Patent SG-11201809684Y-A (SG)
Title: Adeno-associated virus variant capsids and methods of use thereof
Inventor: KIRN DAVID
IPC: C12Q1/70
Filed: 2017-05-12
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 11111111111111111111101011111111111 01110111110111111011111111111111111011110111111 Organization International Bureau (10) International Publication Number (43) International Publication Date ......0\"\"1 WO 2017/197355 A3 16 November 2017 (16.11.2017) WIPO I PCT (51) (21) International Application Number: (22) International Filing Date: (25) Filing Language: (26) Publication Language: (30) International Patent Classification: (71) Applicant: 4D MOLECULAR THERAPEUTICS INC. C07K 14/075 (2006.01) C12N 15/63 (2006.01) [US/US]; 5980 Horton Street, Suite 460, Emeryville, CA C12Q 1/70 (2006.01) C12N 15/861 (2006.01) 94608 (US). C12N 15/62 (2006.01) (72) Inventors: KIRN, David, H.; 5980 Horton Street, Suite 460, Emeryville, CA 94608 (US). KOTTERMAN, Melis- PCT/US2017/032542 sa; 5980 Horton Street, Suite 460, Emeryville, CA 94608 (US). SCHAFFER, David; 5980 Horton Street, Suite 460, Emeryville, CA 94608 (US). 12 May 2017 (12.05.2017) (74) Agent: CLOUGH, David, W. et al.; Polsinelli, PC, 150 N. English Riverside Plaza, Suite 3000, Chicago, IL 60606-1599 (US). English (81) Designated States (unless otherwise indicated, for every Priority Data: kind of national protection available): AE, AG, AL, AM, 62/336,441 13 May 2016 (13.05.2016) US AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, 62/378,106 22 August 2016 (22.08.2016) US CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, 62/384,590 07 September 2016 (07.09.2016) US DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, 62/454,612 03 February 2017 (03.02.2017) US 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, Title: ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF FIGURE I (54) = = = (d) 1 \"i.,Vector Select10-, Vivo Selection: Nge 1 ( its Identfied 'Jut?! Hits Identified ..— : Provided herein are variant adeno-assoeiated virus (AAV) capsid proteins having one or more modifications in amino sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one more types of retinal cells as compared to the infectivity of the retinal cells by an AA V virion comprising the unmodified parental capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or cells of the retina for the treatment of retinal disorders and diseases. AAV = = = _ = = = = = = _ = en .4 in en acid N or v—I --.... capsid I N capsid v—I cz more ei O [Continued on next page] WO 2017/197355 A3 MIDEDIMOMOIDEIROIDEMOMOHMOIllOMMODEfin 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: — as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(ii)) — of inventorship (Rule 4.17(iv)) 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)) — with sequence listing part of description (Rule 5.2(a)) (88) Date of publication of the international search report: 28 December 2017 (28.12.2017)