公开(公告)号：EP4053277A1

公开(公告)日：2022-09-07

申请号：EP21213766.5

申请日：2016-12-26

申请人： Novartis AG ,  Intellia Therapeutics, Inc.

发明人： BOITANO, Anthony Edward ,  COOKE, Michael ,  KLICKSTEIN, Lloyd B. ,  LESCARBEAU, Reynald ,  MICKANIN, Craig Stephen ,  MULUMBA, Kabungo ,  POLICE, Seshidhar Reddy ,  SNEAD, Jennifer ,  STEVENSON, Susan C. ,  STEWART, Morag ,  YANG, Yi

IPC分类号： C12N15/113

摘要： The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.

公开(公告)号：EP2844302A2

公开(公告)日：2015-03-11

申请号：EP13730648.6

申请日：2013-05-02

申请人： Novartis AG

发明人： BIGELOW, Chad Eric ,  CHOI, Vivian ,  DRYJA, Thaddeus Peter ,  POLICE, Seshidhar Reddy

IPC分类号： A61K48/00 ,  C12N15/864

CPC分类号： C12N15/86 ,  A61K38/1709 ,  A61K48/005 ,  A61K48/0058 ,  C07H21/04 ,  C07K14/47 ,  C12N7/00 ,  C12N15/79 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2750/14343 ,  C12N2750/14345 ,  C12N2750/14371 ,  C12N2800/22 ,  C12N2810/6027 ,  C12N2810/85

摘要： The present invention relates to viral vectors that are capable of delivering a heterologous gene to the retina and in particular delivering RLBP1 to RPE and Muller cells of the retina. The invention also relates nucleic acids useful for producing viral vectors, compositions comprising the viral vectors and uses of the compositions and viral vectors. The invention also relates to methods of delivering and/or expressing a heterologous gene to the retina, improving the rate of dark adaption in a subject and treating RLBP1-associated retinal dystrophy.

摘要翻译： 本发明涉及能够将异源基因递送至视网膜，特别是将RLBP1递送至视网膜的RPE和Müller细胞的病毒载体。 本发明还涉及可用于产生病毒载体的核酸，包含病毒载体的组合物和组合物和病毒载体的用途。 本发明还涉及向视网膜递送和/或表达异源基因的方法，改善受试者的暗适应速率并治疗RLBP1相关的视网膜营养不良症。

公开(公告)号：EP3326655A1

公开(公告)日：2018-05-30

申请号：EP17204499.2

申请日：2013-05-02

申请人： Novartis AG

发明人： BIGELOW, Chad Eric ,  CHOI, Vivian ,  DRYJA, Thaddeus Peter ,  POLICE, Seshidhar Reddy

IPC分类号： A61K48/00 ,  C12N15/864 ,  C07K14/47 ,  A61K38/17 ,  C07H21/04

CPC分类号： C12N15/86 ,  A61K38/1709 ,  A61K48/005 ,  A61K48/0058 ,  C07H21/04 ,  C07K14/47 ,  C12N7/00 ,  C12N15/79 ,  C12N2750/14143 ,  C12N2750/14171 ,  C12N2750/14343 ,  C12N2750/14345 ,  C12N2750/14371 ,  C12N2800/22 ,  C12N2810/6027 ,  C12N2810/85

摘要： The present invention relates to viral vectors that are capable of delivering a heterologous gene to the retina and in particular delivering RLBP1 to RPE and Muller cells of the retina. The invention also relates nucleic acids useful for producing viral vectors, compositions comprising the viral vectors and uses of the compositions and viral vectors. The invention also relates to methods of delivering and/or expressing a heterologous gene to the retina, improving the rate of dark adaption in a subject and treating RLBP1-associated retinal dystrophy.

公开(公告)号：EP1377671A2

公开(公告)日：2004-01-07

申请号：EP02714960.8

申请日：2002-02-22

申请人： Novartis AG

发明人： ENNIST, David, Leonard ,  FORRY-SCHAUDIES, Suzanne ,  GORZIGLIA, Mario ,  HALLEN-BECK, Paul, L. ,  HAY, Carl, M. ,  JAKUBCZAK, John, Leonard ,  KALEKO, Michael ,  RYAN, Patricia, Clara ,  STEWART, David, A. ,  XIE, Yuefeng ,  CONNELLY, Sheila ,  POLICE, Seshidhar Reddy ,  CLARKE, Lori ,  PHIPPS, Sandrina ,  CHENG, Cheng

IPC分类号： C12N15/861

CPC分类号： C12N15/86 ,  C12N2710/10343 ,  C12N2800/30 ,  C12N2830/007 ,  C12N2830/008 ,  C12N2830/85 ,  C12N2840/203

摘要： The present invention relates to oncolytic adenoviral vectors and their use in methods of gene therapy. Provided is a recombinant viral vector comprising an adenoviral nucleic acid backbone, wherein said nucleic acid backbone comprises in sequential order : A left ITR, a termination signal sequence, an E2F responsive promoter which is operably linked to a gene essential for replication of the recombinant viral vector, an adenoviral packaging signal, and a right ITR.