公开(公告)号：US12138318B2

公开(公告)日：2024-11-12

申请号：US16541516

申请日：2019-08-15

Applicant: UCL Business LTD

Inventor： Amit Nathwani ,  Allison Dane

IPC: A61K48/00 ,  C07K14/005 ,  C12N15/86

Abstract: There is described an AAV capsid protein having an amino acid sequence which has at least 98% identity to the sequence of SEQ ID NO: 3 or at least 94% identity to the sequence of SEQ ID NO: 4. Also described is a pharmaceutical composition, an AAV capsid and a viral particle comprising the capsid protein, a recombinant AAV vector comprising a nucleotide sequence which encodes for the capsid protein, and a host cell and a transgenic animal comprising the capsid protein or the vector. In addition, there is described a method of transferring a nucleic acid of interest into a mammal comprising introducing a recombinant AAV vector into the mammal, wherein the recombinant AAV vector comprises a gene of interest which is encapsidated into a capsid comprising the capsid protein.

公开(公告)号：US20200079821A1

公开(公告)日：2020-03-12

申请号：US16541516

申请日：2019-08-15

Applicant: UCL Business LTD

Inventor： Amit Nathwani ,  Allison Dane

IPC: C07K14/005 ,  A61K48/00 ,  C12N15/86

Abstract: There is described an AAV capsid protein having an amino acid sequence which has at least 98% identity to the sequence of SEQ ID NO: 3 or at least 94% identity to the sequence of SEQ ID NO: 4. Also described is a pharmaceutical composition, an AAV capsid and a viral particle comprising the capsid protein, a recombinant AAV vector comprising a nucleotide sequence which encodes for the capsid protein, and a host cell and a transgenic animal comprising the capsid protein or the vector. In addition, there is described a method of transferring a nucleic acid of interest into a mammal comprising introducing a recombinant AAV vector into the mammal, wherein the recombinant AAV vector comprises a gene of interest which is encapsidated into a capsid comprising the capsid protein.