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1. WO2021021615 - RECOMBINANT HEME OXYGENASE-1 (HO-1) FOR THE TREATMENT OF SICKLE CELL DISEASE

Publication Number WO/2021/021615
Publication Date 04.02.2021
International Application No. PCT/US2020/043452
International Filing Date 24.07.2020
IPC
A61K 38/44 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
44Oxidoreductases (1)
A61P 7/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
7Drugs for disorders of the blood or the extracellular fluid
C12N 5/10 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
5Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
CPC
A61K 38/44
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
44Oxidoreductases (1)
A61P 7/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
7Drugs for disorders of the blood or the extracellular fluid
Applicants
  • SHIRE HUMAN GENETIC THERAPIES, INC. [US]/[US]
  • UNIVERSITY OF PITTSBURGH-OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION [US]/[US]
Inventors
  • OFORI-ACQUAH, Solomon, Fiifi
  • GHOSH, Samit
  • SYPEK, Joseph
  • ZHANG, Bohong
  • SUN, Xiuxia
  • PAN, Clark, Q.
  • LAJOIE, Daniel, Minard
  • SHEN, Chuan
Agents
  • CHEN, Fangli
  • MCGOUGH, Kevin
  • FURMAN, Margo
  • COUSINS, Abigail
  • SHALEK, James
Priority Data
62/879,13126.07.2019US
63/049,28508.07.2020US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) RECOMBINANT HEME OXYGENASE-1 (HO-1) FOR THE TREATMENT OF SICKLE CELL DISEASE
(FR) HÈME OXYGÉNASE-1 (HO-1) RECOMBINANTE POUR LE TRAITEMENT DE LA DRÉPANOCYTOSE
Abstract
(EN)
The present invention provides, among other things, methods and compositions for making and using recombinant heme oxygenase for treating sickle cell disease. In some embodiments, recombinant heme oxygenase proteins are truncation variants, or Fc fusion proteins with increased half-life and/or reduced aggregation.
(FR)
La présente invention concerne, entre autres, des procédés et des compositions permettant de fabriquer et d'utiliser de l'hème oxygénase recombinante pour traiter la drépanocytose. Dans certains modes de réalisation, des protéines d'hème oxygénase recombinante sont des variants de troncature ou des protéines de fusion Fc présentant une demi-vie accrue et/ou une agrégation réduite.
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