SG11201810816QA - Modified rna encoding vegf-a polypeptides, formulations, and uses relating thereto - Google Patents

Modified rna encoding vegf-a polypeptides, formulations, and uses relating thereto

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Publication number
SG11201810816QA
SG11201810816QA SG11201810816QA SG11201810816QA SG11201810816QA SG 11201810816Q A SG11201810816Q A SG 11201810816QA SG 11201810816Q A SG11201810816Q A SG 11201810816QA SG 11201810816Q A SG11201810816Q A SG 11201810816QA SG 11201810816Q A SG11201810816Q A SG 11201810816QA
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SG
Singapore
Prior art keywords
international
molndal
astrazeneca
modified rna
formulations
Prior art date
Application number
SG11201810816QA
Inventor
Leif Karlsson Parinder
Danielson Regina Desirée Fritsche
Kenny Mikael Hansson
Li Ming Gan
Jonathan Clarke
Ann-Charlotte Eva Egnell
Kenneth Randall Chien
Original Assignee
Modernatx 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 Modernatx Inc filed Critical Modernatx Inc
Publication of SG11201810816QA publication Critical patent/SG11201810816QA/en

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/515—Angiogenesic factors; Angiogenin
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1858—Platelet-derived growth factor [PDGF]
    • A61K38/1866—Vascular endothelial growth factor [VEGF]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/12—Carboxylic acids; Salts or anhydrides thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0021—Intradermal administration, e.g. through microneedle arrays, needleless injectors
    • 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
    • A61K9/08—Solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/12—Antihypertensives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 111111101111011101111111101011111001111110101111101011111111111111111111111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\"\" WO 2017/214175 Al 14 December 2017 (14.12.2017) WIP0 I PCT (51) International Patent Classification: EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, C07K 14/52 (2006.01) A61K 31/7088 (2006.01) MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, A61K 47/02 (2006.01) A61K 39/00 (2006.01) TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, A61K 48/00 (2006.01) KM, ML, MR, NE, SN, TD, TG). (21) International Application Number: PCT/US2017/036188 Published: — with international search report (Art. 21(3)) (22) International Filing Date: — before the expiration of the time limit for amending the 06 June 2017 (06.06.2017) claims and to be republished in the event of receipt of (25) Filing Language: English amendments (Rule 48.2(h)) — with sequence listing part of description (Rule 5.2(a)) (26) Publication Language: English (30) Priority Data: 62/346,979 07 June 2016 (07.06.2016) US 62/411,091 21 October 2016 (21.10.2016) US 62/432,005 09 December 2016 (09.12.2016) US (71) Applicant: MODERNATX, INC. [US/US]; 200 Technol- ogy Square, Cambridge, Massachusetts 02139 (US). (72) Inventors: PARINDER, Leif Karlsson; AstraZeneca AB, SE-431 83 Molndal (SE). FRITSCHE DANIELSON, Regina Desiree; AstraZeneca AB, SE-431 83 Molndal (SE). HANSSON, Kenny Mikael; AstraZeneca AB, SE-431 83 Molndal (SE). GAN, Li Ming; AstraZeneca AB, SE-431 83 Molndal (SE). CLARKE, Jonathan; Karolin- ska Institutet, 35 Berzelius Vag, 171 65 Stockholm (SE). EGNELL, Ann-Charlotte Eva; AstraZeneca AB, SE-431 83 Molndal (SE). CHIEN, Kenneth Randall; Karolinska Institutet, 35 Berzelius Vag, SE 171 65 Stockholm (SE). Agent: CHAPMAN, Ernest F. et al.; Finnegan, Hender- son, Farabow, Garrett & Dunner, LLP, 901 New York Av- enue, N.W., Washington, District of Columbia 20001-4413 (74) (US). 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, 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. (81) ,_ 1 (84) ir) IN 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, 1-1 TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, 71' 1-1 ei N (54) Title: MODIFIED RNA ENCODING VEGF-A POLYPEPTIDES, FORMULATIONS, AND USES RELATING THERETO 1-1 0 (57) : The disclosure relates to modified RNA molecules encoding VEGF-A polypeptides and formulations comprising the ei modified RNA. Aspects of the disclosure further relate to preparations and uses of formulations comprising the modified RNA in 0 treating subjects suffering from diseases responsive to VEGF-A therapy.
SG11201810816QA 2016-06-07 2017-06-06 Modified rna encoding vegf-a polypeptides, formulations, and uses relating thereto SG11201810816QA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662346979P 2016-06-07 2016-06-07
US201662411091P 2016-10-21 2016-10-21
US201662432005P 2016-12-09 2016-12-09
PCT/US2017/036188 WO2017214175A1 (en) 2016-06-07 2017-06-06 Modified rna encoding vegf-a polypeptides, formulations, and uses relating thereto

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SG11201810816QA true SG11201810816QA (en) 2018-12-28

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US (1) US11866475B2 (en)
EP (2) EP3971204A1 (en)
JP (3) JP6859369B2 (en)
KR (4) KR102376245B1 (en)
CN (2) CN109790207B (en)
AU (2) AU2017277277B2 (en)
CA (1) CA3026500A1 (en)
CY (1) CY1124667T1 (en)
DK (1) DK3464338T3 (en)
ES (1) ES2895421T3 (en)
HR (1) HRP20211569T1 (en)
HU (1) HUE056055T2 (en)
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MA (1) MA45172B1 (en)
MX (1) MX2018015110A (en)
PL (1) PL3464338T3 (en)
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WO (1) WO2017214175A1 (en)

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MA45172B1 (en) 2021-11-30
KR20230107891A (en) 2023-07-18
HRP20211569T1 (en) 2022-01-21
SI3464338T1 (en) 2022-02-28
HUE056055T2 (en) 2022-01-28
KR20220038819A (en) 2022-03-29
KR102376245B1 (en) 2022-03-21
JP2019521976A (en) 2019-08-08
TW202218670A (en) 2022-05-16
CA3026500A1 (en) 2017-12-14
JP2021100961A (en) 2021-07-08
DK3464338T3 (en) 2021-10-18
MA45172A (en) 2019-04-10
MX2018015110A (en) 2019-09-06
CN116606861A (en) 2023-08-18
JP7167226B2 (en) 2022-11-08
IL263370A (en) 2018-12-31
ES2895421T3 (en) 2022-02-21
PL3464338T3 (en) 2021-12-27
AU2017277277B2 (en) 2023-05-04
US20200407411A1 (en) 2020-12-31
RU2018147229A3 (en) 2020-09-23
SG10202012175YA (en) 2021-01-28
AU2017277277A1 (en) 2019-01-03
US11866475B2 (en) 2024-01-09
EP3464338B1 (en) 2021-07-21
RU2018147229A (en) 2020-07-10
CN109790207A (en) 2019-05-21
CN109790207B (en) 2023-06-20
KR102494499B1 (en) 2023-01-31
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KR20230019505A (en) 2023-02-08
TW201808325A (en) 2018-03-16
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TWI818375B (en) 2023-10-11
PT3464338T (en) 2021-10-19
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