SG11201903042VA - Engineered meganucleases specific for recognition sequences in the hepatitis b virus genome - Google Patents

Engineered meganucleases specific for recognition sequences in the hepatitis b virus genome

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Publication number
SG11201903042VA
SG11201903042VA SG11201903042VA SG11201903042VA SG11201903042VA SG 11201903042V A SG11201903042V A SG 11201903042VA SG 11201903042V A SG11201903042V A SG 11201903042VA SG 11201903042V A SG11201903042V A SG 11201903042VA SG 11201903042V A SG11201903042V A SG 11201903042VA
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SG
Singapore
Prior art keywords
international
subunit
engineered
hepatitis
pct
Prior art date
Application number
SG11201903042VA
Inventor
Derek Jantz
James Smith
Original Assignee
Precision Biosciences 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.)
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Publication date
Application filed by Precision Biosciences Inc filed Critical Precision Biosciences Inc
Publication of SG11201903042VA publication Critical patent/SG11201903042VA/en

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2730/00—Reverse transcribing DNA viruses
    • C12N2730/00011—Details
    • C12N2730/10011—Hepadnaviridae
    • C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
    • C12N2730/10122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property MillEIMO REE N EVIEHEDERIMEEIIMMEEMIE Organization - ' (10) International Publication Number International Bureau (43) International Publication Date .....0\"\" WO 2018/071849 A2 19 April 2018 (19.04.2018) WIP0 1 PCT _ (51) (21) (22) (25) Filing Language: (26) (30) (71) International Patent Classification: (72) Inventors: JANTZ, C12N 9/22 (2006.01) A61K 38/00 (2006.01) NC 27701 (US). Forest Way, Morrisville, NC 27560 (US). International Application Number: PCT/US2017/056638 (74) Agent: DIPIETRANTONIO, International Filing Date: field & Sacks, 02210-2206 (US). 13 October 2017 (13.10.2017) English (81) Designated States kind of national Publication Language: English AO, AT, AU, AZ, Priority Data: CA, CH, CL, CN, CO, CR, CU, CZ, DE, 62/408,356 14 October 2016 (14.10.2016) US DZ, EC, EE, EG, ES, FF GB, GD, GE, 62/452,506 31 January 2017 (31.01.2017) US HR, HU, ID, IL, 62/527,159 30 June 2017 (30.06.2017) US KR, KW, KZ, LA, 62/527,196 30 June 2017 (30.06.2017) US MG, MK, MN, OM, PA, PE, PG, PH, PL, PT, QA, RO, Applicant: PRECISION BIOSCIENCES, INC. SC, SD, SE, SG, [US/US]; 302 E. Pettigrew Street, Dibrell Building, Suite TR, TT, TZ, UA, A-100, Durham, NC 27701 (US). (84) Designated States kind of regional GM, KE, LR, LS, Derek; 1011 Gloria Avenue, Durham, 1916 Weaver J.; Wolf, Green- Boston, MA for every AE, AG, AL, AM, BR, BW, BY, BZ, DJ, DK, DM, DO, GH, GM, GT, HN, KG, KH, KN, KP, LY, MA, MD, ME, NG, NI, NO, NZ, RS, RU, RW, SA, TH, TJ, TM, TN, ZA, ZM, ZW. for every ARIPO (BW, GH, SL, ST, SZ, TZ, SMITH, James, Jefferson; Heather, P.C., 600 Atlantic Avenue, (unless otherwise indicated, protection available): BA, BB, BG, BH, BN, IN, IR, IS, JO, JP, KE, LC, LK, LR, LS, LU, MW, MX, MY, MZ, NA, SK, SL, SM, ST, SV, SY, UG, US, UZ, VC, VN, (unless otherwise indicated, protection available): MW, MZ, NA, RW, SD, = Title: ENGINEERED : The an open reading methods symptoms of a HBV infection, or treating hepatocellular carcinoma (HCC). Further, the invention encompasses (54) GENOME ---- encompasses 0 compositions N compositions for treating HBV infections or HCC. MEGANUCLEASES SPECIFIC FOR RECOGNITION SEQUENCES IN THE HEPATITIS FIGURE 5 First Subunit Second Subunit B VIRUS I recognition sequence present invention also treating or reducing pharmaceutical and the use of such = EE = EE = = I I 6 = M. iail i: \"i \V ::;::::;::;:::: = HBV1 Second Subunit Linker HBV2 First Subunit i = = = -- M . .M. ei -4! CT 71' GO 1-1 (57) Ir----- within 0 GO , 1 the comprising HBV2 Linker HBV1 present invention encompasses engineered meganucleases which recognize and cleave a frame (ORF) of the genome of at least two genotypes of the Hepatitis B virus (HBV). The of using such engineered meganucleases in a pharmaceutical composition and in methods for engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, C [Continued on next page] WO 2018/071849 A2 IMIOHOIIIMIIMEIHOMMOHOIRIONMOIME UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: — without international search report and to be republished upon receipt of that report (Rule 48.2(g)) — with sequence listing part of description (Rule 5.2(a))
SG11201903042VA 2016-10-14 2017-10-13 Engineered meganucleases specific for recognition sequences in the hepatitis b virus genome SG11201903042VA (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662408356P 2016-10-14 2016-10-14
US201762452506P 2017-01-31 2017-01-31
US201762527196P 2017-06-30 2017-06-30
US201762527159P 2017-06-30 2017-06-30
PCT/US2017/056638 WO2018071849A2 (en) 2016-10-14 2017-10-13 Engineered meganucleases specific for recognition sequences in the hepatitis b virus genome

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US (5) US10662416B2 (en)
EP (2) EP3526323B1 (en)
JP (4) JP6811857B2 (en)
KR (4) KR102305215B1 (en)
CN (2) CN117402852A (en)
AU (2) AU2017342536A1 (en)
BR (1) BR112019007450A2 (en)
CA (1) CA3040143A1 (en)
CL (1) CL2019000993A1 (en)
CO (1) CO2019003675A2 (en)
CR (1) CR20190181A (en)
DK (1) DK3526323T5 (en)
DO (1) DOP2019000097A (en)
FI (1) FI3526323T3 (en)
IL (2) IL308894A (en)
MA (1) MA46535A (en)
MX (2) MX2019004349A (en)
PE (1) PE20191353A1 (en)
PH (1) PH12019500788A1 (en)
SG (2) SG11201903042VA (en)
WO (1) WO2018071849A2 (en)

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