Patent SG-11201810256X-A (SG)
Title: Polynucleotides encoding relaxin
Inventor: TICHO BARRY
IPC: C07K19/00
Filed: 2017-05-18
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property C.--.` 11111111111111111111111111111111111111110101110111001111011011111011110111111 Organization International Bureau (10) International Publication Number 03 (43) International Publication Date ......°' WO 2017/201340 A2 23 November 2017 (23.11.2017) WIPO I PCT (51) International Patent Classification: Not classified (21) International Application (22) International Filing Date: (25) Filing Language: (26) Publication Language: (30) Priority Data: 62/338,470 18 May 2016 (18.05.2016) (71) Applicant: MODERNATX, Street, Cambridge, MA 02141 (US). (72) Inventor; and (71) Applicant: XIA, Zhinan Square, Cambridge, MA 02139 (US). (72) Inventors: TICHO, Barry; 200 Technology Square, Cam- bridge, MA 02139 (US). BRIANCON-ERIS, Nadege; Number: 200 Technology Square, Cambridge, MA 02139 (US). PCT/US2017/033411 DOUSIS, Athanasios; 2 Derne Street, #3, Boston, MA 02114 (US). DE PICCIOTTO, Seymour; 200 Technol- ogy Square, Cambridge, MA 02139 (US). PRESNYAK, 18 May 2017 (18.05.2017) Vladimir; 123 Webster Avenue #3, Cambridge, MA 02141 (US). HOGE, Stephen; 66 Summit Avenue, Brook- English line, MA 02446 (US). MCFADYEN, Iain; 11 Amherst English Street, Arlington, MA 02474 (US). BENENATO, Ker- ry; 415 Lincoln Road, Sudbury, MA 01776 (US). KU- MARASINGHE, Ellalahewage, Sathyajith; 15 Littleton US Road, 4B, Harvard, MA 01451 (US). INC. [US/US]; 320 Bent (74) Agent: LOCKHART, Helen, C.; Wolf, Greenfield & Sacks, P.C., 600 Atlantic Avenue, Boston, MA 02210-2206 (US). [US/US]; 200 Technology (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, ENCODING RELAXIN (Daylight cycle i Fig. 3A Heart averaged rate data, mean * +1- SD) = (54) Title: POLYNUCLEOTIDES 'Start of Treatment I 1 475- 450- 425- s a 400- i 375 350 325 300 275 t. /7 ` PN / 4 ,69 ,,a 4 ,r, 43 s oy 43 relates to in vivo, the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect in subjects. =feeeee vs. Luaferase 4. 0 . , / ` PN i s d i '' \ 0 ? / / / 1 1. e l ' , 1 3 I ' tiP cfl JP OP 4. tP ,.6 ,t, os- 83. a. , ,o, 4. 6 0 ,zy ,,.a. ,ty . 1 / 4 4:. 4. 6 4 ,6 ,r, „a 4. 6 4 3 43\"' , z , 9 4) s oy , 4 0 g o ,\" s o y 9 d o 9 0 4) s e 4 :1' g o ,z.' ic y day e ,z,0 e e •, .0 e ..z.'' •eo e e e Al , op 0 Al , ,t? OP en' ,,a. (a d' ) mRNAs for use in and fusion proteins efficient delivery to deficient levels of metabolites associated 1 — ...b ***, v0.001 — mo5LT Construct 1 mRNA — recombinant human RLN2 protein —mo5U wildtype hRLN2 mRNA — Luciferase naRN A (ml `P. no miR *\", p<0.01 — *, v0.05 el 7r en (57) : The invention Il the invention, when administered comprising relaxin, mRNAs of el ---- cells and/or tissues in subjects, when administered thereto, mRNA therapies of the invention increase and/or restore IN relaxin expression and/or activity in subjects, mRNA therapies of the invention further decrease levels of toxic © with deficient relaxin activity N C Note. dl rRLN2 data points are significantly different from siLk data points. mRNA therapy for the treatment of fibrosis and/or cardiovascular disease, encode human relaxin, isoforms thereof, functional fragments thereof, [Continued on next page] WO 2017/201340 A2 IMIOHOIIIThililill10010101110111 INIIHNHOIS 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. (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). Published: — without international search report and to be republished upon receipt of that report (Rule 48.2(g)) — with sequence listing part of description (Rule 5.2(a))