Patent SG-11201900543S-A (SG)
Title: Gene therapy for the treatment of aldehyde dehydrogenase deficiency
Inventor: GASMI MEHDI
IPC: C12Q1/68
Filed: 2017-07-26
Abstract: Timeun t il fa ll (sec ) 60 — 50 — 40 — 30 -- 20 10 — therapy Female,+ therapy Female, no therapy W O 20 18/0 227 83 Al (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 01 February 2018 (01.02.2018) WIP0 I PCT °Ens °nolo omolom olo mil °nom ow (10) International Publication Number WO 2018/022783 Al (51) International Patent Classification: A61K 48/00 (2006.01) Cl 2N 15/09 (2006.01) C12N 9/00 (2006.01) C12Q 1/32 (2006.01) C12N 9/02 (2006.01) C12Q 1/68 (2006.01) C12N 9/04 (2006.01) (21) International Application Number: PCT/US2017/043999 (22) International Filing Date: 26 July 2017 (26.07.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/367,012 26 July 2016 (26.07.2016) US (71) Applicants: CORNELL UNIVERSITY [US/US]; Cen- ter for Technology Licensing (CTL), 395 Pine Tree Road, Suite 310, Ithaca, New York 14850 (US). ADVERUM BIOTECHNOLOGIES, INC. [US/US]; 1035 O'Brien Drive, Menlo Park, California 94025 (US). (72) Inventors: GASMI, Mehdi; 1035 O'Brien Drive, Menlo Park, California 94025 (US). CRYSTAL, Ronald G.; 435 East 70th Street, Apt. 34-B, New York, New York 10021 (US). PAGOVICH, Odelya E.; 1330 First Avenue, New York, New York 10021 (US). (74) Agent: GASE, John L. et al.; Two Prudential Plaza, Suite 4900, 180 N. Stetson Ave., Chicago, Illinois 60601-6731 (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, (54) Title: GENE THERAPY FOR THE TREATMENT OF ALDEHYDE DEHYDROGENASE DEFICIENCY Et0E1 by gavage Male, no therapy 0 pre-Et0H 24 hr post-EtOH FIG. 3 (57) : A vector comprising a promoter operably linked to a nucleic acid sequence encoding human aldehyde dehydrogenase, as well as a composition comprising the vector and method of using the vector to treat aldehyde dehydrogenase deficiency, or to prevent or treat a disease characterized by aldehyde dehydrogenase deficiency. [Continued on next page] WO 2018/022783 Al IMEDIMOMMIDIRE10101011010HOMMOINVOIMIE 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))