Patent SG-11201901941Y-A (SG)
Title: High purity rna compositions and methods for preparation thereof
Inventor: HOGE STEPHEN
IPC: C12N15/10
Filed: 2017-09-14
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 111111111111111111111111111111111111111111111111111111111111111 111111111 01111 Organization International Bureau (43) International Publication Date . . .., . .. (10) International Publication Number WO 2018/053209 Al 22 March 2018 (22.03.2018) WIP0 I PCT (51) International Patent Classification: MA 02451 (US). BUTORA, Gabor; 200 Technology Cl 2N 15/10 (2006.01) Square, Cambridge, MA 02139 (US). (21) International Application Number: (74) Agent: LOCKHART, Helen C.; Wolf, Greenfield & PCT/US2017/051674 Sacks, P.C., 600 Atlantic Avenue, Boston, MA 02210-2206 (22) International Filing Date: (US). 14 September 2017 (14.09.2017) (81) Designated States (unless otherwise indicated, for every (25) Filing Language: English kind AO, of national protection available): AE, AG, AL, AM, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, (26) Publication Language: English CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, (30) Priority Data: DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, 62/394,711 14 September 2016 (14.09.2016) US 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, (71) Applicant: MODERNATX, INC. [US/US]; 320 Bent MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, Street, Cambridge, MA 02141 (US). 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, (72) Inventors: HOGE, Stephen; 320 Bent Street, Cambridge, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. MA 02141 (US). ISSA, William; 95 Kimball Road, Ded- ham, MA 02026 (US). MIRACCO, Edward J.; 6 Frost (84) Designated States (unless otherwise indicated, for every Street, Arlington, MA 02474 (US). NELSON, Jennifer; kind of regional protection available): ARIPO (BW, GH, — 465 Washington Street, #2, Brookline, MA 02246 (US). GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, RABIDEAU, Amy, E.; 121 Pond Street, Unit 1, Waltham, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, = = (54) Title: HIGH PURITY RNA COMPOSITIONS AND METHODS FOR PREPARATION THEREOF = = Figure 40 = = = = DNA plasmid = NTPs = = Linearized dsDNA template 2+ = Mg — = = = 5 'PPP - ,-- -\",, .........vappp 5' = 5 '13PP 5 'PPP -A ino NN... 3' Full-length product Run-on transcripts Reverse Abortive/ truncated transcripts 1 complement ,.._ impurities , = 1€ 5'PPP ;€€€€€€€ 5 'P P :.1 5'PPP EM 113 A ioo N N... 3' wituunsww-ppp 5' samonsitutu-ppp 5' Ennwansmeam-ppp 5' meWmammtata-ppp 5' ireamfamaros-PPP 5' CD . IMIH liJilli © ei PP P 5' Kw:mamma-pp p 5' M 0 i n (57) : The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are ---- particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by inproved *1: processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and 1-1 © purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof. ei O [Continued on next page] WO 2018/053209 Al MIDEDIMOMOIDEIREEMONIMEINEMECIONVOIS 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: — with international search report (Art. 21(3)) — with sequence listing part of description (Rule 5.2(a))