Patent SG-11201901718V-A (SG)
Title: Gene therapy for patients with fanconi anemia
Inventor: BUEREN JUAN A
IPC: C12Q1/68
Filed: 2017-09-08
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1111111011110 DOI HIM 0111 II 0 0111111111111111111111111111111111111110111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2018/049273 Al 15 March 2018 (15.03.2018) W I P0 I P C T (51) International Patent Classification: TIL UNIVERSITARIO NINO JESUS [ES/ES]; Avda Cl 2N 15/85 (2006.01) C12Q 1/68 (2006.01) Menendez Pelayo 65, 28009 Madrid (ES). Cl 2N 15/63 (2006.01) (71) Applicant (for OM only): SHERIN, Mridula [US/US]; (21) International Application Number: 1700 Seventh Avenue, Suite 1900, Seattle, Washington PCT/U52017/050837 98101 (US). (22) International Filing Date: (72) Inventors; and 08 September 2017 (08.09.2017) (71) Applicants: BUEREN, Juan A. [ES/US]; c/o RTW Invest- (25) Filing Language: English ments, LLC, 250 W 55th St., Suite A, New York, New York 10019 (US). RIO, Paula [ES/US]; c/o RTW Invest- (26) Publication Language: English ments, LLC, 250 W 55th St., Suite A, New York, New (30) Priority Data: York 10019 (US). NAVARRO, Susana [ES/US]; c/o RTW 62/385,185 08 September 2016 (08.09.2016) US Investments, LLC, 250 W 55th St., Suite A, New York, 62/412,028 24 October 2016 (24.10.2016) US New York 10019 (US). SEVILLA, Julian [ES/US]; c/o RTW Investments, LLC, 250 W 55th St., Suite A, New (71) Applicants: CENTRO DE INVESTIGACIONES York, New York 10019 (US). SEGOVIA, Jose Carlos ENERGETICAS MEDIOAMBIENTALES Y TEC- [ES/US]; c/o RTW Investments, LLC, 250 W 55th St., NOLOGICAS [ES/ES]; Avda. de la Complutense, Suite A, New York, New York 10019 (US). GONZALEZ, 40, 28040 Madrid (ES). FUNDACION INSTITUTO Africa [ES/US]; c/o RTW Investments, LLC, 250 W 55th DE INVESTIGACION SANITARIA FUNDACION St., Suite A, New York, New York 10019 (US). CASA- _ JIMENEZ DIAZ [ES/ES]; Avda. de los Reyes Catoli- DO, Jose Antonio [ES/US]; c/o RTW Investments, LLC, cos, 2, 28040 Madrid (ES). CENTRO DE INVESTI- 250 W 55th St., Suite A, New York, New York 10019 GACION BIOMEDICA EN RED [ES/ES]; Monforte de (US). GUENECHEA, Guillermo [ES/US]; c/o RTW In- — = Lemos, 5, 28029 Madrid (ES). FUNDACION PARA LA vestments, LLC, 250 W 55th St., Suite A, New York, New = INVESTIGACION BIOMEDICA HOSPITAL INFAN- York 10019 (US). = (54) Title: GENE THERAPY FOR PATIENTS WITH FANCONI ANEMIA Figure 1. = = = = = = Lentiviral backbone = = A113 = 43 1-1 ,:•ouorio,-„,,„ i i ppt / w9 r .4 S D SA LTR e 4 \") ei CI' iv Therapeutic cassette 0 -.... 00 1-1 (57) : The present invention provides compositions and methods for rescuing FANCA expression in cells with diminished or C:::: ) no FANCA gene product. In particular, methods and compositions for gene therapy of Fanconi anemia are disclosed. ei O [Continued on next page] WO 2018/049273 Al MIDEDIMOMOIDEIREEMOOVIEEIMEIMOVOIS (74) Agent: SHERIN, Mridula et al.; Cooley LLP, 1299 Penn- sylvania 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). Published: — with international search report (Art. 21(3)) — with sequence listing part of description (Rule 5.2(a))