SG11201901364VA - Engineered target specific nucleases - Google Patents

Engineered target specific nucleases

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Publication number
SG11201901364VA
SG11201901364VA SG11201901364VA SG11201901364VA SG11201901364VA SG 11201901364V A SG11201901364V A SG 11201901364VA SG 11201901364V A SG11201901364V A SG 11201901364VA SG 11201901364V A SG11201901364V A SG 11201901364VA SG 11201901364V A SG11201901364V A SG 11201901364VA
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SG
Singapore
Prior art keywords
international
suite
residue
richmond
pct
Prior art date
Application number
SG11201901364VA
Inventor
Jeffrey C Miller
Edward J Rebar
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Sangamo Therapeutics Inc
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Publication of SG11201901364VA publication Critical patent/SG11201901364VA/en

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    • 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
    • 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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21004—Type II site-specific deoxyribonuclease (3.1.21.4)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Gastroenterology & Hepatology (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 Organization International Bureau (43) International Publication Date 01 March 2018 (01.03.2018) WIPO I PCT °mons °nolo ollloolmo Ho mil ioluom oimIE (10) International Publication Number WO 2018/039448 Al IGIHI (51) International Patent Classification: C12N 9/14 (2006.01) A61K 38/16 (2006.01) C12N 9/22 (2006.01) (21) International Application Number: PCT/US2017/048409 (22) International Filing Date: 24 August 2017 (24.08.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/378,978 24 August 2016 (24.08.2016) US 62/443,981 09 January 2017 (09.01.2017) US (71) Applicant: SANGAMO THERAPEUTICS, INC. [US/US]; 501 Canal Blvd., Suite A100, Richmond, Califor- nia 94804 (US). (72) Inventors: MILLER, Jeffrey C.; c/o Sangamo Therapeu- tics, Inc., 501 Canal Blvd., Suite A100, Richmond, Califor- nia 94804 (US). REBAR, Edward J.; c/o Sangamo Thera- peutics, Inc., 501 Canal Blvd., Suite A100, Richmond, Cal- ifornia 94804 (US). (74) Agent: PASTERNAK, Dahna; PASTERNAK PATENT LAW, 1900 Embarcadero Rd., Suite 211, Palo Alto, Cali- fornia 94303 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, 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, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, 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, (54) Title: ENGINEERED TARGET SPECIFIC NUCLEASES 416 418 QL VEISELEEMMSELMIIKLMYVPHEYIELIEIMAIMIMIM CAGCTGGTGAAGAGCGAGCTGGAGGAGAAGAAGTCOGAGCTGCGOCACAAGCTGAAGTACGTGCCCCACGAGTACATCGAGCTGATCGAGATCGCCAGGAACAGC 422 448 450 170711711711711LIEIMIKI V M E F F MMV Y G YEIGIKIHIL SIGISIRIKIP OG A I ACCCAGGACCGCATCOTGGAGATGAAGGTGATGGAGTTOTTCATSAAGGTGTACGGCTACAGGGGARAGCACCTGGGCGGAAGCAGMAGCCTGACGGCGCCATC 467 469 476 479, 480, 481 486 Y T V GS P ID Y G V I V OMOIAIYIISIGI 4ElY TATACAGTGGGCAGGCCCATCGAT2ACGGCMGATCGTGGACACAAAGGCCTACAGCGGCGSCTACAATCTGCCTATCGGCCAGGCCGACGAGATGTAGAGATAC 490 496 499 T E EINIQI TMNMH I N I P I N I E tic WIM V Y P S S V T E FMF L F GTGGTGGAGAACCP,GACCCGGAITAP,GOACAT2CAACCCCAACGAGTGGTSGAAGGTGTACCCTAGCAGCGTGACCGAGT2CAAGTPCOTGT2CGTGACGGCCAC 525 527 531 537, 538 F EINIYIKIEML TEILMH I TMCMG A V L S V EEL L I G G EM I TTCAAGGGCAACTACAAGGCCCAGCTGACCAGGCTGAACACF2CACCAACTGCAATGGCGCCGTGCTGAGCGTGGAGGAGCTGCTGATCGGCGGCGAGATGATC MA GT L TL EE VMIRIKIFMMG E IMF (196 RESIDUE) AAAGCCGGOACCCTGACACTGGAGGAGGTGCGSCGCAAGTTCAACAACGGCGAGATCAACTTC OH BASES) POSITIONS MUTATED AND CHAREACTERIZED FOR IMPROVEMENTS IN ACTIVITY AND/OR SPECIFICITY 1-1 0 ACTIVE SITE RESIDUE RESIDUE MUTATED IN ELD/KKR VARIANTS 71' RESIDUE NUMBERS ATE INDICATED FOR ACTIVE SITE RESIDUES, ELD/KKR VARIANTS, AND HEAVILY MUTATED POSITIONS 71' 01 FIG. 1 O 00 (57) : Described herein are engineered nucleases comprising mutations in the cleavage domain (e.g., Fold or homologue 0 thereof) and/or DNA binding domain (zinc fmger protein, TALE, single guide RNA) such that on-target specificity is increased. o [Continued on next page] WO 2018/039448 Al MIDEDIMOMMIDIIMERINIMIIMMOHOHIMMEEM TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: with international search report (Art. 21(3)) before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h))
SG11201901364VA 2016-08-24 2017-08-24 Engineered target specific nucleases SG11201901364VA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662378978P 2016-08-24 2016-08-24
US201762443981P 2017-01-09 2017-01-09
PCT/US2017/048409 WO2018039448A1 (en) 2016-08-24 2017-08-24 Engineered target specific nucleases

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US (4) US10975393B2 (en)
EP (2) EP3964573A1 (en)
JP (2) JP7272948B2 (en)
KR (2) KR20220145913A (en)
CN (1) CN110418841A (en)
AU (1) AU2017315414B2 (en)
BR (1) BR112019003327A2 (en)
CA (1) CA3034884A1 (en)
ES (1) ES2901989T3 (en)
IL (2) IL264792B2 (en)
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WO (1) WO2018039448A1 (en)

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