BR112022003598A2 - Métodos para tratamento da doença cln2 em indivíduos pediátricos - Google Patents

Métodos para tratamento da doença cln2 em indivíduos pediátricos

Info

Publication number
BR112022003598A2
BR112022003598A2 BR112022003598A BR112022003598A BR112022003598A2 BR 112022003598 A2 BR112022003598 A2 BR 112022003598A2 BR 112022003598 A BR112022003598 A BR 112022003598A BR 112022003598 A BR112022003598 A BR 112022003598A BR 112022003598 A2 BR112022003598 A2 BR 112022003598A2
Authority
BR
Brazil
Prior art keywords
methods
subject
disease
cln2 disease
cln2
Prior art date
Application number
BR112022003598A
Other languages
English (en)
Inventor
David Jacoby
Joshua Henshaw
Original Assignee
Biomarin Pharm 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 Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
Publication of BR112022003598A2 publication Critical patent/BR112022003598A2/pt

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4813—Exopeptidases (3.4.11. to 3.4.19)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0024—Solid, semi-solid or solidifying implants, which are implanted or injected in body tissue
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A61K9/0051—Ocular inserts, ocular implants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/06—Ointments; Bases therefor; Other semi-solid forms, e.g. creams, sticks, gels
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Organic Chemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Dermatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Inorganic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

métodos para tratamento da doença cln2 em indivíduos pediátricos. a presente invenção refere-se a métodos de tratamento da doença lipofuscinose ceroide neuronal (cln2) em um indivíduo com menos de 3 anos de idade. em modalidades exemplificativas, o método compreende administrar ao indivíduo uma formulação que compreende tripeptidil peptidase-1 humana recombinante (rhtpp1) em uma quantidade eficaz para tratar a doença cln2 no indivíduo. também são fornecidos métodos para retardar o início da doença cln2, ou um sintoma da mesma, em um indivíduo com menos de 3 anos de idade. em modalidades exemplificativas, o método compreende administrar ao indivíduo uma formulação que compreende tripeptidil peptidase-1 humana recombinante (rhtpp1) em uma quantidade eficaz para retardar o início da doença ou sintoma de cln2 no indivíduo.
BR112022003598A 2019-08-29 2020-08-31 Métodos para tratamento da doença cln2 em indivíduos pediátricos BR112022003598A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962893535P 2019-08-29 2019-08-29
PCT/US2020/048704 WO2021042020A1 (en) 2019-08-29 2020-08-31 Methods for treating cln2 disease in pediatric subjects

Publications (1)

Publication Number Publication Date
BR112022003598A2 true BR112022003598A2 (pt) 2022-05-24

Family

ID=72470612

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022003598A BR112022003598A2 (pt) 2019-08-29 2020-08-31 Métodos para tratamento da doença cln2 em indivíduos pediátricos

Country Status (21)

Country Link
US (1) US20220273775A1 (pt)
EP (2) EP4021575B1 (pt)
JP (1) JP2022546042A (pt)
KR (1) KR20220054375A (pt)
CN (1) CN114828960A (pt)
AR (1) AR119871A1 (pt)
AU (1) AU2020336984A1 (pt)
BR (1) BR112022003598A2 (pt)
CA (1) CA3152521A1 (pt)
CL (1) CL2022000496A1 (pt)
DK (1) DK4021575T3 (pt)
FI (1) FI4021575T3 (pt)
HR (2) HRP20220370A1 (pt)
IL (1) IL290763A (pt)
LT (1) LT4021575T (pt)
MX (1) MX2022002451A (pt)
PL (1) PL4021575T3 (pt)
PT (1) PT4021575T (pt)
RS (1) RS65763B1 (pt)
TW (1) TW202122106A (pt)
WO (1) WO2021042020A1 (pt)

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6302685B1 (en) 1997-09-16 2001-10-16 University Of Medicine And Dentistry Of New Jersey Human lysosomal protein and methods of its use
US20020006400A1 (en) * 2000-05-11 2002-01-17 Peter Lobel Recombinant human CLN2 protein and methods of its production and use
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
EP2793922B1 (en) 2011-12-23 2019-10-30 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
TWI752907B (zh) * 2015-05-08 2022-01-21 美商拜奧馬林製藥公司 用於治療cln2疾病之tpp1調配物及方法
EP3364970B1 (en) * 2015-10-23 2024-02-07 University of Iowa Research Foundation Gene therapy for use in treating lysosomal storage disease
EP3880235A4 (en) * 2018-11-14 2022-08-10 REGENXBIO Inc. GENE THERAPY OF NEURAL ZEROID LIPOFUSCINOSES

Also Published As

Publication number Publication date
EP4438120A2 (en) 2024-10-02
AU2020336984A1 (en) 2022-03-10
HRP20220370A1 (hr) 2022-09-16
KR20220054375A (ko) 2022-05-02
RS65763B1 (sr) 2024-08-30
MX2022002451A (es) 2022-06-02
IL290763A (en) 2022-04-01
PT4021575T (pt) 2024-07-30
DK4021575T3 (da) 2024-08-05
HRP20240871T1 (hr) 2024-10-11
AR119871A1 (es) 2022-01-19
LT4021575T (lt) 2024-08-12
US20220273775A1 (en) 2022-09-01
FI4021575T3 (fi) 2024-07-24
EP4021575A1 (en) 2022-07-06
WO2021042020A1 (en) 2021-03-04
EP4021575B1 (en) 2024-05-22
CL2022000496A1 (es) 2022-10-21
CN114828960A (zh) 2022-07-29
CA3152521A1 (en) 2021-03-04
TW202122106A (zh) 2021-06-16
PL4021575T3 (pl) 2024-09-23
JP2022546042A (ja) 2022-11-02

Similar Documents

Publication Publication Date Title
Feng et al. Neuroprotective effects of resatorvid against traumatic brain injury in rat: involvement of neuronal autophagy and TLR4 signaling pathway
Nimma et al. Holistic healing through herbs: Effectiveness of Aloe vera on post extraction socket healing
Zykova et al. Macrophage stimulating agent soluble yeast β‐1, 3/1, 6‐glucan as a topical treatment of diabetic foot and leg ulcers: A randomized, double blind, placebo‐controlled phase II study
BR112017024777A2 (pt) uso de uma composiçao farmacêutica e de um antibiótico não absorvível para tratamento de transtorno do espectro autista referência cruzada a pedidos relacionados?
BR112018016001A2 (pt) composição farmacêutica, métodos de tratamento e usos da mesma
BR112013017446A2 (pt) composição, uso de dose baixa de pioglitazona, método de tratar a deterioração cognitiva do tipo de alzheimer em um indivíduo humano, de tratar o declínio cognitivo em um indivíduo humano, de determinar o risco aumentado de desenvolver a deterioração cognitiva do tipo de alzheimer em um indivíduo humano em uma uma idade ou faixa de idade pré-determinadas, de determinar se administrar dose baixa de pioglitazona a um indivíduo humano para o tratamento da deterioração congnitiva do tipo de alzheimer, de retardar o início da doença de alzheimer em um indivíduo em risco de desenvolver a doença alzheimer, de retardar o início da deterioração cognitiva amnésia branda em um indivíduo em risco de desenvolver a doença de alzheimer, de retardar o início da doença de alzheimer pré-clínica em um indivíduo em risco de desenvolver a doença de alzheimer, de retardar o início da doença de alzheimer pródroma em um indivíduo em risco de desenvolver a doença de alzheimer, dose baixa de pioglitazona
BR112017023805A2 (pt) formulações de tpp1 e métodos para o tratamento de doença lcn2
Theilen et al. Exercise preconditioning diminishes skeletal muscle atrophy after hindlimb suspension in mice
Di Marco et al. The time course of action of two neuroprotectants, dietary saffron and photobiomodulation, assessed in the rat retina
BRPI0512503A (pt) composição que compreende ácido lático e lactoferrina
Machado-Vieira et al. A longitudinal (6-week) 3T 1H-MRS study on the effects of lithium treatment on anterior cingulate cortex metabolites in bipolar depression
BR112023001143A2 (pt) Conjugado, composto, composição farmacêutica, métodos para tratar uma doença e reduzir o nível de uma proteína brm alvo em um indivíduo
Ahmed et al. Efficacy of clarithromycin in pityriasis rosea
BR112022003598A2 (pt) Métodos para tratamento da doença cln2 em indivíduos pediátricos
BR112022009881A2 (pt) Inibidores de caspase 6 e usos dos mesmos
Ivo et al. Woman in grey: hydroxychloroquine-induced hyperpigmentation
Kocer et al. Clinical and electrophysiological evaluation of patients with thalidomide-induced neuropathy
Xue et al. Analgesic mechanism of electroacupuncture in a rat L5 spinal nerve ligation model
Sordi et al. Elastic bandage as a therapeutic resource for the control of sialorrhea: an analysis of its efficacy
BR112022027124A2 (pt) Formulações para promover a hidratação e métodos de uso das mesmas
Hirabayashi et al. Analgesic effect of mineral cream containing natural spa minerals for use on the skin
Bassi et al. Inflammatory morphea presenting as a hemifacial acquired port-wine stain
Ghuman et al. Ketorolac and morphine for analgesia in acute renal colic: Is this combination more effective than monotherapy?
EP3534874B1 (en) Cosmetic method
Ediriweera et al. A clinical Study on effect of paste of Haritaki in Padadari (Cracked Feet)