公开(公告)号：EP4230196A1

公开(公告)日：2023-08-23

申请号：EP22382147.1

申请日：2022-02-21

申请人： Som Innovation Biotech, S.A. ,  Regents of the University of Minnesota

发明人： PERICOT MOHR, Gal.la ,  INSA BORONAT, Raúl ,  HUERTAS GAMBÍN, Óscar ,  REIG BOLAÑO, Núria ,  ASAKURA, Atsushi ,  ASAKURA, Yoko ,  MONTOLIO DEL OLMO, Marisol.

IPC分类号： A61K31/18 ,  A61K31/35 ,  A61K31/4184 ,  A61K31/4402 ,  A61K31/4439 ,  A61K31/4704 ,  A61K31/4725 ,  A61K31/54 ,  A61K31/621 ,  A61P21/00 ,  A61K31/37 ,  A61K31/47 ,  A61K31/4709 ,  A61K31/5377 ,  A61K31/549

摘要： The present invention relates to a compound selected from the group consisting of Veliflapon, Difenpiramide, Amopyroquine, Cloricromen, Alpelisib, Benorilate, Xipamide, Fabomotizole and Altizide, or a pharmaceutically acceptable salt thereof, and to combinations thereof, for use in the treatment or prevention of a dystrophinopathy.

公开(公告)号：EP3367785A2

公开(公告)日：2018-09-05

申请号：EP16860830.5

申请日：2016-10-27

申请人： Regents of the University of Minnesota ,  Recombinetics, Inc.

发明人： ASAKURA, Atsushi ,  ARAVALLI, Raj ,  CHEERAN, Maxin ,  DUTTON, James ,  GARRY, Daniel, J. ,  GARRY, Mary, G. ,  GRAHAM, Melanie ,  LI, Ling ,  LOW, Walter ,  PANOSKALTSIS-MORTARI, Angela ,  O'BRIEN, Timothy ,  STEER, Clifford ,  TOLAR, Jakup ,  CARLSON, Daniel, F. ,  FAHRENKRUG, Scott, C. ,  KOYANO-NAKAGAWA, Naoko ,  PARR, Ann

IPC分类号： A01K67/00 ,  C12N5/07 ,  C12N15/85 ,  A01N1/02 ,  A61D19/04

CPC分类号： C12N15/907 ,  A01K67/0276 ,  A01K2217/075 ,  A01K2227/108 ,  A01K2267/025

摘要： Human or humanized tissues and organs suitable for transplant are disclosed herein. Gene editing of a host animal provides a niche for complementation of the missing genetic information by donor stem cells. Editing of a host genome to knock out or disrupt genes responsible for the growth and/or differentiation of a target organ and injecting that animal at an embryo stage with donor stem cells to complement the missing genetic information for the growth and development of the organ. The result is a chimeric animal in which the complemented tissue (human/humanized organ) matches the genotype and phenotype of the donor. Such organs may be made in a single generation and the stem cell may be taken or generated from the patient's own body. As disclosed herein, it is possible to do so by simultaneously editing multiple genes in a cell or embryo creating a "niche" for the complemented tissue. Multiple genes can be targeted for editing using targeted nucleases and homology directed repair (HDR) templates in vertebrate cells or embryos.

公开(公告)号：EP2948476B1

公开(公告)日：2018-06-06

申请号：EP14704040.6

申请日：2014-01-28

申请人： Shire Human Genetic Therapies, Inc. ,  Regents of the University of Minnesota

发明人： JOSIAH, Serene ,  LUBY, Thomas M. ,  ASAKURA, Atsushi ,  KEEFE, Dennis ,  CHARNAS, Lawrence ,  VERMA, Mayank

IPC分类号： C07K16/28 ,  A61K39/395

CPC分类号： C07K16/2863 ,  A61K2039/505 ,  C07K16/2866 ,  C07K2317/33 ,  C07K2317/54 ,  C07K2317/55 ,  C07K2317/76

摘要： The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to DMD an effective amount of an anti-Flt-1 antibody, or antigen binding fragment thereof, such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset.

公开(公告)号：EP2948476A1

公开(公告)日：2015-12-02

申请号：EP14704040.6

申请日：2014-01-28

申请人： Shire Human Genetic Therapies, Inc. ,  Regents of the University of Minnesota

发明人： JOSIAH, Serene ,  LUBY, Thomas M. ,  ASAKURA, Atsushi ,  KEEFE, Dennis ,  CHARNAS, Lawrence

IPC分类号： C07K16/28 ,  A61K39/395

CPC分类号： C07K16/2863 ,  A61K2039/505 ,  C07K16/2866 ,  C07K2317/33 ,  C07K2317/54 ,  C07K2317/55 ,  C07K2317/76

摘要： The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to DMD an effective amount of an anti-Flt-1 antibody, or antigen binding fragment thereof, such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset.

摘要翻译： 本发明尤其提供了用于治疗肌营养不良症的方法和组合物，特别是杜烯肌营养不良症（DMD）。 在一些实施方案中，根据本发明的方法包括对患有或易感DMD的个体施用有效量的抗Flt-1抗体或其抗原结合片段，使得至少一种症状或特征 的DMD强度，严重程度或频率降低，或延迟发作。