MX2018007987A - Composiciones y metodos para el tratamiento de hemoglobinopatias. - Google Patents

Composiciones y metodos para el tratamiento de hemoglobinopatias.

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Publication number
MX2018007987A
MX2018007987A MX2018007987A MX2018007987A MX2018007987A MX 2018007987 A MX2018007987 A MX 2018007987A MX 2018007987 A MX2018007987 A MX 2018007987A MX 2018007987 A MX2018007987 A MX 2018007987A MX 2018007987 A MX2018007987 A MX 2018007987A
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MX
Mexico
Prior art keywords
hemoglobinopathies
treatment
methods
compositions
reagents
Prior art date
Application number
MX2018007987A
Other languages
English (en)
Inventor
B Klickstein Lloyd
Lescarbeau Reynald
Stewart Morag
Edward Boitano Anthony
Cooke Michael
Stephen Mickanin Craig
Snead Jennifer
C Stevenson Susan
Yang Yi
Mulumba Kabungo
Reddy Police Seshidhar
Original Assignee
Novartis Ag
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 Novartis Ag filed Critical Novartis Ag
Publication of MX2018007987A publication Critical patent/MX2018007987A/es

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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/15—Cells of the myeloid line, e.g. granulocytes, basophils, eosinophils, neutrophils, leucocytes, monocytes, macrophages or mast cells; Myeloid precursor cells; Antigen-presenting cells, e.g. dendritic cells
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    • A61K39/00—Medicinal preparations containing antigens or antibodies
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    • A61K39/461—Cellular immunotherapy characterised by the cell type used
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    • A61K39/46—Cellular immunotherapy
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    • A—HUMAN NECESSITIES
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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    • C12N5/0602—Vertebrate cells
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    • C12N9/14—Hydrolases (3)
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
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    • A61K2239/31—Indexing codes associated with cellular immunotherapy of group A61K39/46 characterized by the route of administration
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K39/46
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification

Abstract

La presente invención se refiere a sistemas de edición genómica, reactivos y métodos para el tratamiento de hemoglobinopatías.
MX2018007987A 2015-12-28 2016-12-26 Composiciones y metodos para el tratamiento de hemoglobinopatias. MX2018007987A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562271968P 2015-12-28 2015-12-28
US201662347484P 2016-06-08 2016-06-08
PCT/IB2016/058007 WO2017115268A1 (en) 2015-12-28 2016-12-26 Compositions and methods for the treatment of hemoglobinopathies

Publications (1)

Publication Number Publication Date
MX2018007987A true MX2018007987A (es) 2019-01-10

Family

ID=57822008

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2018007987A MX2018007987A (es) 2015-12-28 2016-12-26 Composiciones y metodos para el tratamiento de hemoglobinopatias.
MX2022009148A MX2022009148A (es) 2015-12-28 2018-06-27 Composiciones y metodos para el tratamiento de hemoglobinopatias.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2022009148A MX2022009148A (es) 2015-12-28 2018-06-27 Composiciones y metodos para el tratamiento de hemoglobinopatias.

Country Status (16)

Country Link
US (2) US20190010495A1 (es)
EP (2) EP4053277A1 (es)
JP (3) JP2019500043A (es)
KR (1) KR20180103923A (es)
CN (1) CN108779462A (es)
AU (3) AU2016381313B2 (es)
BR (1) BR112018013065A2 (es)
CA (1) CA3009727A1 (es)
EA (1) EA201891532A1 (es)
HK (1) HK1258205A1 (es)
IL (2) IL260257B1 (es)
MX (2) MX2018007987A (es)
MY (1) MY192848A (es)
PH (1) PH12018501378A1 (es)
SG (1) SG11201805217XA (es)
WO (1) WO2017115268A1 (es)

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