SG11201807400TA - Recombinant follistatin-fc fusion proteins and use in treating duchenne muscular dystrophy - Google Patents

Abstract

1aQ 150 time (h) FS315K75N/C77T1K82T-hFcLALA FS315K(75,31,82)D-hFcLALA FS315K(76,81,82)E-hFcLALA -6- F5315WT-hFc 200 1000 100 U (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (10) International Publication Number (43) International Publication Date WO 2017/152090 A3 8 September 2017 (08.09.2017) WIPO I PCT (51) International Patent Classification: C07K 14/47 (2006.01) A61K 38/17 (2006.01) A61P 21/00 (2006.01) A61K 38/22 (2006.01) (21) International Application Number: PCT/US2017/020723 (22) International Filing Date: 3 March 2017 (03.03.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/303,954 4 March 2016 (04.03.2016) US (71) Applicant: SHIRE HUMAN GENETIC THERAPIES, INC. [US/US]; 300 Shire Way, Lexington, MA 02421 (US). (72) Inventors: ISKENDERIAN, Andrea; c/o Shire Human Genetic Therapies, Inc., 300 Shire Way, Lexington, MA 02421 (US). NORTON, Angela W.; c/o Shire Human Ge- netic Therapies, Inc., 300 Shire Way, Lexington, MA 02421 (US). SHEN, Chuan; c/o Shire Human Genetic Therapies, Inc., 300 Shire Way, Lexington, MA 02421 (US). PAN, Clark; c/o Shire Human Genetic Therapies, Inc., 300 Shire Way, Lexington, MA 02421 (US). (74) Agents: CHEN, Fangli et al.; Proskauer Rose LLP, One International Place, Boston, MA 02110 (US). (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 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). Decl arations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.1700) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Publ ished: with international search report (Art. 21(3)) before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) (88) Date of publication of the international search report: 19 October 2017 (54) Title: RECOMBINANT FOLLISTATIN-FC FUSION PROTEINS AND USE IN TREATING DUCHENNE MUSCULAR = DYSTROPHY PK Study CD-1 mice 1 malkg IV alma° Figure 1A (57) : The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes ad- ministering to an individual who is suffering from or susceptible to DMD an effective amount of a recombinant follistatin fusion protein such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset. 1111111111111101110101011111010111110 011111111010110111101111111111111110111111 W O 20 17 / 15 2090 A3