Title:
RAAV-CONE OPSIN COMPOSITIONS AND METHODS FOR TREATING BLUE CONE MONOCHROMACY AND COLOR BLINDNESS
Document Type and Number:
WIPO Patent Application WO/2023/205758
Kind Code:
A3
Abstract:
Viral vector compositions are provided comprising polynucleotide sequences that express one or more biologically-active mammalian cone opsin proteins, under the control of a cone photoreceptor cell specific promoter, and a pharmaceutically acceptable carrier. Methods for use of these viral vector compositions are disclosed for in preventing, treating, and/or ameliorating a mammalian subject having a disease associated with cone monochromacies, such as blue cone monochromacy (BCM). Specifically, these compositions are disclosed which are useful in methods for treating or ameliorating symptoms of blue cone monochromacy caused by mis-sense point mutations, such as C203R, in the red and/or green cone opsin genes
Inventors:
DENG WEN-TAO (US)
Application Number:
PCT/US2023/066041
Publication Date:
November 30, 2023
Filing Date:
April 21, 2023
Export Citation:
Assignee:
WEST VIRGINIA UNIV BOARD OF GOVERNORS ON BEHALF OF WEST VIRGINIA UNIV (US)
International Classes:
C12N15/864; A61K38/17; A61P27/02; C07K14/705; C12N7/00
Foreign References:
| US20180320145A1 | 2018-11-08 | |||
| US20200399656A1 | 2020-12-24 |
Attorney, Agent or Firm:
COCHENOUR, Craig G. (US)
Download PDF: