tumoren
WO2023133284A3 (en) Compounds and methods for treating friedreich's ataxia
PL371205A1 (en) Composition inhibiting matrix-metallproteinases for the treatment of neoplastic diseases
MX2023015520A (es) Conjugados de oligonucleotidos dirigidos al receptor de transferrina.
EA200701852A1 (ru) Производные аминокислот
WO2007121326A3 (en) Compositions and methods for modulating gene expression