公开(公告)号：WO2023034942A1

公开(公告)日：2023-03-09

申请号：PCT/US2022/075871

申请日：2022-09-01

申请人： JAGUAR GENE THERAPY, LLC

发明人： HUGHES, Michael

IPC分类号： A61K38/45 ,  C07K14/075 ,  C12N15/52 ,  C12N15/86 ,  A61K38/16 ,  A61K38/17 ,  A61K48/00 ,  A61P3/00 ,  C12P21/02

摘要： Provided are recombinant adeno-associated virus (rAAV) vectors comprising a transgene to express galactose- 1 -phosphate uridylyl transferase (GALT); virions comprising said vectors (rAAV virions); methods of their production; methods of their use, including methods for treating galactosemia, GALT-deficiency, symptoms therefrom; and kits.

公开(公告)号：WO2023023781A1

公开(公告)日：2023-03-02

申请号：PCT/AU2022/051008

申请日：2022-08-25

申请人： CHILDREN'S MEDICAL RESEARCH INSTITUTE ,  THE SYDNEY CHILDREN'S HOSPITALS NETWORK (RANDWICK AND WESTMEAD) (INCORPORATING THE ROYAL ALEXANDRA HOSPITAL FOR CHILDREN) ,  LOGICBIO THERAPEUTICS, INC.

发明人： CABANES CREUS, Marti ,  LISOWSKI, Leszek ,  ALEXANDER, Ian ,  HEBBEN, Matthias Charles Jerome

IPC分类号： C07K14/005 ,  C07K14/075 ,  C12N7/00 ,  C12N15/86

摘要： The present disclosure relates generally to modified 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：WO2022003083A1

公开(公告)日：2022-01-06

申请号：PCT/EP2021/068124

申请日：2021-07-01

申请人： REITHERA SRL

发明人： COLLOCA, Stefano ,  LAHM, Armin ,  RAGGIOLI, Angelo ,  VITELLI, Alessandra

IPC分类号： C07K14/075 ,  C12N15/861 ,  C07K14/005 ,  C12N15/86 ,  C12N2710/10321 ,  C12N2710/10323 ,  C12N2710/10334 ,  C12N2710/10343 ,  C12N2770/20034 ,  C12N7/00

摘要： The present invention relates to novel adenovirus strains with a high immunogenicity and very low pre-existing immunity in the general human population. The absence of detectable neutralizing antibodies is due to novel hypervariable regions in the adenoviral capsid protein hexon. The present invention provides nucleotide and amino acid sequences of these novel adenovirus strains, as well as recombinant viruses, virus-like particles and vectors based on these strains. Further provided are pharmaceutical compositions and medical uses in the therapy or prophylaxis of a disease, and methods for producing an adenovirus or virus-like particles utilizing the novel sequences, recombinant viruses, virus-like particles and vectors.

公开(公告)号：WO2021224397A1

公开(公告)日：2021-11-11

申请号：PCT/EP2021/062002

申请日：2021-05-06

申请人： IMCYSE SA

发明人： ERAK, Milos

IPC分类号： A61K39/00 ,  C07K14/075 ,  C07K14/195 ,  C07K14/435 ,  C12N9/02

摘要： The invention relates to immunogenic peptides comprising MHC class II T-cell epitopes and oxidoreductase motifs with increased activity, and their use in regulating the immune response in subjects.

公开(公告)号：WO2021202817A1

公开(公告)日：2021-10-07

申请号：PCT/US2021/025281

申请日：2021-03-31

申请人： UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATED

发明人： BOYE, Shannon, E. ,  BOYE, Sanford, L. ,  SWEENEY, H., Lee ,  CALABRO, Kaitlyn

IPC分类号： A61K48/00 ,  C07K14/075 ,  C07K14/47 ,  C12N7/01 ,  C12N15/09 ,  C12N15/11

摘要： Disclosed are compositions and methods for treating diseases of the mammalian eye, and in particular, complications of the retina associated with Usher syndrome IB (USH1B). Further disclosed are compositions and methods for treating diseases of the mammalian inner ear, and in particular, complications of ear hair cells associated with Usher syndrome IB (USH1B). The disclosure provides improved AAV -based, dual vector systems that facilitate the expression of full-length proteins whose coding sequences exceed that of the polynucleotide packaging capacity of an individual AAV vector. Described herein are modified hybrid dual vector systems that shift the coding sequence for the MY07A tail domain from the front-half vector to the back-half vector by altering the split point (e.g., from between exons 23 and 24, to between exons 21 and 22), in order to eliminate the production of truncated MY07A protein. Further described herein are improved, codon-modified hybrid and overlap vector systems in which putative stop codons and residual sequences in non-coding sequences are removed.

公开(公告)号：WO2021011034A1

公开(公告)日：2021-01-21

申请号：PCT/US2020/022326

申请日：2020-03-12

申请人： VIGENE BIOSCIENCES INC.

发明人： WANG, Qizhao

IPC分类号： C12N15/861 ,  A61K35/761 ,  C07K14/075 ,  A61K48/00 ,  C12N15/69

摘要： The present invention is directed to recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency, pharmaceutical compositions comprising such rAAV, and methods for their production and use. The present invention is particularly directed to recombinantly-modified adeno-associated virus (rAAV) that have been further modified to comprise Cis-Elements, including replication origins, promoters and enhancers, that are capable of regulating the replication of an rAAV genome and that improve rAAV replication. Preferably, such Cis-Elements are provided within domains of the rAAV that precede and/or follow the 5' and/or 3' inverted terminal repeated sequences (ITR) of an rAAV. The invention particularly concerns the presence and the use of polynucleotide Cis-Elements that comprise actual or potential G-Quadruplex Sequences, polynucleotide Cis-Elements that comprise DNA sequences from wild-type AAV (wt AAV) and polynucleotide Cis- Elements that comprise DNA sequences from other viral genomes or from the human genome.

公开(公告)号：WO2020106924A1

公开(公告)日：2020-05-28

申请号：PCT/US2019/062547

申请日：2019-11-21

申请人： MAYO FOUNDATION FOR MEDICAL EDUCATION AND RESEARCH

发明人： BARRY, Michael A.

IPC分类号： A61K35/76 ,  A61K35/761 ,  A61K48/00 ,  C07K14/075 ,  C12N15/86 ,  C12N15/861

摘要： This invention relates to methods and materials for nucleic acid delivery, vaccination, and/or treating cancer. More specifically, methods and materials for nucleic acid delivery, vaccination, and/or treating cancer using one or more recombinant adenoviruses (Ads) as an oncolytic agent are provided.

公开(公告)号：WO2019126356A1

公开(公告)日：2019-06-27

申请号：PCT/US2018/066551

申请日：2018-12-19

申请人： THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL

发明人： LI, Chengwen ,  SAMULSKI, Richard, Jude

IPC分类号： C12N7/00 ,  C12N7/01 ,  C12N15/86 ,  C07K14/00 ,  C07K14/775 ,  C07K14/16 ,  C07K14/075 ,  B82Y5/00 ,  A61K39/00 ,  A61K39/12 ,  A61K35/761

摘要： Disclosed herein is an adeno-associated virus (AAV) particle comprising a surface-bound peptide that enhances transduction of cells across the blood-brain barrier (BBB). Also disclosed herein is a modified AAV capsid protein comprising an insertion of a polypeptide that enhances transduction of cells across the BBB, and an AAV particle comprising the modified AAV capsid protein. Specific peptides are provided. Pharmaceutical formulations and method of administering/delivering a nucleic acid to a cell of the brain and/or central nervous system are also disclosed.

公开(公告)号：WO2019010301A8

公开(公告)日：2019-01-10

申请号：PCT/US2018/040913

申请日：2018-07-05

申请人： KAPILOFF, Michael S.

发明人： KAPILOFF, Michael S.

IPC分类号： A61K31/713 ,  A61K38/45 ,  A61K48/00 ,  A61P9/00 ,  A61P9/10 ,  C07K14/075

摘要： The present invention provides a method of protecting the heart from damage, by administering to a patient at risk of such damage, a pharmaceutically effective amount of a composition which inhibits the interaction of RSK3 and mAKAPβ, or the expression or activity of one or both of those molecules. This composition is preferably in the form of an siRNA construct, more preferably an shRNA construct, which inhibits the expression of mAKAPβ.

公开(公告)号：WO2018209216A1

公开(公告)日：2018-11-15

申请号：PCT/US2018/032291

申请日：2018-05-11

申请人： UNIVERSITY OF MASSACHUSETTS

发明人： XIE, Jun ,  GAO, Guangping

IPC分类号： C07K14/075 ,  C12N15/09 ,  C12N15/63 ,  A61K35/76

摘要： In some aspects, the disclosure relates to methods for improving titer and yield of viral vector production. In some embodiments, the methods comprise transient silencing of transgene expression during packaging of a viral vector.