Patent SG-11201909440U-A (SG)
Title: Delivery of autologous cells comprising matrix metalloproteinase for treatment of scleroderma
Inventor: THOMAS DARBY
IPC: C12N5/07
Filed: 2018-04-20
Abstract: IndaElePrOptil f. Gene Progral O O C (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 25 October 2018 (25.10.2018) %NIP() I PCT omit IIl °nolo mu loom Ho 11011111° oimIE (10) International Publication Number WO 2018/195410 Al (51) International Patent Classification: C07K 14/435 (2006.01) C12N 9/50 C07K 14/705 (2006.01) C12N 9/64 C07K 16/28 (2006.01) C12N 5/06 A61K 35/36 (2015.01) C12N 5/07 A61K 38/17 (2006.01) (21) International Application Number: PCT/US2018/028551 (22) International Filing Date: 20 April 2018 (20.04.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/488,207 21 April 2017 (21.04.2017) US 62/512,382 30 May 2017 (30.05.2017) US (71) Applicant: INTREXON CORPORATION [US/US]; 1750 Kraft Drive, Suite 1400, Blacksburg, VA 24060 (US). (72) Inventors: THOMAS, Darby; 1750 Kraft Drive, Suite 1400, Blacksburg, VA 24060 (US). MASLOWSKI, John; 405 Eagleview Blvd., Exton, PA 19341 (US). MALYALA, Anna; 405 Eagleview Blvd., Exton, PA 19341 (US). (74) Agent: SIEVER, Doyle, A. et al.; Intrexon Corporation, 20358 Seneca Meadows Parkway, Germantown, MD 20876 (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, JO, 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. (2006.01) (2006.01) (2006.01) (2010.01) (54) Title: DELIVERY OF AUTOLOGOUS CELLS COMPRISING MATRIX METALLOPROTEINASE FOR TREATMENT OF SCLERODERMA FIGURE 1 Schematic - One example of a RHEOSWITCH THERAPEUTIC SYSTEM (RTS) gene switch (57) : The present invention relates to a method for the treatment of scleroderma through the delivery of matrix metallopro- teinase (MMP) to a patient in need thereof, preferably under the control of a gene switch. In this manner, the use of a ligand activator to activate or deactivate the expression of MMP controls the gene switch. In another embodiment, the invention is directed to the delivery of autologous genetically modified cells transfected/transduced with a polynucleotide encoding MMP under the control of a gene switch activatable through the use of an activator ligand for the treatment or scleroderma. [Continued on next page] WO 2018/195410 Al O II (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). Published: — with international search report (Art. 21(3)) — with sequence listing part of description (Rule 5.2(a))