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1. WO2020198367 - COMPOUNDS FOR USES IN PHARMACOLOGICAL INDUCTION OF HBF FOR TREATMENT OF SICKLE CELL DISEASE AND β-THALASSEMIA

Publication Number WO/2020/198367
Publication Date 01.10.2020
International Application No. PCT/US2020/024727
International Filing Date 25.03.2020
IPC
C07D 473/16 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
473Heterocyclic compounds containing purine ring systems
02with oxygen, sulfur, or nitrogen atoms directly attached in positions 2 and 6
16two nitrogen atoms
C07D 473/18 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
473Heterocyclic compounds containing purine ring systems
02with oxygen, sulfur, or nitrogen atoms directly attached in positions 2 and 6
18one oxygen and one nitrogen atom, e.g. guanine
A61K 31/52 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
495having six-membered rings with two nitrogen atoms as the only ring hetero atoms, e.g. piperazine
505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
519ortho- or peri-condensed with heterocyclic rings
52Purines, e.g. adenine
A61K 31/5377 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
535having six-membered rings with at least one nitrogen and at least one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
53751,4-Oxazines, e.g. morpholine
5377not condensed and containing further heterocyclic rings, e.g. timolol
A61P 7/06 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
06Antianaemics
Applicants
  • ACADEMIA SINICA
  • NATIONAL HEALTH RESEARCH INSTITUTES
Inventors
  • JIAANG, Weir-Torn
  • SHEN, Che-Kun James
Agents
  • CHAO, Jessica J.
  • BAKER, C., Hunter
  • ALAM, Saad
  • ATTISHA, Michael, J.
  • AMUNDSEN, Eric, L.
Priority Data
62/823,99026.03.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOUNDS FOR USES IN PHARMACOLOGICAL INDUCTION OF HBF FOR TREATMENT OF SICKLE CELL DISEASE AND β-THALASSEMIA
(FR) COMPOSÉS DESTINÉS À ÊTRE UTILISÉS DANS L'INDUCTION PHARMACOLOGIQUE DE HBF POUR LE TRAITEMENT DE LA DRÉPANOCYTOSE ET DE LA β-THALASSÉMIE
Abstract
(EN)
Provided herein are compounds of Formula (I). The compounds described herein are useful in treating a disease associated with the expression of endogenous embryonic/fetal globin (e.g., γ globin) in erythrocytes (e.g., treating β -thalassemia and/or sickle cell disease through at least the induction of the globin gene expression in erythrocytes), and/or anemia (β-thalassemia and/or sickle cell anemia). The compounds described herein are useful in treating, delaying, and/or preventing the adverse effects of β-thalassemia and/or sickle cell disease, inducing γ globin production, and/or inducing the expression of embryonic/fetal globin genes in a subject, cell, tissue, or biological sample. Also provided in the present disclosure are pharmaceutical compositions, kits, and methods of using the compounds for inducing γ globin production described herein and for treating any of the target diseases described herein.
(FR)
L'invention concerne des composés de formule (I). Les composés selon l'invention sont utiles dans le traitement d'une maladie associée à l'expression de globine embryonnaire/fœtale endogène (par exemple, γ globine) dans des érythrocytes (par exemple, le traitement de β-thalassémie et/ou de la drépanocytose par au moins l'induction de l'expression du gène de globine dans les érythrocytes), et/ou l'anémie (β-thalassémie et/ou anémie à cellules falciformes). Les composés selon l'invention sont utiles dans le traitement, le retardement et/ou la prévention d'effets secondaires de la β-thalassémie et/ou de la drépanocytose, l'induction de la production de γ globine, et/ou l'induction de l'expression de gènes de globine embryonnaire/fœtale chez un sujet, une cellule, un tissu ou un échantillon biologique. La présente invention concerne également des compositions pharmaceutiques, des kits et des procédés d'utilisation des composés pour induire la production de γ globine décrite dans la description et pour traiter l'une quelconque des maladies cibles décrites dans la description.
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