Patent SG-11201804375W-A (SG)
Title: Compositions and methods for internalizing enzymes
Inventor: BAIK ANDREW
IPC: C12N9/42
Filed: 2016-12-08
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -' Organization International Bureau (43) International Publication Date .... ....r .„,„1 15 June 2017(15.06.2017) WIPO I PCT (10) WO International 111111111111311111111111111111111111111111111111111111111111111111111111111111111111111111 2017/100467 Publication A4 Number (51) International Patent Classification: DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, A61K 38/47 (2006.01) C12N 9/42 (2006.01) HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, A61K 38/46 (2006.01) A61P 43/00 (2006.01) KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, C12N 9/40 (2006.01) MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, (21) International Application Number: RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, PCT/US2016/065647 TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, (22) International Filing Date: ZA, ZM, ZW. 8 December 2016 (08.12.2016) (84) Designated States (unless otherwise indicated, for every (25) Filing Language: English kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, (26) Publication Language: English TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, (30) Priority Data: TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, 62/264,702 8 December 2015 (08.12.2015) US DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, 62/379,629 25 August 2016 (25.08.2016) US 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, (71) Applicant: REGENERON PHARMACEUTICALS, GW, KM, ML, MR, NE, SN, TD, TG). INC. [US/US]; 777 Old Saw Mill River Road, Tarrytown, New York 10591 (US). Declarations under Rule 4.17: (72) Inventors: BAIK, Andrew; 777 Old Saw Mill River of inventorship (Rule 4.17(iv)) Road, Tarrytown, New York 10591 (US). CYGNAR, Published: Katherine; 777 Old Saw Mill River Road, Tarrytown, — with international search report (Art 21(3)) New York 10591 (US). — with amended claims (Art 19(1)) (74) Agent: ZAHNER, Joseph; Kutak Rock LLP, 2300 Main Street, Suite 800, Kansas City, Missouri 64108 (US). — with sequence listing part of description (Rule 5.2(a)) (81) Designated States (unless otherwise indicated, for every (88) Date of publication of the international search report: 20 July 2017 kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, Date of publication of the amended claims: 21 September 2017 BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, ll• .4t IN C ) i t C:: , C:: , l'I IN l'I 0 (54) Title: COMPOSITIONS AND METHODS FOR INTERNALIZING ENZYMES el 0 (57) : Compositions and methods for treating lysosomal storage diseases are disclosed. Biotherapeutic complexes contain- ing an internalization effector binding domain and a lysosomal replacement enzyme activity are disclosed. The biotherapeutic com - plexes are capable of entering cells, segregating to the lysosome, and delivering the replacement enzyme activity to the lysosome.