Patent SG-11201811603W-A (SG)
Title: Novel adeno-associated virus capsid proteins
Inventor: COLOSI PETER
IPC: C12N15/86
Filed: 2017-07-25
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property MD 1101111 0 DOI HIM 011101 0 01111111111111111111111111111111111111111111111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2018/022608 A3 01 February 2018 (01.02.2018) W I P0 I P C T (51) International Patent Classification: C07K 14/005 (2006.01) A61K 48/00 (2006.01) Published: C12N 15/86 (2006.01) — with international search report (Art 21(3)) — before the expiration of the time limit for amending the (21) International Application Number: claims and to be republished in the event of receipt of PCT/US2017/043703 amendments (Rule 48.2(h)) (22) International Filing Date: — with sequence listing part of description (Rule 5.2(a)) 25 July 2017 (25.07.2017) (88) Date of publication of the international search report: 01 March 2018 (01.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/366,838 26 July 2016 (26.07.2016) US (71) Applicant: BIOMARIN PHARMACEUTICAL INC. [US/US]; 105 Digital Drive, Novato, CA 94949 (US). (72) Inventors: COLOSI, Peter, Cameron; 105 Digital Drive, Novato, CA 94949 (US). LOCHRIE, Michael; 105 Digital Drive, Novato, CA 94949 (US). NG, Robert; 105 Digital Drive, Novato, CA 94949 (US). Agent: SINTICH, Sharon, M.; Marshall, Gerstein & = (74) Borun LLP, 233 S. Wacker Drive, 6300 Willis Tower, Chicago, IL 60606-6357 (US). — 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, (81) = = = _ 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. _ Designated States (unless otherwise indicated, for every — (84) _ _ 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). = M GC © ei ei © (54) Title: NOVEL ADENO-ASSOCIATED VIRUS CAPSID PROTEINS --.... ,-1 (57) : The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid 0 protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins. The invention also relates to N methods of making the herein described AAV vectors expressing the novel capsid proteins of the invention and associated therapeutic 0 uses of thereof.