Patent SG-11201900049Q-A (SG)
Title: Crispr/cas9-based compositions and methods for treating retinal degenerations
Inventor: JASKULA-RANGA VINOD
IPC: C12N15/85
Filed: 2017-07-05
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1111111111111111111111111111111111111111111111111111111111111111111111111110111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2018/009562 Al 11 January 2018 (11.01.2018) W I PO I PCT (51) International Patent Classification: TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, A61K 48/00 (2006.01) Cl 2N 15/63 (2006.01) KM, ML, MR, NE, SN, TD, TG). A61K 38/46 (2006.01) Cl 2N 15/85 (2006.01) C12N 15/113 (2010.01) A61P 27/02 (2006.01) Published: C12N 9/16 (2006.01) — with international search report (Art. 21(3)) (21) International Application Number: PCT/US2017/040745 (22) International Filing Date: 05 July 2017 (05.07.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/358,337 05 July 2016 (05.07.2016) US (71) Applicant: THE JOHNS HOPKINS UNIVERSITY [US/US]; 3400 North Charles Street, Baltimore, MD 21218 (US). (72) Inventors: JASKULA-RANGA, Vinod; 1201 West Mount Royal Avenue, Unit 506, Baltimore, MD 21217 (US). ZACK, Donald; 3 Ruby Field Ct., Baltimore, MD 21209 (US). BUNZ, Fred; 10 Roland Mews, Baltimore, MD 21210 (US). WELSBIE, Derek; 400 North Broadway, Smith Building, Room 3035, Baltimore, MD 21231 (US). Agent: RUSSELL, Hathaway P. et al.; Foley Hoag (74) LLP, Seaport West, 155 Seaport Boulevard, Boston, MA — 02210-2600 (US). 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. (81) 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, (84) = 1-1 ei i ln (54) Title: CRISPR/CAS9-BASED COMPOSITIONS AND METHODS FOR TREATING RETINAL DEGENERATIONS 01 © (57) : Described herein are methods for treating a retinal degeneration in a subject, such as Leber's congenital amaurosis 0 --. (LCA), retinitis pigmentosa (RP), and glaucoma. Also provided herein are methods of altering expression of one or more gene products GC 1-1 in a cell, such as a retinal ganglion cell. Such methods may comprise utilizing a modified nuclease system, such as Clustered Regu- larly Interspaced Short Palindromic Repeats (CRISPR) system comprising a bidirectional HI promoter and gRNAs directed to retinal N degeneration related genes, packaged in a single, compact adeno-associated virus (AAV) particle. C