Patent SG-11201901757Y-A (SG)
Title: Pd-1 homing endonuclease variants, compositions, and methods of use
Inventor: MANN JASDEEP
IPC: C12N15/90
Filed: 2017-09-08
Abstract: PDI W O 18/0 49 226 Al FIGURE lA TI M PD1.exon5.ITSM I ITSM (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 15 March 2018 (15.03.2018) WIPO I PCT iiimmommionotiolooluiolomomomovoil# (10) International Publication Number WO 2018/049226 Al (51) International Patent Classification: C12N 15/66 (2006.01) CO7K 16/30 (2006.01) C12N 15/09 (2006.01) C07K 14/54 (2006.01) C12N 5/10 (2006.01) C07K 16/28 (2006.01) C12N 9/22 (2006.01) A61K 38/46 (2006.01) C12N 15/90 (2006.01) A61K 35/17 (2015.01) (21) International Application Number: PCT/US2017/050774 (22) International Filing Date: 08 September 2017 (08.09.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/385,079 08 September 2016 (08.09.2016) US 62/414,279 28 October 2016 (28.10.2016) US (71) Applicant: BLUEBIRD BIO, INC. [US/US]; 60 Binney Street, Cambridge, Massachusetts 02142 (US). (72) Inventors: MANN, Jasdeep; 1415 2nd Ave, Apt #2002, Seattle, Washington 98101 (US). GAY, Joel; 141 N 77th St., Seattle, Washington 98103 (US). JARJOUR, Jordan; 11014 37th Ave SW, Seattle, Washington 98146 (US). ZHANG, Joy; 7745 40th Ave. NE, Seattle, Washington 98115 (US). (74) Agent: MASHRUWALA, Mary Anne et al.; Cooley LLP, 1299 Pennsylvania Avenue, NW, Suite 700, Washington, District of Columbia 20004 (US). (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). Declarations under Rule 4.17: - as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(ii)) 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)) (54) Title: PD-1 HOMING ENDONUCLEASE VARIANTS, COMPOSITIONS, AND METHODS OF USE (57) : The present disclosure provides improved genome editing compositions and methods for editing a PD-1 gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of, a cancer, an infectious disease, an autoimmune disease, an inflammatory disease, or an immunodeficiency.