Patent SG-11201906200W-A (SG)
Title: Nucleic acid-polypeptide compositions and methods of inducing exon skipping
Inventor: LEVIN ARTHUR A
IPC: C07K7/08
Filed: 2018-01-05
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))