Patent SG-11201909814W-A (SG)
Title: Gene therapy
Inventor: NALDINI LUIGI
IPC: A61K48/00
Filed: 2018-04-20
Abstract: 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) WIPO I PCT oimiolollm °nolo Oil mum olom Env oimIE (10) International Publication Number WO 2018/193119 Al (51) International Patent Classification: C12N 5/0789 (2010.01) A61K 38/21 (2006.01) C12N 5/0786 (2010.01) A61K 39/395 (2006.01) C12N 5/10 (2006.01) A61K 48/00 (2006.01) A61K 31/405 (2006.01) A61P 35/00 (2006.01) A61K 35/15 (2015.01) (21) International Application Number: PCT/EP2018/060238 (22) International Filing Date: 20 April 2018 (20.04.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 1706410.6 21 April 2017 (21.04.2017) GB 1801511.5 30 January 2018 (30.01.2018) GB (71) Applicants: OSPEDALE SAN RAFFAELE S.R.L. [IT/IT]; Via Olgettina 60, 20132 Milan (IT). FON- DAZIONE TELETHON [IT/IT]; Via Varese 16B, 00185 Rome (IT). (72) Inventors: NALDINI, Luigi; San Raffaele Telethon Insti- tute for Gene Therapy (TIGET), Via Olgettina 58, 20132 Milan (IT). ESCOBAR, Giulia; San Raffaele Telethon In- stitute for Gene Therapy (TIGET), Via Olgettina 58, 20132 Milan (IT). GENTNER, Bernhard Rudolf; San Raffaele Telethon Institute for Gene Therapy (TIGET), Via Olget- tina 58, 20132 Milan (IT). MUCCI, Adele; San Raffaele Telethon Institute for Gene Therapy (TIGET), Via Olgetti- na 58, 20132 Milan (IT). (74) Agent: D YOUNG & CO LLP; 120 Holborn, 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, 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. (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)) 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)) (54) Title: GENE THERAPY (57) : A hematopoietic stem cell (HSC), a hematopoietic progenitor cell (HPC) a myeloid/monocyte-committed progenitor cell, a macrophage or a monocyte comprising a vector wherein the vector comprises at least one mir-130a and/or mir-126 target sequence operably linked to a nucleotide sequence encoding interferon-alpha (IFNa) for use in treating or preventing a cancer in a patient, wherein the HSC, the HPC, the myeloid/monocyte-committed progenitor cell, the macrophage or the monocyte is used in combination with an immune checkpoint inhibitor and/or a tumor associated antigen (TAA)-specific T-cell.