(12) International Application Status Report

Received at International Bureau: 13 October 2005 (13.10.2005)

Information valid as of: Not available

Report generated on: 15 May 2021 (15.05.2021)

(10) Publication number: (43) Publication date: (26) Publication language:
WO 2006/00756019 January 2006 (19.01.2006) English (EN)

(21) Application number: (22) Filing date: (25) Filing language:
PCT/US2005/02352901 July 2005 (01.07.2005) English (EN)

(31) Priority number(s): (32) Priority date(s): (33) Priority status:
60/584,648 (US)01 July 2004 (01.07.2004) Priority document received (in compliance with PCT Rule 17.1)

(51) International Patent Classification:
A61K 38/46 (2006.01); A61K 38/47 (2006.01); C12N 9/14 (2006.01); C12N 9/20 (2006.01); C07H 21/04 (2006.01)

(71) Applicant(s):
UNIVERSITY OF PENNSYLVANIA [US/US]; Center for Technology Transfer Suite 200 Philadelphia, PA 19104-6283 (US) (for all designated states except US)
MOUNT SINAI SCHOOL OF MEDICINE [US/US]; One Gustave L. Levy Place Box 1675 New York, NY 10029 (US) (for all designated states except US)
GALINDO, Silvia, Muro [ES/US]; 4247 Locust St., Apt. 402 Philadelphia, PA 19104 (US) (for US only)
MUZYKANTOV, Vlamidir, R. [US/US]; 101 White Pine Court Warminster, PA 18974 (US) (for US only)
SCHUCHMAN, Edward, Howard [US/US]; 300 Lakeshore Drive Haworth, NJ 07641 (US) (for US only)

(72) Inventor(s):
GALINDO, Silvia, Muro; 4247 Locust St., Apt. 402 Philadelphia, PA 19104 (US)
MUZYKANTOV, Vlamidir, R.; 101 White Pine Court Warminster, PA 18974 (US)
SCHUCHMAN, Edward, Howard; 300 Lakeshore Drive Haworth, NJ 07641 (US)

(74) Agent(s):
ABRAMS, Samuel, B.; Jones Day 222 East 41st Street New York, NY 10017-6702 (US)

(54) Title (EN): TARGETED PROTEIN REPLACEMENT FOR THE TREATMENT OF LYSOSOMAL STORAGE DISORDERS
(54) Title (FR): REMPLACEMENT DE PROTEINES CIBLEES POUR LE TRAITEMENT DE MALADIES LYSOSOMALES

(57) Abstract:
(EN): The present invention relates to compositions and methods for delivering lysosomal proteins. The compositions and methods described herein permit the targeted delivery of exogenous lysosomal proteins to cell surface proteins that allow their internalization via non-clathrin pathways. The present invention further relates to the use 10 of the compositions and methods for enzyme replacement therapy of lysosomal storage diseases. Nucleic acids, recombinant cells and kits useful for making and using the compositions of the invention are also provided.
(FR): La présente invention concerne des compositions et des méthodes de transport de protéines lysosomales. Les compositions et les méthodes de l'invention permettent d'acheminer de façon ciblée des protéines lysosomales exogènes vers des protéines de surface permettant leur internalisation par des voies autres que celles de la clathrine. L'invention concerne également l'utilisation desdites compositions et méthodes destinées au traitement par remplacement enzymatique de maladies lysosomales. Elle concerne en outre des acides nucléiques, des cellules recombinantes, et des trousses utiles servant à produire et mettre en oeuvre les compositions de l'invention.

International search report:
Received at International Bureau: 02 August 2006 (02.08.2006) [US]

International Report on Patentability (IPRP) Chapter II of the PCT:
Not available

(81) Designated States:
AE, AG, AL, AM, AT, AU, AZ, BA, BB, BG, BR, BW, BY, BZ, CA, CH, CN, CO, CR, CU, CZ, DE, DK, DM, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, HR, HU, ID, IL, IN, IS, JP, KE, KG, KM, KP, KR, KZ, LC, LK, LR, LS, LT, LU, LV, MA, MD, MG, MK, MN, MW, MX, MZ, NA, NG, NI, NO, NZ, OM, PG, PH, PL, PT, RO, RU, SC, SD, SE, SG, SK, SL, SM, SY, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, YU, ZA, ZM, ZW
European Patent Office (EPO) : AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HU, IE, IS, IT, LT, LU, LV, MC, NL, PL, PT, RO, SE, SI, SK, TR
African Intellectual Property Organization (OAPI) : BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, ML, MR, NE, SN, TD, TG
African Regional Intellectual Property Organization (ARIPO) : BW, GH, GM, KE, LS, MW, MZ, NA, SD, SL, SZ, TZ, UG, ZM, ZW
Eurasian Patent Organization (EAPO) : AM, AZ, BY, KG, KZ, MD, RU, TJ, TM