SG11201910183SA - C-terminal cdnf and manf fragments, pharmaceutical compositions comprising same and uses thereof - Google Patents

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 08 November 2018 (08.11.2018) WIPO I PCT onion ono o ioo lII 1111111 ow (10) International Publication Number WO 2018/202957 Al (51) International Patent Classification: C07K 14/475 (2006.01) A61K 38/18 (2006.01) (21) International Application Number: PCT/FI2018/050332 (22) International Filing Date: 04 May 2018 (04.05.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 20175392 04 May 2017 (04.05.2017) FI (71) Applicant: HELSINGIN YLIOPISTO [FI/FI]; Fabi- aninkatu 33, 00014 Helsingin yliopisto (FI). (72) Inventors: SAARMA, Mart; c/o Helsingin yliopis- to, Fabianinkatu 33, 00014 Helsingin yliopisto (FI). AIRAVAARA, Mikko; c/o Helsingin yliopisto, Fabi- aninkatu 33, 00014 Helsingin yliopisto (FI). VOUTILAI- NEN, Merja; c/o Helsingin yliopisto, Fabianinkatu 33, 00014 Helsingin yliopisto (FI). YU, Li Ying; c/o Helsingin yliopisto, Fabianinkatu 33, 00014 Helsingin yliopisto (FI). LINDAHL, Maria; c/o Helsingin yliopisto, Fabianinkatu 33, 00014 Helsingin yliopisto (FI). (74) Agent: SEPPO LAINE OY; Itamerenkatu 3 A, 00180 Helsinki (FI). (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)) with sequence listing part of description (Rule 5.2(a)) 1-1 • (54) Title: C-TERMINAL CDNF AND MANF FRAGMENTS, PHARMACEUTICAL COMPOSITIONS COMPRISING SAME AND USES THEREOF c7 , (57) : The present invention provides a C-terminal CDNF fragment sequence or a sequence which has at least 90 % homology to ei said sequence. The C-terminal CDNF fragment protects ER stressed neurons, motoneurons and dopaminergic neurons and the fragment is capable of penetrating neuronal cell membrane as well as the blood-brain-barrier. The present invention further provides said fragment ▪ and pharmaceutical compositions comprising said fragment for use in treatments of degenerative diseases and disorders including central nervous system diseases, diabetes and retinal disorders. The present invention is also providing a C-terminal MANF fragment 1-1 sequence or a sequence which has at least 90 % homology to the said sequence and pharmaceutical compositions comprising said MANF fragment for use in the treatment of degenerative diseases and disorders including central nervous system diseases, diabetes 0 and retinal disorders.