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1. (WO2019067766) METHODS, COMPOSITIONS, AND IMPLANTABLE ELEMENTS COMPRISING ACTIVE CELLS
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Pub. No.: WO/2019/067766 International Application No.: PCT/US2018/053191
Publication Date: 04.04.2019 International Filing Date: 27.09.2018
IPC:
A61K 48/00 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
Applicants:
SIGILON THERAPEUTICS, INC. [US/US]; 100 Binney St. Ste 600 Cambridge, MA 02142, US
Inventors:
CARMONA, Guillaume; US
GONZALEZ, Francisco, Caballerro; US
HEIDEBRECHT, Richard; US
MILLER, Robert, James; US
OBERLI, Matthias, Alexander; US
PERITT, David; US
SMITH, Devyn, McKinley; US
VEISEH, Omid; US
WOTTON, Paul, Kevin; US
SEWELL, Jered, A.; US
Agent:
COLLAZO, Diana, M.; US
LARKIN, Angelyn; US
Priority Data:
62/563,87727.09.2017US
62/652,88104.04.2018US
62/652,88204.04.2018US
Title (EN) METHODS, COMPOSITIONS, AND IMPLANTABLE ELEMENTS COMPRISING ACTIVE CELLS
(FR) PROCÉDÉS, COMPOSITIONS ET ÉLÉMENTS IMPLANTABLES COMPRENANT DES CELLULES ACTIVES
Abstract:
(EN) Described herein are cell compositions comprising an active cell (e.g., an engineered active cell, e.g., an engineered RPE cell) or derivatives thereof, as well as compositions, pharmaceutical products, and implantable elements comprising an active cell, and methods of making and using the same. The cells and compositions may express a therapeutic agent useful for the treatment of a disease, disorder, or condition described herein.
(FR) L'invention concerne des compositions cellulaires comprenant une cellule active (p. ex., une cellule active génétiquement modifiée, p. ex., une cellule RPE génétiquement modifiée) ou des dérivés de celle-ci, ainsi que des compositions, des produits pharmaceutiques et des éléments implantables comprenant une cellule active, et leurs procédés de préparation et d'utilisation. Les cellules et les compositions peuvent exprimer un agent thérapeutique utile pour le traitement d'une maladie, d'un trouble ou d'une affection tel que décrit dans la présente.
Designated States: 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
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (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)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)