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1. WO2021055801 - IMPROVED ALPHA-GALACTOSIDASE PROTEIN FOR ENZYME REPLACEMENT THERAPY (ERT) AND METHODS OF USE

Publication Number WO/2021/055801
Publication Date 25.03.2021
International Application No. PCT/US2020/051570
International Filing Date 18.09.2020
IPC
A61P 3/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
A61P 43/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43Drugs for specific purposes, not provided for in groups A61P1/-A61P41/108
A61K 38/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
A61P 13/12 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
13Drugs for disorders of the urinary system
12of the kidneys
A61K 38/47 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43Enzymes; Proenzymes; Derivatives thereof
46Hydrolases (3)
47acting on glycosyl compounds (3.2), e.g. cellulases, lactases
CPC
A61K 38/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
A61K 38/47
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43Enzymes; Proenzymes; Derivatives thereof
46Hydrolases (3)
47acting on glycosyl compounds (3.2), e.g. cellulases, lactases
A61P 13/12
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
13Drugs for disorders of the urinary system
12of the kidneys
A61P 3/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3Drugs for disorders of the metabolism
A61P 43/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
43Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
Applicants
  • THE MEDICAL COLLEGE OF WISCONSIN, INC. [US]/[US]
Inventors
  • MEDIN, Jeffrey, A.
  • SCALIA, Simone
Agents
  • BABEL, Angeline, R.
Priority Data
62/902,21118.09.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) IMPROVED ALPHA-GALACTOSIDASE PROTEIN FOR ENZYME REPLACEMENT THERAPY (ERT) AND METHODS OF USE
(FR) PROTÉINE ALPHA-GALACTOSIDASE AMÉLIORÉE POUR ENZYMOTHÉRAPIE DE REMPLACEMENT (ERT) ET PROCÉDÉS D'UTILISATION
Abstract
(EN)
The present disclosure provides less immunogenic mutant α-galactosidase A protein (α- gal), methods of making and methods of use. The less immunogenic mutant α-gal of the present invention provides a reduced immune response when administered to a subject.
(FR)
La présente invention concerne une protéine A α-galactosidase (α-gal) mutante moins immunogène, des procédés de fabrication et des procédés d'utilisation. L'α-gal mutante moins immunogène selon la présente invention fournit une réponse immunitaire réduite lorsqu'elle est administrée à un sujet.
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