Patent SG-11201804713V-A (SG)
Title: TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
Inventor: DEVERMAN BENJAMIN
IPC: C07K14/005
Filed: 2016-12-09
Abstract: Title: TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs) AAV-PHRS 1 x 10' vg AAV-PHPeB 1 x 10 1 vg AAV-PHRB 1 x 10 1 vg AAV9 1x 10' 2 vg FIG. 23A (57) : Disclosed herein are peptide sequences capable of directing adeno-associated viruses (AAV) to target specific envir- onments, for example the nervous system and the heart, in a subject. Also disclosed are AAVs having non-naturally occurring capsid proteins comprising the disclosed peptide sequences, and methods of using the AAVs to treat diseases. 999900 ASH (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau 111111111111110 III OE III OE III II III 1111111111111111111111111 RIII 11111111 (10) International Publication Number WO 2017/100671 Al WIPO I PCT (43) International Publication Date 15 June 2017 (15.06.2017) (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, 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). Declarations under Rule 4.17: (51) International Patent Classification: C12N 15/86 (2006.01) C07K 14/005 (2006.01) CO7K 7/06 (2006.01) (21) International Application Number: PCT/US2016/065969 (22) International Filing Date: 9 December 2016 (09.12.2016) English English (25) Filing Language: (26) Publication Language: (30) Priority Data: 62/266,184 11 December 2015 (11.12.2015) 62/421,891 14 November 2016 (14.11.2016) US US (71) Applicant: CALIFORNIA INSTITUTE OF TECHNO- LOGY [US/US]; Office Of Technology Transfer, 1200 E. California Blvd., Mail Code 6-32, Pasadena, CA 91125 (US). (72) Inventors: DEVERMAN, Benjamin, E.; c/o California Institute of Technology, 1200 E. California Blvd., Mail Code 6-32, Pasadena, CA 91125 (US). GRADINARU, Viviana; c/o California Institute of Technology, 1200 E. California Blvd., Mail Code 6-32, Pasadena, CA 91125 (US). CHAN, Ken, Y.; c/o California Institute of Techno- logy, 1200 E. California Blvd., Mail Code 6-32, Pasadena, CA 91125 (US). (74) Agent: ALTMAN, Daniel E.; Knobbe, Martens, Olson & Bear, LLP, 2040 Main Street, 14th Floor, Irvine, CA 92614 (US). as to applicant's entitlement to apply for and be granted a patent (Rule 4.170) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Published: with international search report (Art 21(3)) with sequence listing part of description (Rule 5.2(a)) W O 20 17 / 10067 1 Al