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1. WO2022072706 - ASSAYS FOR FABRY DISEASE TREATMENT

Publication Number WO/2022/072706
Publication Date 07.04.2022
International Application No. PCT/US2021/052982
International Filing Date 30.09.2021
IPC
A61P 5/00 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
5Drugs for disorders of the endocrine system
A61K 38/47 2006.1
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 38/47
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 5/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
5Drugs for disorders of the endocrine system
Applicants
  • SANGAMO THERAPEUTICS, INC. [US]/[US]
Inventors
  • HOLTER, Joshua Nelson
Agents
  • KIM, Ji Eun
  • BANOWIT, Donald R.
  • BEMBEN, Richard M.
  • BEZOS, Salvador M.
  • BLOCK, Daniel S.
  • BODENSTEIN, Matthew S.
  • CALVO, Paul A.
  • CAMARCE, Christian
  • CIMBALA, Michele A.
  • COLLER III, Richard D.
  • CONKLIN, Kyle E.
  • COVERT, John M.
  • DURKIN, Tracy-Gene
  • EISENBERG, Jason D.
  • ELLISON, Eldora L.
  • ESMOND, Robert W.
  • FEATHERSTONE, Donald A.
  • FITZSIMMONS, Jason A.
  • FRUEAUF, Jeremiah B.
  • GAJEWSKI, Daniel A.
  • GILLENTINE, Marsha Rose
  • GOLDSTEIN, Jorge A.
  • HELVEY, Jeffrey T.
  • HICKS, Ross G.
  • HOLOUBEK, Michelle K.
  • JACKMAN, Peter A.
  • KENTON JR., Lestin L.
  • LEE, Michael Q.
  • LONGSWORTH, Gaby L.
  • MILLONIG, Robert C.
  • MUTSCHELKNAUS, Joseph
  • NANNENGA-COMBS, Bonnie W.
  • POWERS III, R. Wilson
  • RAY, Michael B.
  • RICHARDSON, Ryan C.
  • RYGIEL, Mark W.
  • SHEA JR., Timothy J.
  • SOKOHL, Robert E.
  • SPECHT, Michael D.
  • STEFFE, Eric K.
  • STERLING, Deborah A.
  • STERNE, Robert Greene
  • GLENN, Dallin G.
  • HOLMAN, David H.
  • PHERO, Graham C.
  • HOPFINGER, Todd
  • KIM, Josephine
Priority Data
63/086,52301.10.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) ASSAYS FOR FABRY DISEASE TREATMENT
(FR) DOSAGES POUR LE TRAITEMENT DE LA MALADIE DE FABRY
Abstract
(EN) The disclosure provides the enzyme and non-enzyme replacement therapies for treating Fabry disease in a human subject, determined based on measuring the presence or absence of an anti-α-Galactosidase A (GLA) neutralizing antibody in a biological sample of the subject, as measured by an anti-GLA neutralizing antibody assay.
(FR) L'invention concerne des thérapies de remplacement enzymatiques et non enzymatiques pour le traitement de la maladie de Fabry chez un sujet humain, déterminées sur la base de la mesure de la présence ou de l'absence d'un anticorps de neutralisation anti-α-Galactosidase A (GLA) dans un échantillon biologique du sujet, tel que mesurée par un dosage d'anticorps de neutralisation anti-GLA.
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