Patent SG-11201808440Y-A (SG)
Title: Treatment of ocular diseases with fully-human post-translationally modified anti-vegf fab
Inventor: SIMPSON CURRAN
IPC: A61K48/00
Filed: 2017-04-14
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) ---' (19) World Intellectual Property Organization 11111111011110111010101111101011111011101111111101011010111011111101111011111 International Bureau 0.. .jd (10) International Publication Number (43) International Publication Date ;.;..;; ...., WO 2017/181021 Al 19 October 2017(19.10.2017) WIPO I PCT (51) International Patent Classification: (72) Inventors: SIMPSON, Curran, Matthew; 2149 Thurston A61K 39/395 (2006.01) C07K 16/22 (2006.01) Road, Frederick, MD 21704 (US). YOO, Stephen; 5521 A61K 48/00 (2006.01) C12N 15/13 (2006.01) Southwick Street, Bethesda, MD 20817 (US). KOZ- A61P 27/02 (2006.01) ARSKY, Karen, Fran; 56 E Levering Mill Road, Bala (21) International Application Number: Cynwyd, PA 19004 (US). REINHARDT, Rickey, Robert; PCT/US2017/027650 108 Apple Grove Road, Silver Spring, MD 20904 (US). CORUZZI, Laura, A.; 70 East 10th Street, Apartment (22) International Filing Date: 15M, New York, NY 10003 (US). 14 April 2017 (14.04.2017) (74) Agents: MARTINEK, Sebastian et al.; Jones Day, 250 (25) Filing Language: English Vesey Street, New York, NY 10281-1047 (US). (26) Publication Language: English (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (30) Priority Data: AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, 62/323,285 15 April 2016 (15.04.2016) US BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, 62/442,802 5 January 2017 (05.01.2017) US DO, DZ, EC, EE, EG, ES, FE GB, GD, GE, GH, GM, GT, 62/450,438 25 January 2017 (25.01.2017) US HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, 62/460,428 17 February 2017 (17.02.2017) US KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, (71) Applicant: REGENXBIO INC. [US/US]; 9712 Medical MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, Center Drive, Suite 100, Rockville, MD 20850 (US). 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, [Continued on next page] Title: TREATMENT OF OCULAR DISEASES WITH FULLY-HUMAN POST-TRANSLATIONALLY MODIFIED AN- (54) - TI-VEGF FAB (57) : Compositions and methods are described for the delivery of a Amino Acid Sequence of Ranibizumabltevackumab Fab Heavy Chain fully human post- translationally modified (HuPTM) monoclonal antibody 2U 30 90 30 60 (\"mAb\") or the antigen-binding fragment of a mAb against human vascular —>V8 EVQLVESGGGIVQPGGSLRLSCAASGYDF_HI , giVitQAPH&G,EWVG/t 1NTY 100212 endothelial factor (\"hVEGF\") - such as, e.g., a fully human-glyc - growth T T osylated (HuGly) anti-hVEGF antigen-binding fragment - to the retina/vitreal o 80 90 100 110 120 humour in the eye(s) of human subjects diagnosed with ocular diseases AAnKRAY0J3LDTSASTAY_IQYASLRELDIAvIYCAKYPYnG102n1LiVAG]ELVT by increased for example, neovascular age-related caused neovascularization, Y-siLe j-sli, 11 S macular degeneration (\"nAMD\"), also known as \"wet\" age-related macular i :io 190 180 160 180 degeneration (\"WAMD\"), age-related macular degeneration (\"AMD\"), and ->c. diabetic VSSASHGPSVI2LAPSSKSL'SGGIASIGC,VHDYYPEPVL\"IStiltEal SGV,ITg PAU retinopathy. G-site ' 9() '00 710 730 QSSGLYSISST VTVPSSSLC:QTYICITVNIIKP7N'2KVDKKVICKSCDKTEL Amino Acid Sequence of Ranibizumabltevacizumab Fab Light Chain 10 20 30 40 30 60 —>V H 01;21,0Q3SSLSASVGLRVIA . ,13A3W1SNYLNWUQKPGAAPKVLin03SLHSGVP3 M /0 80 90 100 110 120 +0, RFSGSGSGTDFTT.T T SS 1,01016126AT 0' -1 .QQY,S^VPW T FG2)STKV7TK2TVAAFSVFT FPP -elte 9-9 .2e Il 130 190 150 160 170 180 7.17.QTRSCTASVVOTTNNFYPRF.AKVQWKV1:19AT cSqS ' VTF.OSKDSTYST SSTT T Il G-Erre N © 190 200 210 11 C LSKADYEKEKVYACEVTI-WISSPV: SFNE839: ..- 11 ....... FIGURE 1 11 © P1 C WO 2017/181021 Al IIMMIMOMOIDEIRMEMOMOIEHOEHHHOIRVOIME (84) TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. Designated States (unless otherwise indicated, for every 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). kind of regional protection available): ARIPO (BW, GH, Published: GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, — TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, with international search report (Art. 21(3)) TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, — with sequence listing part of description (Rule 5.2(a)) DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT,