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1. WO2020198685 - COMPOSITIONS, DEVICES AND METHODS FOR TREATING FABRY DISEASE

Publication Number WO/2020/198685
Publication Date 01.10.2020
International Application No. PCT/US2020/025485
International Filing Date 27.03.2020
IPC
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
A61K 48/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61L 27/52 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
LMETHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION, OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS, OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS, OR SURGICAL ARTICLES
27Materials for prostheses or for coating prostheses
50Materials characterised by their function or physical properties
52Hydrogels or hydrocolloids
Applicants
  • SIGILON THERAPEUTICS, INC. [US]/[US]
Inventors
  • BARNEY, Lauren, Emily
  • BEAUREGARD, Michael
  • CARMONA, Guillaume
  • GONZALEZ, Francisco, Caballero
  • HEIDEBRECHT, Richard
  • JOHNSTON, Erika, Ellen
  • MILLER, Robert, James
  • O'CONNOR, Owen
  • OBERLI, Matthias, Alexander
  • PERITT, David
  • SEWELL, Jared, A.
  • SMITH, Devyn, McKinley
  • VEISEH, Omid
  • WAY, Jeffrey, Charles
  • WOTTON, Paul, Kevin
  • YIN, Zoe
Agents
  • COLLAZO, Diana, M.
  • LARKIN, Angelyn
Priority Data
62/824,96927.03.2019US
62/907,38027.09.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS, DEVICES AND METHODS FOR TREATING FABRY DISEASE
(FR) COMPOSITIONS, DISPOSITIFS ET MÉTHODES DE TRAITEMENT DE LA MALADIE DE FABRY
Abstract
(EN)
Described herein are RPE cells engineered to secrete a GLA protein, as well as compositions, pharmaceutical preparations, and implantable devices comprising the engineered RPE cells, and methods of making and using the same for treating Fabry disease.
(FR)
L'invention concerne des cellules RPE modifiées pour sécréter une protéine GLA, ainsi que des compositions, des préparations pharmaceutiques et des dispositifs implantables comprenant les cellules RPE modifiées, et des méthodes de fabrication et d'utilisation de celles-ci pour le traitement de la maladie de Fabry.
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