Patent SG-11201809543Q-A (SG)
Title: Treatment of complement-mediated disorders
Inventor: LACHMANN PETER
IPC: C07K14/47
Filed: 2017-05-03
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1111111011110111111111111110101110 III 0111110111111111110111011111110 III 0111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2017/194912 Al 16 November 2017 (16.11.2017) WIPO I PCT (51) International Patent Classification: MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, A61K 48/00 (2006.01) CO7K 14/47 (2006.01) TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). (21) International Application Number: PCT/GB2017/051233 Published: (22) International Filing Date: — with international search report (Art. 21(3)) 03 May 2017 (03.05.2017) — with sequence listing part of description (Rule 5.2(a)) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 1608046.7 09 May 2016 (09.05.2016) GB (71) Applicants: CAMBRIDGE ENTERPRISE LIMIT- ED [GB/GB]; The Old Schools, Trinity Lane, Cam- bridge Cambridgeshire CB2 1TN (GB). THE SYD- NEY CHILDREN'S HOSPITALS NETWORK (RAND- WICK AND WESTMEAD) [AU/AU]; (incorporating The Royal Alexandra Hospital For Children), Cnr Hawkesbury Road and Hainsworth Street, Westmead, New South Wales (AU). = (72) Inventors: LACHMANN, Peter; Department of Veteri- _ nary Medicine, University of Cambridge, Madingley Road, = Cambridge Cambridgeshire CB3 OES (GB). ALEXAN- DER, Ian; Children's Medical Research Institute, 214 Hawkesbury Road, Westmead, Sydney, New South Wales = 2145 (AU). (74) Agent: O'BRIEN Simon; D Young & Co LLP, 120 Hol- born, London, EC1N 2DY (GB). (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, 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 ARIPO GH, of regional protection available): (BW, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, — UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, 1-1 TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, .4 EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, ei ,-1 C:N (54) Title: TREATMENT OF COMPLEMENT-MEDIATED DISORDERS 71' C:N (57) : Methods of treatment of complement-mediated disorders, in particular disorders associated with over-activity of the 1-1 ---- complement C3b feedback cycle (for example, age-related macular degeneration (AMD)), using gene therapy is described. According IN to the methods, levels of complement Factor I are elevated by administration of a recombinant viral vector encoding Factor I such that a © therapeutically effective amount of the encoded Factor I is expressed from the vector in the subject. Recombinant viral vectors encoding N Factor I, recombinant virus particles encapsidating the vectors, and their use in the methods of treatment, is also described. O