WO2010030963 MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES

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Publication Number WO/2010/030963

Publication Date 18.03.2010

International Application No. PCT/US2009/056770

International Filing Date 14.09.2009

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Applicants

CHILDREN'S MEDICAL CENTER CORPORATION [US]/[US] (AllExceptUS)
PRESIDENT AND FELLOWS OF HARVARD COLLEGE [US]/[US] (AllExceptUS)
ORKIN, Stuart, H. [US]/[US] (UsOnly)
SANKARAN, Vijay, G. [US]/[US] (UsOnly)

Inventors

ORKIN, Stuart, H.
SANKARAN, Vijay, G.

Agents

RESNICK, David

Priority Data

61/097,017		15.09.2008		US
61/222,571		02.07.2009		US

Publication Language English (en)

Filing Language English (EN)

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Title

(EN) MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES

(FR) MODULATION DE BCL11A POUR LE TRAITEMENT D'HÉMOGLOBINOPATHIES

Abstract

(EN) The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.

(FR) La présente invention concerne des procédés et des utilisations de modulation de l'expression d'hémoglobine fœtale (HbF) dans des cellules progénitrices hématopoïétiques par l'intermédiaire d'inhibiteurs de l'expression ou l'activité de BCL11A, tels que des ARNi et des anticorps.

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15	Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09	Recombinant DNA-technology
63	Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15	Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09	Recombinant DNA-technology
11	DNA or RNA fragments; Modified forms thereof

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48	Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15	Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09	Recombinant DNA-technology
63	Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15	Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09	Recombinant DNA-technology
11	DNA or RNA fragments; Modified forms thereof

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1. WO2010030963 - MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES

A	HUMAN NECESSITIES
01	AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
K	ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2217	Genetically modified animals
05	Animals comprising random inserted nucleic acids (transgenic)
052	inducing gain of function

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
P	SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
7	Drugs for disorders of the blood or the extracellular fluid

C	CHEMISTRY; METALLURGY
07	ORGANIC CHEMISTRY
K	PEPTIDES
14	Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435	from animals; from humans
46	from vertebrates
47	from mammals
4701	not used
4702	Regulators; Modulating activity
4705	stimulating, promoting or activating activity

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
Q	MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS
1	Measuring or testing processes involving enzymes, nucleic acids or microorganisms

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35	Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12	Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
28	Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells