WO2011072086A1 - Procédés et régimes à faible dose pour traiter des troubles des globules rouges - Google Patents

Procédés et régimes à faible dose pour traiter des troubles des globules rouges Download PDF

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Publication number
WO2011072086A1
WO2011072086A1 PCT/US2010/059584 US2010059584W WO2011072086A1 WO 2011072086 A1 WO2011072086 A1 WO 2011072086A1 US 2010059584 W US2010059584 W US 2010059584W WO 2011072086 A1 WO2011072086 A1 WO 2011072086A1
Authority
WO
WIPO (PCT)
Prior art keywords
dmb
less
weeks
dimethylbutyrate
blood
Prior art date
Application number
PCT/US2010/059584
Other languages
English (en)
Other versions
WO2011072086A9 (fr
Inventor
Susan Perrine
Ronald J. Berenson
Douglas V. Faller
Original Assignee
Hemaquest Pharmaceuticals, Inc.
The Trustees Of Boston University
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Hemaquest Pharmaceuticals, Inc., The Trustees Of Boston University filed Critical Hemaquest Pharmaceuticals, Inc.
Priority to CN2010800630167A priority Critical patent/CN102802412A/zh
Priority to MA35032A priority patent/MA33899B1/fr
Priority to EP10836658A priority patent/EP2509418A4/fr
Publication of WO2011072086A1 publication Critical patent/WO2011072086A1/fr
Priority to IL220040A priority patent/IL220040A0/en
Priority to TNP2012000270A priority patent/TN2012000270A1/en
Publication of WO2011072086A9 publication Critical patent/WO2011072086A9/fr

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/16—Amides, e.g. hydroxamic acids
    • A61K31/17—Amides, e.g. hydroxamic acids having the group >N—C(O)—N< or >N—C(S)—N<, e.g. urea, thiourea, carmustine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/4406—Non condensed pyridines; Hydrogenated derivatives thereof only substituted in position 3, e.g. zimeldine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7068—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics

Abstract

L'invention porte sur des procédés et des régimes à faibles doses pour augmenter les taux d'hémoglobine fœtale dans des patients présentant des troubles des globules rouges, tels que la bêta-thalassémie, l'anémie falciforme, d'autres anémies, ou une perte de sang. Les taux d'hémoglobine fœtale et totale et les comptages des globules rouges sont augmentés par l'administration de 2,2-diméthylbutyrate (DMB) seul ou en combinaison avec de l'hydroxyurée, de la décitabine ou un inhibiteur de HDAC. Le traitement peut être poursuivi pendant au moins deux semaines.
PCT/US2010/059584 2009-12-08 2010-12-08 Procédés et régimes à faible dose pour traiter des troubles des globules rouges WO2011072086A1 (fr)

Priority Applications (5)

Application Number Priority Date Filing Date Title
CN2010800630167A CN102802412A (zh) 2009-12-08 2010-12-08 用于治疗红细胞病症的方法及低剂量方案
MA35032A MA33899B1 (fr) 2009-12-08 2010-12-08 Procedes et regimes a faible dose pour des troubles des globules rouges
EP10836658A EP2509418A4 (fr) 2009-12-08 2010-12-08 Procédés et régimes à faible dose pour traiter des troubles des globules rouges
IL220040A IL220040A0 (en) 2009-12-08 2012-05-29 Methods and low dose regimens for treating red blood cell disorders
TNP2012000270A TN2012000270A1 (en) 2009-12-08 2012-05-29 Methods and low dose regimens for treating red blood cell disorders

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US26772709P 2009-12-08 2009-12-08
US61/267,727 2009-12-08
US29565010P 2010-01-15 2010-01-15
US61/295,650 2010-01-15
US30637610P 2010-02-19 2010-02-19
US61/306,376 2010-02-19
US31209010P 2010-03-09 2010-03-09
US61/312,090 2010-03-09
US36732610P 2010-07-23 2010-07-23
US61/367,326 2010-07-23
US41136910P 2010-11-08 2010-11-08
US61/411,369 2010-11-08

Publications (2)

Publication Number Publication Date
WO2011072086A1 true WO2011072086A1 (fr) 2011-06-16
WO2011072086A9 WO2011072086A9 (fr) 2012-09-27

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2010/059584 WO2011072086A1 (fr) 2009-12-08 2010-12-08 Procédés et régimes à faible dose pour traiter des troubles des globules rouges

Country Status (7)

Country Link
US (1) US8618068B2 (fr)
EP (1) EP2509418A4 (fr)
CN (1) CN102802412A (fr)
IL (1) IL220040A0 (fr)
MA (1) MA33899B1 (fr)
TN (1) TN2012000270A1 (fr)
WO (1) WO2011072086A1 (fr)

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EP2766041A4 (fr) * 2011-10-12 2015-04-08 Childrens Medical Center Compositions combinatoires et méthodes de traitement d'hémoglobinopathies
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US10472619B2 (en) 2012-11-27 2019-11-12 The Children's Medical Center Corporation Targeting BCL11A distal regulatory elements for fetal hemoglobin reinduction
US10287588B2 (en) 2014-04-25 2019-05-14 The Childrens's Medical Center Corporation Compositions and methods to treating hemoglobinopathies
US11124794B2 (en) 2014-04-25 2021-09-21 The Children's Medical Center Corporation Compositions and methods to treating hemoglobinopathies
US10662429B2 (en) 2014-04-25 2020-05-26 The Children's Medical Center Corporation Compositions and methods to treating hemoglobinopathies
US11739329B2 (en) 2014-04-25 2023-08-29 The Children's Medical Center Corporation Compositions and methods to treating hemoglobinopathies
US11572543B2 (en) 2015-05-08 2023-02-07 The Children's Medical Center. Corporation Targeting BCL11A enhancer functional regions for fetal hemoglobin reinduction
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US8618068B2 (en) 2013-12-31
IL220040A0 (en) 2012-07-31
EP2509418A1 (fr) 2012-10-17
TN2012000270A1 (en) 2013-12-12
EP2509418A4 (fr) 2013-03-20
MA33899B1 (fr) 2013-01-02
US20110251149A1 (en) 2011-10-13
CN102802412A (zh) 2012-11-28
WO2011072086A9 (fr) 2012-09-27

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