SG11201803934YA - Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies - Google Patents

Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies

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Publication number
SG11201803934YA
SG11201803934YA SG11201803934YA SG11201803934YA SG11201803934YA SG 11201803934Y A SG11201803934Y A SG 11201803934YA SG 11201803934Y A SG11201803934Y A SG 11201803934YA SG 11201803934Y A SG11201803934Y A SG 11201803934YA SG 11201803934Y A SG11201803934Y A SG 11201803934YA
Authority
SG
Singapore
Prior art keywords
international
rule
compositions
expression
biologically active
Prior art date
Application number
SG11201803934YA
Inventor
Dimki S Patel
Amit N Patel
Original Assignee
Intrexon Corp
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Publication date
Application filed by Intrexon Corp filed Critical Intrexon Corp
Publication of SG11201803934YA publication Critical patent/SG11201803934YA/en

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4728Calcium binding proteins, e.g. calmodulin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1738Calcium binding proteins, e.g. calmodulin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18Growth factors; Growth regulators
    • A61K38/1858Platelet-derived growth factor [PDGF]
    • A61K38/1866Vascular endothelial growth factor [VEGF]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19Cytokines; Lymphokines; Interferons
    • A61K38/195Chemokines, e.g. RANTES
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K41/00Medicinal preparations obtained by treating materials with wave energy or particle radiation ; Therapies using these preparations
    • A61K41/0028Disruption, e.g. by heat or ultrasounds, sonophysical or sonochemical activation, e.g. thermosensitive or heat-sensitive liposomes, disruption of calculi with a medicinal preparation and ultrasounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475Growth factors; Growth regulators
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52Cytokines; Lymphokines; Interferons
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52Cytokines; Lymphokines; Interferons
    • C07K14/521Chemokines
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/50Fusion polypeptide containing protease site
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/002Vectors comprising a special translation-regulating system controllable or inducible
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/007Vectors comprising a special translation-regulating system cell or tissue specific

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property - Organization International Bureau irestYlid (43) International Publication Date ..... ..sr ,„,„01 18 May 2017 (18.05.2017) WIPO I PCT (10) WO International Publication Number 111111111111311111111111111111111111111111111111111111111111111111111311111111111111111 2017/083750 Al (51) International Patent Classification: KW, C07K 14/52 (2006.01) A61K 48/00 (2006.01) MG, A61K 38/16 (2006.01) C12N 15/62 (2006.01) OM, SC, (21) International Application Number: TN, PCT/US2016/061668 ZW. (22) International Filing Date: (84) Designated 11 November 2016 (11.11.2016) kind (25) Filing Language: English GM, TZ, (26) Publication Language: English TJ, (30) Priority Data: DK, 62/254,139 11 November 2015 (11.11.2015) US LV, SM, KZ, LA, LC, LK, LR, LS, MK, MN, MW, MX, MY, PA, PE, PG, PH, PL, PT, SD, SE, SG, SK, SL, SM, TR, TT, TZ, UA, UG, States (unless otherwise of regional protection available): KE, LR, LS, MW, MZ, UG, ZM, ZW), Eurasian (AM, TM), European (AL, AT, EE, ES, FI, FR, GB, GR, MC, MK, MT, NL, NO, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, LU, LY, MA, MD, ME, MZ, NA, NG, NI, NO, NZ, QA, RO, RS, RU, RW, SA, ST, SV, SY, TH, TJ, TM, US, UZ, VC, VN, ZA, ZM, indicated, for every ARIPO (BW, GH, NA, RW, SD, SL, ST, SZ, AZ, BY, KG, KZ, RU, BE, BG, CH, CY, CZ, DE, HR, HU, IE, IS, IT, LT, LU, PL, PT, RO, RS, SE, SI, SK, (71) Applicant: INTREXON CORPORATION [US/US]; GW, KM, ML, MR, NE, SN, TD, 20358 Seneca Meadows Pkwy, Germantown, Maryland 20876 (US). Declarations (72) Inventors: PATEL, Dimki S ; 20358 Seneca Meadows as under Rule 4.17: to applicant's entitlement to TG). apply for and be granted a Pkwy, Germantown, Maryland 20876 (US). PATEL, Amit patent (Rule 4.17(ii)) N.; 1040 Chartwell Ct., Salt Lake City, Utah 84103 (US). — as earlier application (Rule 4.17(iii)) to the applicant's entitlement to claim the priority of the (74) Agent: GIRINATH, Prashant; Wilson Sonsini Goodrich & Rosati, 650 Page Mill Road, Palo Alto, California 94304 Published: (US). — with international search report (Art 21(3)) Designated States indicated, (81) (unless otherwise for every _ before the expiration of the time limit for amending the kind of national available): AE, AG, AL, AM, protection claims and to be republished in the event of receipt of AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, amendments (Rule 48.2(h)) 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, with sequence listing part of description (Rule 5.2(a)) HN, HR, HU, ID, IL, IN, IR, IS, KE, KG, KN, KP, KR, JP, (54) Title: COMPOSITIONS AND METHODS FOR EXPRESSION OF MULTIPLE BIOLOGICALLY ACTIVE POLY- PEPTIDES FROM A SINGLE VECTOR FOR TREATMENT OF CARDIAC CONDITIONS AND OTHER PATHOLOGIES ORI ter -2 Car d iac Il .4t C un N en cc o FIG. I IN Il C (57) : The present invention provides compositions and methods useful for treating disorders amenable to therapy via intro - N duction of multigenic expression vectors. More particularly, the invention provides vectors and polynucleotides encoding poly- 0 peptides for treatment of cardiac disorders wherein said polypeptides may comprise a cytokine, a chemokine, and/or an angiogenic ,..I polypeptide, or functional derivatives thereof. Also provided, as compositions of the invention, are linkers useful for connecting and expressing functional (biologically active) polypeptides from single, multigenic-expression constructs.
SG11201803934YA 2015-11-11 2016-11-11 Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies SG11201803934YA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562254139P 2015-11-11 2015-11-11
PCT/US2016/061668 WO2017083750A1 (en) 2015-11-11 2016-11-11 Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies

Publications (1)

Publication Number Publication Date
SG11201803934YA true SG11201803934YA (en) 2018-06-28

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Country Status (14)

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US (2) US11154622B2 (en)
EP (1) EP3387010A1 (en)
JP (1) JP6976244B2 (en)
KR (1) KR20180069081A (en)
CN (1) CN108713025A (en)
AU (1) AU2016353342B2 (en)
BR (1) BR112018009645A2 (en)
CA (1) CA3004742A1 (en)
HK (1) HK1259137A1 (en)
MA (1) MA43404A (en)
MX (1) MX2018005886A (en)
RU (1) RU2758489C2 (en)
SG (1) SG11201803934YA (en)
WO (1) WO2017083750A1 (en)

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CA3004742A1 (en) 2017-05-18
HK1259137A1 (en) 2019-11-29
AU2016353342B2 (en) 2021-04-01
BR112018009645A2 (en) 2018-11-13
US11154622B2 (en) 2021-10-26
JP2018536405A (en) 2018-12-13
AU2016353342A1 (en) 2018-05-24
US20180360992A1 (en) 2018-12-20
WO2017083750A1 (en) 2017-05-18
RU2018117674A (en) 2019-12-13
MX2018005886A (en) 2018-08-15
US20220072159A1 (en) 2022-03-10
JP6976244B2 (en) 2021-12-08
MA43404A (en) 2018-10-17
EP3387010A1 (en) 2018-10-17
KR20180069081A (en) 2018-06-22
RU2018117674A3 (en) 2020-04-20
RU2758489C2 (en) 2021-10-28
CN108713025A (en) 2018-10-26

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