公开(公告)号：US20240197919A1

公开(公告)日：2024-06-20

申请号：US18288910

申请日：2022-05-03

Applicant: California Institute of Technology

Inventor： Viviana Gradinaru ,  Xinhong Chen

IPC: A61K48/00 ,  C07K14/005 ,  C07K14/47 ,  C12N15/86

CPC classification number: A61K48/0058 ,  C07K14/005 ,  C07K14/47 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145

Abstract: Disclosed herein include compositions and kits comprising recombinant adeno-associated viruses (rAAVs) with tropisms to the central nervous system with increased specificity and transduction efficiency, including endothelial cells of the neurovascular unit. Also described include methods of treating various diseases and conditions using the rAAVs.

公开(公告)号：US20250001007A1

公开(公告)日：2025-01-02

申请号：US18709720

申请日：2022-11-16

Applicant: California Institute of Technology

Inventor： Viviana Gradinaru ,  Xinhong Chen

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

Abstract: Described herein are compositions and kits comprising recombinant adeno-associated viruses (rAAVs) with increased transduction enrichment in the heart and/or skeletal muscle and, in some cases, reduced transduction in the liver. The rAAV compositions described herein encapsidate a transgene, such as a therapeutic nucleic acid. Gene therapy using the rAAVs is described. Also described are methods of treating diseases and conditions of the heart and/or skeletal muscle.

公开(公告)号：US20240139329A1

公开(公告)日：2024-05-02

申请号：US18495508

申请日：2023-10-26

Applicant: California Institute of Technology

Inventor： Viviana Gradinaru ,  Timothy F. Shay ,  Xinhong Chen ,  Seongmin Jang

IPC: A61K47/64 ,  A61K48/00 ,  C07K14/00

CPC classification number: A61K47/64 ,  A61K48/0041 ,  C07K14/001

Abstract: Disclosed herein include novel blood-brain barrier (BBB)-crossing receptors on the BBB interface, targeting peptides and derivatives thereof capable of binding to the novel receptors, and related methods of using the receptors to increase the permeability of the BBB and to deliver an agent to a nervous system (e.g., CNS). In some embodiments, the BBB-crossing receptor is LRP6. Disclosed herein also include recombinant adeno-associated viruses (rAAVs) with increased specificity and transduction efficiency across the BBB and related compositions and methods of treating various diseases and conditions.