CL2024002029A1 - Compuestos y métodos para tratar la ataxia de friedreich - Google Patents

Compuestos y métodos para tratar la ataxia de friedreich

Info

Publication number
CL2024002029A1
CL2024002029A1 CL2024002029A CL2024002029A CL2024002029A1 CL 2024002029 A1 CL2024002029 A1 CL 2024002029A1 CL 2024002029 A CL2024002029 A CL 2024002029A CL 2024002029 A CL2024002029 A CL 2024002029A CL 2024002029 A1 CL2024002029 A1 CL 2024002029A1
Authority
CL
Chile
Prior art keywords
terminus
compounds
methods
ataxia
fxn
Prior art date
Application number
CL2024002029A
Other languages
English (en)
Spanish (es)
Inventor
Ansari Aseem
Bhat Abhijit
Jeffries Sean
Shah Pratik
Zhang Chengzhi
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 CL2024002029A1 publication Critical patent/CL2024002029A1/es

Links

Classifications

    • 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
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/437Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/56Medicinal 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
    • A61K47/59Medicinal 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
    • A61K47/595Polyamides, e.g. nylon
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/56Medicinal 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
    • A61K47/59Medicinal 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
    • A61K47/60Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07FACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/547Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6561Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
CL2024002029A 2022-01-06 2024-07-03 Compuestos y métodos para tratar la ataxia de friedreich CL2024002029A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202263297090P 2022-01-06 2022-01-06
US202263382854P 2022-11-08 2022-11-08

Publications (1)

Publication Number Publication Date
CL2024002029A1 true CL2024002029A1 (es) 2024-11-04

Family

ID=87074158

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2024002029A CL2024002029A1 (es) 2022-01-06 2024-07-03 Compuestos y métodos para tratar la ataxia de friedreich

Country Status (14)

Country Link
US (1) US20250109132A1 (https=)
EP (1) EP4460302A4 (https=)
JP (1) JP2025502064A (https=)
KR (1) KR20240138079A (https=)
AU (1) AU2023205889A1 (https=)
CA (1) CA3246468A1 (https=)
CL (1) CL2024002029A1 (https=)
CO (1) CO2024010651A2 (https=)
CR (1) CR20240321A (https=)
IL (1) IL314006A (https=)
MX (1) MX2024008474A (https=)
PE (1) PE20241730A1 (https=)
TW (1) TW202334148A (https=)
WO (1) WO2023133284A2 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025076181A1 (en) * 2023-10-03 2025-04-10 Design Therapeutics, Inc. Methods and compounds for modulating huntington's disease
WO2025076219A1 (en) * 2023-10-03 2025-04-10 Design Therapeutics, Inc. Methods and compounds for modulating huntington's disease
WO2025076236A1 (en) * 2023-10-03 2025-04-10 Design Therapeutics, Inc. Methods and compounds for modulating huntington's disease

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013097052A1 (en) * 2011-12-30 2013-07-04 Abbott Laboratories Bromodomain inhibitors
CN106068266A (zh) * 2013-10-11 2016-11-02 千叶县 以癌症驱动基因的基因突变为靶点进行烷基化的新型烷基化剂
US11759533B2 (en) * 2017-03-29 2023-09-19 Wisconsin Alumni Research Foundation Methods and compositions for modulating gene expression
WO2020253711A1 (zh) * 2019-06-17 2020-12-24 中国科学院上海药物研究所 一种吡咯并吡啶酮类化合物、其制备方法、其组合物和用途
KR102677015B1 (ko) * 2019-07-02 2024-06-19 누베이션 바이오 인크. Bet 억제제로서의 헤테로시클릭 화합물
JP2024502469A (ja) * 2021-01-08 2024-01-19 デザイン セラピューティクス,インク. フリードライヒ運動失調症を治療するための方法及び化合物

Also Published As

Publication number Publication date
WO2023133284A3 (en) 2023-08-24
CO2024010651A2 (es) 2024-08-08
CA3246468A1 (en) 2023-07-13
AU2023205889A1 (en) 2024-08-15
CR20240321A (es) 2024-10-16
EP4460302A2 (en) 2024-11-13
JP2025502064A (ja) 2025-01-24
KR20240138079A (ko) 2024-09-20
MX2024008474A (es) 2024-07-12
TW202334148A (zh) 2023-09-01
US20250109132A1 (en) 2025-04-03
IL314006A (en) 2024-08-01
PE20241730A1 (es) 2024-08-19
EP4460302A4 (en) 2025-12-24
WO2023133284A2 (en) 2023-07-13

Similar Documents

Publication Publication Date Title
CO2024010651A2 (es) Compuestos y métodos para tratar la ataxia de friedreich
Jullian et al. N-terminus FITC labeling of peptides on solid support: the truth behind the spacer
CY1123733T1 (el) Γονιδιακα προϊοντα που εκφραζονται διαφορετικα στους ογκους και η χρηση αυτων
ATE544473T1 (de) Chimäre moleküle zur modulation der genexpression
CA3081441A1 (en) Kits for analysis using nucleic acid encoding and/or label
ATE432983T1 (de) Mage-3 peptide, die durch hla-klasse-ii molecule presentiert werden
JP2019506163A5 (https=)
WO2020205605A3 (en) OLIGONUCLEOTIDE-BASED MODULATION OF C9orf72
CY1117786T1 (el) Διαφορικα εκφραζομενα σε ογκους γονιδιακα προϊοντα και χρηση αυτων
CY1117065T1 (el) Υβριδικη και διαδοχικη εκφραση νεϊσσεριακα λαμβανομενων πρωτεϊνων
AR076349A1 (es) Peptido auxiliar del antigeno del cancer
ES2689746T3 (es) Anticuerpos monoclonales contra la claudina-18 para el tratamiento de cáncer
CY1111001T1 (el) Ουρικη οξειδαση
CY1109715T1 (el) IL-1β ΔΕΣΜΕΥΤΙΚΑ ΑΝΤΙΣΩΜΑΤΑ ΚΑΙ ΘΡΑΥΣΜΑΤΑ ΑΥΤΩΝ
PE20071235A1 (es) Metodo para producir l-amino acidos
MX2023008153A (es) Metodos y compuestos para tratar la ataxia de friedreich.
NO20044628L (no) Immunglobulindomene som inneholder celleoverflategjenkjennende molekyler
EA201070229A1 (ru) Нуклеотидные последовательности msca1, влияющие на мужскую фертильность растений, и способ их применения
ATE433993T1 (de) Modulatorische verbindungen und verfahren für glutamattransport
EA200600042A1 (ru) Способы образования дисульфидных связей и гликозилирования белков и реагенты, применяемые в этих способах
EP4234550A3 (en) Methods and compounds for the treatment of genetic disease
US12570688B2 (en) Activated cysteine-directed polypeptide ligation technique
AR128224A1 (es) Compuestos y métodos para tratar la ataxia de friedreich
AR049224A1 (es) Composiciones y metodos para la modificacion de la expresion genica
AR121257A1 (es) Métodos y compuestos para el tratamiento de enfermedad genética