SG11201906200WA - Nucleic acid-polypeptide compositions and methods of inducing exon skipping - Google Patents

Nucleic acid-polypeptide compositions and methods of inducing exon skipping

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Publication number
SG11201906200WA
SG11201906200WA SG11201906200WA SG11201906200WA SG11201906200WA SG 11201906200W A SG11201906200W A SG 11201906200WA SG 11201906200W A SG11201906200W A SG 11201906200WA SG 11201906200W A SG11201906200W A SG 11201906200WA SG 11201906200W A SG11201906200W A SG 11201906200WA
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SG
Singapore
Prior art keywords
jolla
suite
california
international
torrey pines
Prior art date
Application number
SG11201906200WA
Inventor
Arthur A Levin
Andrew John Geall
Venkata Ramana Doppalapudi
Michael Caramian COCHRAN
Hanhua Huang
Rob Burke
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Avidity Biosciences Llc
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Filing date
Publication date
Application filed by Avidity Biosciences Llc filed Critical Avidity Biosciences Llc
Publication of SG11201906200WA publication Critical patent/SG11201906200WA/en

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    • 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
    • A61K47/50—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
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    • A61K47/59—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
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    • A61K47/68—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 the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
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    • C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
    • C07K14/003—Peptide-nucleic acids (PNAs)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
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    • C—CHEMISTRY; METALLURGY
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    • C07K—PEPTIDES
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    • C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3513—Protein; Peptide
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    • C12N2320/33—Alteration of splicing

Abstract

FigA Gastrocnemius (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 12 July 2018 (12.07.2018) WIPOIPCT omit Oil ommll 0 II ion En oimIE (10) International Publication Number WO 2018/129384 Al (51) International Patent Classification: A61K 38/16 (2006.01) CO7K 7/08 (2006.01) A61K 47/64 (2017.01) CO7K 16/00 (2006.01) CO7K 7/06 (2006.01) (21) International Application Number: PCT/US2018/012672 (22) International Filing Date: 05 January 2018 (05.01.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/443,514 06 January 2017 (06.01.2017) US 62/561,939 22 September 2017 (22.09.2017) US (71) Applicant: AVIDITY BIOSCIENCES LLC [US/US]; 10975 North Torrey Pines Road, Suite 150, La Jolla, Cali- fornia 92037 (US). (72) Inventors: LEVIN, Arthur A.; 10975 North Torrey Pines Road, Suite 150, La Jolla, California 92037 (US). GEALL, Andrew John; 10975 North Torrey Pines Road, Suite 150, La Jolla, California 92037 (US). DOPPALAPUDI, Venka- ta Ramana; 10975 North Torrey Pines Road, Suite 150, La Jolla, California 92037 (US). COCHRAN, Michael Caramian; 10975 North Torrey Pines Road, Suite 150, La Jolla, California 92037 (US). HUANG, Hanhua; 10975 North Torrey Pines Road, Suite 150, La Jolla, California 92037 (US). BURKE, Rob; 10975 North Torrey Pines Road, Suite 150, La Jolla, California 92037 (US). (74) Agent: YANG, Xiaofan; Wilson Sonsini Goodrich & Rosati, 650 Page Mill Road, Palo Alto, California 94304 (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, (54) Title: NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON SKIPPING 1-1 71' 00 01 (57) : Disclosed herein are molecules and pharmaceutical compositions that induce an insertion, deletion, duplication, or al- teration in an incorrectly spliced mRNA transcript to induce exon skipping or exon inclusion. Also described herein include methods for treating a disease or disorder that comprises a molecule or a pharmaceutical composition that induces an insertion, deletion, duplication, O or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exon inclusion. C [Continued on next page] WO 2018/129384 Al MIDEDIMOMOIDEIREEMOMOHOHMINEMOIMEN 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, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(11)) 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)) with sequence listing part of description (Rule 5.2(a))
SG11201906200WA 2017-01-06 2018-01-05 Nucleic acid-polypeptide compositions and methods of inducing exon skipping SG11201906200WA (en)

Applications Claiming Priority (3)

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US201762443514P 2017-01-06 2017-01-06
US201762561939P 2017-09-22 2017-09-22
PCT/US2018/012672 WO2018129384A1 (en) 2017-01-06 2018-01-05 Nucleic acid-polypeptide compositions and methods of inducing exon skipping

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US (5) US10994020B2 (en)
EP (1) EP3565577A4 (en)
JP (2) JP2020505330A (en)
KR (1) KR20190104381A (en)
CN (1) CN110381980A (en)
AU (2) AU2018205259B2 (en)
CA (1) CA3049424A1 (en)
IL (1) IL267793A (en)
MX (1) MX2019008199A (en)
SG (2) SG11201906200WA (en)
WO (1) WO2018129384A1 (en)

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MA45328A (en) 2016-04-01 2019-02-06 Avidity Biosciences Llc NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
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