3-(5-oxy)-1-oxoisoindolin-2-yl)piperidine-2,6-dione derivatives and uses thereof

The present disclosure relates to compounds of formula (I) and pharmaceutical compositions and their use in reducing Widely Interspaced Zinc Finger Motifs (WIZ) expression levels, or inducing fetal hemoglobin (HbF) expression, and in the treatment of inherited blood disorders (e.g., hemoglobinopathi...

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Hauptverfasser: BONAZZI, SIMONE, CERNIJENKO, ARTIOM, COBB, JENNIFER STROKA, DEWHURST, JANETTA, O'BRIEN, GARY, KERRIGAN, JOHN RYAN, SUNG, MOO JE, THOMSEN, NOEL MARIE-FRANCE, TING, PAMELA YF
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creator BONAZZI, SIMONE
CERNIJENKO, ARTIOM
COBB, JENNIFER STROKA
DEWHURST, JANETTA
O'BRIEN, GARY
KERRIGAN, JOHN RYAN
SUNG, MOO JE
THOMSEN, NOEL MARIE-FRANCE
TING, PAMELA YF
description The present disclosure relates to compounds of formula (I) and pharmaceutical compositions and their use in reducing Widely Interspaced Zinc Finger Motifs (WIZ) expression levels, or inducing fetal hemoglobin (HbF) expression, and in the treatment of inherited blood disorders (e.g., hemoglobinopathies, e.g., beta-hemoglobinopathies), such as sickle cell disease and beta-thalassemia.
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subjects CHEMISTRY
HETEROCYCLIC COMPOUNDS
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
title 3-(5-oxy)-1-oxoisoindolin-2-yl)piperidine-2,6-dione derivatives and uses thereof
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