IL314212A - Treatment of GM2 gangliosidosis - Google Patents

Treatment of GM2 gangliosidosis

Info

Publication number
IL314212A
IL314212A IL314212A IL31421224A IL314212A IL 314212 A IL314212 A IL 314212A IL 314212 A IL314212 A IL 314212A IL 31421224 A IL31421224 A IL 31421224A IL 314212 A IL314212 A IL 314212A
Authority
IL
Israel
Prior art keywords
compound
subject
formula
crystalline form
disease
Prior art date
Application number
IL314212A
Other languages
English (en)
Hebrew (he)
Inventor
Jennifer Robin
Fritz Blatter
Robert Hett
Kyle Landskroner
Original Assignee
Azafaros B V
Jennifer Robin
Fritz Blatter
Robert Hett
Kyle Landskroner
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
Priority claimed from EP22150783.3A external-priority patent/EP4209215A1/en
Application filed by Azafaros B V, Jennifer Robin, Fritz Blatter, Robert Hett, Kyle Landskroner filed Critical Azafaros B V
Publication of IL314212A publication Critical patent/IL314212A/en

Links

Classifications

    • 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Pyridine Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
IL314212A 2022-01-10 2023-01-10 Treatment of GM2 gangliosidosis IL314212A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP22150783.3A EP4209215A1 (en) 2022-01-10 2022-01-10 Treatment of gm2 gangliosidosis
EP22193136 2022-08-31
PCT/EP2023/050459 WO2023131720A1 (en) 2022-01-10 2023-01-10 Treatment of gm2 gangliosidosis

Publications (1)

Publication Number Publication Date
IL314212A true IL314212A (en) 2024-09-01

Family

ID=84982296

Family Applications (1)

Application Number Title Priority Date Filing Date
IL314212A IL314212A (en) 2022-01-10 2023-01-10 Treatment of GM2 gangliosidosis

Country Status (9)

Country Link
US (1) US20250120958A1 (https=)
EP (2) EP4714497A2 (https=)
JP (1) JP2025501646A (https=)
AU (1) AU2023205441A1 (https=)
CA (1) CA3242701A1 (https=)
IL (1) IL314212A (https=)
MX (1) MX2024008583A (https=)
TW (1) TW202337456A (https=)
WO (1) WO2023131720A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202539666A (zh) * 2024-02-01 2025-10-16 荷蘭商雅捷法羅斯公司 Gm1神經節苷脂儲積症之治療
EP4595964A1 (en) * 2024-02-01 2025-08-06 Azafaros B.V. Treatment of gm1 gangliosidosis
WO2026013261A1 (en) 2024-07-11 2026-01-15 Azafaros B.V. Nizubaglustat for use in the treatment of lysosomal storage disorders
EP4678174A1 (en) 2024-07-11 2026-01-14 Azafaros B.V. Nizubaglustat for use in the treatment of lysosomal storage disorders

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3461812B1 (en) 2014-03-27 2021-10-13 Academisch Medisch Centrum N-(5-(biphen-4-ylmethyloxy)pentyl)-substituted iminosugars as inhibitors of glucosylceramide synthase
EP3978474A1 (en) * 2020-10-02 2022-04-06 Azafaros B.V. Crystalline forms of a pharmaceutical compound

Also Published As

Publication number Publication date
WO2023131720A1 (en) 2023-07-13
TW202337456A (zh) 2023-10-01
EP4714497A2 (en) 2026-03-25
US20250120958A1 (en) 2025-04-17
JP2025501646A (ja) 2025-01-22
EP4463157A1 (en) 2024-11-20
CA3242701A1 (en) 2023-07-13
MX2024008583A (es) 2024-07-23
EP4463157C0 (en) 2026-02-25
EP4463157B1 (en) 2026-02-25
AU2023205441A1 (en) 2024-07-25

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