公开(公告)号：US11104917B2

公开(公告)日：2021-08-31

申请号：US15572101

申请日：2016-05-04

申请人： Children's Medical Research Institute ,  The Sydney Children's Hospital Network (Randwich and Westmead) (incorporating The Royal Alexandra Hospital For Children

发明人： Grant Logan ,  Ian Alexander ,  Allison Dane

IPC分类号： C12N15/864 ,  C12N15/85 ,  C12N15/11 ,  C12N15/63 ,  A61K48/00 ,  C12N15/113 ,  C12N15/86 ,  C12N15/867 ,  C12N15/869

摘要： The present disclosure relates generally to promoters derived from the AAV anti-sense strand, their use in the expression of one or more heterologous coding sequences, and isolated polynucleotides, vectors and recombinant viruses comprising the promoters. The present disclosure also relates to enhancers derived from the AAV anti-sense strand, their use in increasing the expression of one or more heterologous coding sequences, and isolated polynucleotides, vectors and recombinant viruses comprising the enhancers.

公开(公告)号：US20150246085A1

公开(公告)日：2015-09-03

申请号：US14440135

申请日：2013-11-01

申请人： THE SYDNEY CHILDREN'S HOSPITAL NETWORK (RANDWICK & WESTMEAD)

发明人： Naz Al-Hafid ,  John Christodoulou ,  Xing Zhang Tong

IPC分类号： A61K35/747 ,  C12N9/88 ,  A61K38/51 ,  A61K35/744

CPC分类号： A61K35/747 ,  A61K35/741 ,  A61K35/744 ,  A61K38/51 ,  A61K2035/115 ,  C12N9/88 ,  C12N15/625 ,  C12Y403/01024

摘要： The present disclosure provides a genetically-modified probiotic expressing recombinent phenylalanine ammonia Lyase (PAL) useful for treating phenylketonuria.

摘要翻译： 本公开提供了用于治疗苯丙酮尿症的基因修饰益生菌表达重组苯丙氨酸氨裂解酶（PAL）。

公开(公告)号：US20230093183A1

公开(公告)日：2023-03-23

申请号：US17801510

申请日：2021-02-25

申请人： Children's Medical Research Institute ,  The Sydney Children's Hospital Network (Randwick and Westmead) (Incorporating the Royal Alexandra Ho ,  LogicBio Therapeutics, Inc.

发明人： Leszek Lisowski ,  Marti Cabanes Creus ,  Ian Alexander ,  Matthias Charles Jerome Hebben

IPC分类号： C07K14/005 ,  C12N15/86

摘要： The present disclosure relates generally to adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

公开(公告)号：US11597765B2

公开(公告)日：2023-03-07

申请号：US17352574

申请日：2021-06-21

申请人： VACCINEX, INC. ,  The Sydney Children's Hospital Network

发明人： Yilin Mao ,  Wendy Gold ,  Elizabeth E. Evans ,  Maurice Zauderer

IPC分类号： A61K39/00 ,  A61K39/395 ,  C07K16/28 ,  A61P25/00

摘要： Provided herein are methods for alleviating symptoms in a subject having Rett syndrome, comprising administering to the subject an effective amount of an isolated binding molecule which specifically binds to semaphorin-4D (SEMA4D).

公开(公告)号：US20210403559A1

公开(公告)日：2021-12-30

申请号：US17352574

申请日：2021-06-21

申请人： VACCINEX, INC. ,  The Sydney Children's Hospital Network

发明人： Yilin Mao ,  Wendy Gold ,  Elizabeth E. Evans ,  Maurice Zauderer

IPC分类号： C07K16/28 ,  A61P25/00

摘要： Provided herein are methods for alleviating symptoms in a subject having Rett syndrome, comprising administering to the subject an effective amount of an isolated binding molecule which specifically binds to semaphorin-4D (SEMA4D).