公开(公告)号：US20230364206A1

公开(公告)日：2023-11-16

申请号：US18030613

申请日：2021-10-06

申请人： REGENXBIO Inc.

发明人： Christina Maria Ohnsman ,  Stephen Joseph Pakola ,  Sherri Van Everen ,  Paulo Falabella ,  Alexander Marston Bailey ,  Nicholas Alexander Piers Sascha Buss ,  Kwi Hye Kim

IPC分类号： A61K38/48 ,  C12N9/48 ,  A61K48/00 ,  C12N15/86 ,  A61P27/02

CPC分类号： A61K38/4813 ,  C12N9/485 ,  A61K48/0066 ,  C12N15/86 ,  C12Y304/14009 ,  A61P27/02 ,  A61K48/0075 ,  C12N2750/14143

摘要： Compositions and methods are described for the delivery of therapeutic products (such as therapeutic proteins (for example, antibodies), therapeutic RNAs (for example, shRNAs, siRNAs, and miRNAs), and therapeutic aptamers) to the retina/vitreal humour in the eyes of human subjects to treat pathologies of the eye, involving, for example, recombinant viral vectors such as recombinant adeno-associated virus (rAAV) vectors.

公开(公告)号：US20240091380A1

公开(公告)日：2024-03-21

申请号：US18274499

申请日：2022-01-31

申请人： REGENXBIO Inc.

发明人： Marie-Laure Nevoret ,  Nicholas Alexander Piers Sascha Buss ,  Paulo Falabella ,  Stephen Joseph Pakola

IPC分类号： A61K48/00 ,  A61K9/00 ,  A61K45/06 ,  A61P25/28 ,  C12N9/48 ,  C12N15/86

CPC分类号： A61K48/005 ,  A61K9/0085 ,  A61K45/06 ,  A61K48/0075 ,  A61P25/28 ,  C12N9/485 ,  C12N15/86 ,  C12N2750/14143 ,  C12Y304/14009

摘要： Provided herein are methods and compositions for treatment of CLN2 Disease. Such compositions include a recombinant adeno-associated virus (rAAV), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising (a) an AAV 5′ inverted terminal repeat (ITR) sequence; (b) a promoter; (c) a CLN2 coding sequence encoding a human TPP1; (d) an AAV 3′ ITR. Also provided herein are methods of treating CLN2 Disease comprising administering to a subject in need thereof the rAAV described herein via more than one route. Also provide herein are pharmaceutical compositions comprising the rAAV described herein and related methods of treating CLN2 Disease.

公开(公告)号：US11986515B2

公开(公告)日：2024-05-21

申请号：US18356961

申请日：2023-07-21

申请人： REGENXBIO Inc.

发明人： Stephen Joseph Pakola ,  Paulo Falabella ,  Marie-Laure Nevoret

IPC分类号： A61P25/28 ,  A61K38/46 ,  C12N15/86 ,  G01N33/53

CPC分类号： A61K38/465 ,  A61P25/28 ,  C12N15/86 ,  C12Y301/06013 ,  G01N33/5308 ,  C12N2750/14143 ,  C12N2750/14171 ,  G01N2400/00 ,  G01N2800/04

摘要： Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).

公开(公告)号：US20240117328A1

公开(公告)日：2024-04-11

申请号：US18276356

申请日：2022-01-31

申请人： REGENXBIO Inc.

发明人： Stephen Joseph Pakola ,  Paulo Falabella ,  Marie-Laure Nevoret

IPC分类号： C12N9/16 ,  A61K38/46 ,  A61K45/06 ,  A61K48/00 ,  A61P3/00 ,  C12N15/86 ,  G01N33/50

CPC分类号： C12N9/16 ,  A61K38/465 ,  A61K45/06 ,  A61K48/005 ,  A61K48/0075 ,  A61P3/00 ,  C12N15/86 ,  G01N33/5008 ,  C12N2750/14143 ,  C12Y301/06013

摘要： Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).

公开(公告)号：US20240058426A1

公开(公告)日：2024-02-22

申请号：US18356961

申请日：2023-07-21

申请人： REGENXBIO Inc.

发明人： Stephen Joseph Pakola ,  Paulo Falabella ,  Marie-Laure Nevoret

IPC分类号： A61K38/46 ,  A61P25/28 ,  C12N15/86 ,  G01N33/53

CPC分类号： A61K38/465 ,  A61P25/28 ,  C12N15/86 ,  C12Y301/06013 ,  G01N33/5308 ,  C12N2750/14143 ,  C12N2750/14171 ,  G01N2400/00 ,  G01N2800/04

摘要： Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).