CA2236989A1 - Therapie innovatrice pour le traitement du deficit du stockage lysosomal - Google Patents

Therapie innovatrice pour le traitement du deficit du stockage lysosomal Download PDF

Info

Publication number
CA2236989A1
CA2236989A1 CA002236989A CA2236989A CA2236989A1 CA 2236989 A1 CA2236989 A1 CA 2236989A1 CA 002236989 A CA002236989 A CA 002236989A CA 2236989 A CA2236989 A CA 2236989A CA 2236989 A1 CA2236989 A1 CA 2236989A1
Authority
CA
Canada
Prior art keywords
disease
mps
microcapsules
lysosomal storage
enzyme
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
CA002236989A
Other languages
English (en)
Inventor
Patricia L. Chang
Colin J.D. Ross
Martin R. Ralph
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Individual
Original Assignee
Individual
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 Individual filed Critical Individual
Priority to CA002236989A priority Critical patent/CA2236989A1/fr
Priority to AU35141/99A priority patent/AU3514199A/en
Priority to PCT/CA1999/000368 priority patent/WO1999056770A2/fr
Publication of CA2236989A1 publication Critical patent/CA2236989A1/fr
Abandoned legal-status Critical Current

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)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • 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
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1629—Organic macromolecular compounds
    • A61K9/1652—Polysaccharides, e.g. alginate, cellulose derivatives; Cyclodextrin

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Health & Medical Sciences (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA002236989A 1998-05-06 1998-05-06 Therapie innovatrice pour le traitement du deficit du stockage lysosomal Abandoned CA2236989A1 (fr)

Priority Applications (3)

Application Number Priority Date Filing Date Title
CA002236989A CA2236989A1 (fr) 1998-05-06 1998-05-06 Therapie innovatrice pour le traitement du deficit du stockage lysosomal
AU35141/99A AU3514199A (en) 1998-05-06 1999-05-06 Novel therapy for treatment of lysosomal storage disease
PCT/CA1999/000368 WO1999056770A2 (fr) 1998-05-06 1999-05-06 Nouveau traitement des maladies a de lysosomes

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
CA002236989A CA2236989A1 (fr) 1998-05-06 1998-05-06 Therapie innovatrice pour le traitement du deficit du stockage lysosomal

Publications (1)

Publication Number Publication Date
CA2236989A1 true CA2236989A1 (fr) 1999-11-06

Family

ID=4162400

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002236989A Abandoned CA2236989A1 (fr) 1998-05-06 1998-05-06 Therapie innovatrice pour le traitement du deficit du stockage lysosomal

Country Status (3)

Country Link
AU (1) AU3514199A (fr)
CA (1) CA2236989A1 (fr)
WO (1) WO1999056770A2 (fr)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2272482T3 (es) 2000-01-20 2007-05-01 Diabcell Pty Limited Preparacion y xenotransplante de islotes porcinos.

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5543390A (en) * 1990-11-01 1996-08-06 State Of Oregon, Acting By And Through The Oregon State Board Of Higher Education, Acting For And On Behalf Of The Oregon Health Sciences University Covalent microparticle-drug conjugates for biological targeting

Also Published As

Publication number Publication date
AU3514199A (en) 1999-11-23
WO1999056770A3 (fr) 2000-01-13
WO1999056770A2 (fr) 1999-11-11

Similar Documents

Publication Publication Date Title
Ross et al. Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells
Ellinwood et al. Gene therapy for lysosomal storage diseases: the lessons and promise of animal models
Haskins Gene therapy for lysosomal storage diseases (LSDs) in large animal models
Chang et al. The in vivo delivery of heterologous proteins by microencapsulated recombinant cells
Floyd et al. Ex vivo gene transfer using adenovirus-mediated full-length dystrophin delivery to dystrophic muscles
EP2269658B1 (fr) Therapie genique pour troubles neurometaboliques
ES2635726T3 (es) Terapia génica para enfermedades de almacenamiento lisosómico
Marcó et al. In vivo gene therapy for mucopolysaccharidosis type III (Sanfilippo syndrome): a new treatment horizon
Novelli et al. Gene therapy for lysosomal storage disorders
Hobert et al. Neuronal ceroid lipofuscinoses therapeutic strategies: past, present and future
TW201625793A (zh) 用於治療溶酶體貯積病之腺相關病毒載體
US20230332112A1 (en) Novel In Vitro and In Vivo Enrichment Strategy Targeting Lymphocytes Derived from Vector Transduced HSCS for Therapy of Disorders
Fairbairn et al. Long-term in vitro correction of alpha-L-iduronidase deficiency (Hurler syndrome) in human bone marrow.
US20230201373A1 (en) Crispr-mediated genome editing with vectors
Suzuki et al. Mouse models of human lysosomal diseases
Sands et al. Gene therapy for murine mucopolysaccharidosis type VII
Brady et al. Enzyme replacement therapy for Gaucher disease: critical investigations beyond demonstration of clinical efficacy
US20040082535A1 (en) Products and methods for gaucher disease therpy
Vogler et al. Murine mucopolysaccharidosis type VII: the impact of therapies on the clinical course and pathology in a murine model of lysosomal storage disease
d’Azzo Gene transfer strategies for correction of lysosomal storage disorders
Tomanin et al. Non‐viral transfer approaches for the gene therapy of mucopolysaccharidosis type II (Hunter syndrome)
CA2236989A1 (fr) Therapie innovatrice pour le traitement du deficit du stockage lysosomal
Nakama et al. Encapsulation cell therapy for mucopolysaccharidosis type VII using genetically engineered immortalized human amniotic epithelial cells
Lagranha et al. In vitro correction of ARSA deficiency in human skin fibroblasts from metachromatic leukodystrophy patients after treatment with microencapsulated recombinant cells
Consiglio et al. Metabolic correction in oligodendrocytes derived from metachromatic leukodystrophy mouse model by using encapsulated recombinant myoblasts

Legal Events

Date Code Title Description
FZDE Dead