SG11201805217XA - Compositions and methods for the treatment of hemoglobinopathies - Google Patents

Compositions and methods for the treatment of hemoglobinopathies

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Publication number
SG11201805217XA
SG11201805217XA SG11201805217XA SG11201805217XA SG11201805217XA SG 11201805217X A SG11201805217X A SG 11201805217XA SG 11201805217X A SG11201805217X A SG 11201805217XA SG 11201805217X A SG11201805217X A SG 11201805217XA SG 11201805217X A SG11201805217X A SG 11201805217XA
Authority
SG
Singapore
Prior art keywords
cambridge
novartis
international
intellia
suite
Prior art date
Application number
SG11201805217XA
Inventor
Anthony Edward Boitano
Michael Cooke
Lloyd B Klickstein
Reynald Lescarbeau
Craig Stephen Mickanin
Kabungo Mulumba
Seshidhar Reddy Police
Jennifer Snead
Susan C Stevenson
Morag Stewart
Yi Yang
Original Assignee
Novartis Ag
Intellia Therapeutics Inc
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, Intellia Therapeutics Inc filed Critical Novartis Ag
Publication of SG11201805217XA publication Critical patent/SG11201805217XA/en

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    • C12N15/09—Recombinant DNA-technology
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/461—Cellular immunotherapy characterised by the cell type used
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • A—HUMAN NECESSITIES
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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
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
    • A—HUMAN NECESSITIES
    • 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
    • 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
    • A61K2239/38—Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the dose, timing or administration schedule
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • 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
    • C—CHEMISTRY; METALLURGY
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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

12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 11111111011110111010101111101011111011101110111110101111011101111101111011111 International Bureau ... ..... ..)d (10) International Publication Number International Publication Date .... ...rr,' WO 2017/115268 Al 6 July 2017 (06.07.2017) WI P0 I P CT (51) (21) (22) (25) Filing Language: (26) Publication Language: (30) (71) (72) Inventors: BOITANO, Anthony Edward; 13037 Calvary International Patent Classification: Reddy; 110 Francis Wyman Road, Burlington, MA 01803 C12N 15/113 (2010.01) (US). SNEAD, Jennifer; c/o Novartis Institute for Func- tional Genomics, Inc., dba Genomics Institute of the No- International Application Number: yards Research Foundation, 10675 John Jay Hopkins PCT/IB2016/058007 Drive, San Diego, CA 92121 (US). STEVENSON, Susan International Filing Date: C.; c/o Novartis Institutes for Biomedical Research Inc., 26 December 2016 (26.12.2016) 250 Massachusetts Avenue, Cambridge, MA 02139 (US). STEWART, Morag; c/o Intellia Therapeutics, Inc., 40 English Erie Street, Suite 130, Cambridge, MA 02139 (US). English YANG, Yi; c/o Novartis Institutes for Biomedical Re- search Inc., 250 Massachusetts Avenue, Cambridge, MA Priority Data: 02139 (US). 62/271,968 28 December 2015 (28.12.2015) US 62/347,484 8 June 2016 (08.06.2016) US (74) Common Representative: NOVARTIS AG; Lichtstrasse 35, 4056 Basel (CH). Applicants: NOVARTIS AG [CH/CH]; Lichtstrasse 35, 4056 Basel (CH). INTELLIA THERAPEUTICS, INC. (81) Designated States (unless otherwise indicated, for every [US/US]; 40 Erie Street, Suite 130, Cambridge, MA 02139 kind of national protection available): AE, AG, AL, AM, (US). AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, Ct., San Diego, CA 92129 (US). COOKE, Michael; 4265 HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, Ingleside Avenue, San Diego, CA 92103 (US). KLICK- KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, STEIN, Lloyd B.; c/o Novartis Institutes for Biomedical MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, Research Inc., 250 Massachusetts Avenue, Cambridge, MA NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, 02139 (US). LESCARBEAU, Reynald; c/o Intellia Thera- RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, peutics, Inc., 40 Erie Street, Suite 130, Cambridge, MA TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, 02139 (US). MICKANIN, Craig Stephen; c/o Novartis ZA, ZM, ZW. Institutes for Biomedical Research Inc., 250 Massachusetts Avenue, Cambridge, MA 02139 (US). MULUMBA, Ka- (84) Designated States (unless otherwise indicated, for every bungo; c/o Intellia Therapeutics, Inc., 40 Erie Street, Suite kind of regional protection available): ARIPO (BW, GH, 130, Cambridge, MA 02139 (US). POLICE, Seshidhar GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, [Continued on next page] (54) Title: COMPOSITIONS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES O pathies. Figure 1 458 Editing Efficiency by Location . , to , 0 Ot: , .4. ' t 2', .. 9. 1r 4 ..) for the treatment of hemoglobino - Il GC V; ei 11 - 1--1 1-1 (57) ei kr) 6449440 0 11 89494600 60494804 mama Generns C42101044 ph , : The present invention is directed to genome editing systems, 50495209 60483400 2) reagents and methods O WO 2017/115268 Al 1#1101MMIMIIMME3111010111IIMEHOIS TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, Published: TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, — DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, with international search report (Art. 21(3)) LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, — before the expiration of the time limit for amending the SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). claims and to be republished in amendments (Rule 48.2(h)) the event of receipt of Declarations under Rule 4.17: — as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(ii))
SG11201805217XA 2015-12-28 2016-12-26 Compositions and methods for the treatment of hemoglobinopathies SG11201805217XA (en)

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
SG11201805217XA true SG11201805217XA (en) 2018-07-30

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Country Status (16)

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

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