公开(公告)号：US20210069349A1

公开(公告)日：2021-03-11

申请号：US16952002

申请日：2020-11-18

Applicant: 4D MOLECULAR THERAPEUTICS INC.

Inventor： David H. KIRN ,  Melissa KOTTERMAN ,  David SCHAFFER

IPC: A61K48/00 ,  A61P27/02 ,  A61K9/00 ,  C07K14/005 ,  C12N15/86

Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.

公开(公告)号：US20240335560A1

公开(公告)日：2024-10-10

申请号：US18466517

申请日：2023-09-13

Applicant: 4D MOLECULAR THERAPEUTICS INC.

Inventor： David H. Kirn ,  Melissa KOTTERMAN ,  David SCHAFFER ,  Paul SZYMANSKI ,  Peter FRANCIS

IPC: A61K48/00 ,  A61K9/00 ,  C07K14/005 ,  C07K16/22 ,  C12N7/00

CPC classification number: A61K48/005 ,  A61K9/0048 ,  A61K48/0008 ,  A61K48/0075 ,  C07K14/005 ,  C07K16/22 ,  C12N7/00 ,  C12N2750/14122 ,  C12N2750/14143

Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina, for the treatment of retinal disorders and diseases.

公开(公告)号：US20210332341A1

公开(公告)日：2021-10-28

申请号：US17240948

申请日：2021-04-26

Applicant: 4D MOLECULAR THERAPEUTICS INC.

Inventor： David H. KIRN ,  Melissa KOTTERMAN ,  Peter FRANCIS ,  David SCHAFFER ,  Paul SZYMANSKI ,  Kevin WHITTLESEY

IPC: C12N9/40 ,  A61K9/00 ,  C12N15/86 ,  A61P3/00

Abstract: The present disclosure provides codon optimized nucleotide sequences encoding human alpha-galactosidase A, vectors, and host cells comprising codon optimized alpha-galactosidase A sequences, and methods of treating disorders such as Fabry disease comprising administering to the subject a codon optimized sequence encoding human alpha-galactosidase A.

公开(公告)号：US20210177990A1

公开(公告)日：2021-06-17

申请号：US17163038

申请日：2021-01-29

Applicant: 4D MOLECULAR THERAPEUTICS INC.

Inventor： David H. KIRN ,  Melissa KOTTERMAN ,  David SCHAFFER

IPC: A61K48/00 ,  A61P27/02 ,  A61K9/00 ,  C07K14/005 ,  C12N15/86

Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of retinal cells as compared to the infectivity of the retinal cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more cells of the retina for the treatment of retinal disorders and diseases.

公开(公告)号：US20200248205A1

公开(公告)日：2020-08-06

申请号：US16648195

申请日：2018-09-19

Applicant: 4D MOLECULAR THERAPEUTICS INC.

Inventor： David H. KIRN ,  Melissa KOTTERMAN ,  David SCHAFFER

IPC: C12N15/86 ,  C07K14/005

Abstract: Provided herein are variant adeno-associated virus (AAV) capsid proteins having one or more modifications in amino acid sequence relative to a parental AAV capsid protein, which, when present in an AAV virion, confer increased infectivity of one or more types of muscle cells as compared to the infectivity of the muscle cells by an AAV virion comprising the unmodified parental AAV capsid protein. Also provided are recombinant AAV virions and pharmaceutical compositions thereof comprising a variant AAV capsid protein as described herein, methods of making these rAAV capsid proteins and virions, and methods for using these rAAV capsid proteins and virions in research and in clinical practice, for example in, e.g., the delivery of nucleic acid sequences to one or more muscle cells for the treatment of muscle disorders and diseases.