公开(公告)号：US11698377B2

公开(公告)日：2023-07-11

申请号：US16325653

申请日：2017-08-14

Applicant: Genzyme Corporation

Inventor： Xiaoying Jin ,  Catherine O'Riordan ,  Lin Liu ,  Kate Zhang

IPC: G01N33/68 ,  C12N15/86 ,  G01N30/72 ,  G01N30/02

CPC classification number: G01N33/6848 ,  C12N15/86 ,  G01N30/72 ,  C12N2750/14123 ,  C12N2750/14143 ,  G01N2030/027

Abstract: Provided herein are methods for determining the serotype of a virus particle and/or or determining the heterogeneity of a virus particle (e.g., an AAV particle). In other embodiments, the invention provides methods to determine the heterogeneity of AAV particles. In some aspects, the invention provides viral particles (e.g., rAAV particles) with improved stability and/or improved transduction efficiency by increasing the acetylation and/or deamidation of capsid proteins.

公开(公告)号：US11554161B2

公开(公告)日：2023-01-17

申请号：US16546070

申请日：2019-08-20

Applicant: Genzyme Corporation

Inventor： James Dodge ,  Lamya Shihabuddin ,  Catherine O'Riordan

IPC: A61K48/00 ,  C12N15/79 ,  C07H21/04 ,  A61K38/30 ,  C12N15/864

Abstract: This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject's spinal cord by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain.

公开(公告)号：US11103598B2

公开(公告)日：2021-08-31

申请号：US16503091

申请日：2019-07-03

Applicant: Genzyme Corporation

Inventor： Catherine O'Riordan ,  Matthew Adamowicz

IPC: A61K48/00 ,  A61K31/7105 ,  C12N15/86 ,  A61K9/00 ,  C12N15/113 ,  A61K38/17

Abstract: Provided herein are methods for treating retinitis pigmentosa using an AAV particles encoding miR-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. Viral particles comprising AAV5 capsids or mutants thereof are contemplated.

公开(公告)号：US10920245B2

公开(公告)日：2021-02-16

申请号：US15858303

申请日：2017-12-29

Applicant: Genzyme Corporation

Inventor： Catherine O'Riordan ,  Samuel Wadsworth

IPC: C12N15/86 ,  C07H21/04 ,  A61K39/00 ,  A61K38/17 ,  A61K48/00

Abstract: This disclosure provides methods and compositions for treating disorders or injuries that affect motor function and control in a subject. In one aspect, the invention a transgene product is delivered to a subject's spinal cord by administering a recombinant viral vector containing the transgene to the spinal cord. The viral vector delivers the transgene which expresses the encoded recombinant viral gene product. The viral gene product comprises HIF1-alpha. Also provided are compositions for delivery of a transgene product to a subject's spinal cord.