Title:
PHARMACEUTICAL COMPOSITION FOR TREATING RETINAL DISEASES COMPRISING AS ACTIVE INGREDIENT CRISPR/CAS COMPLEX THAT OPERATES SPECIFICALLY ON RETINAL PIGMENT EPITHELIUM
Document Type and Number:
WIPO Patent Application WO/2023/249475
Kind Code:
A1
Abstract:
The present invention relates to a pharmaceutical composition for preventing or treating retinal diseases, comprising a CRISPR/Cas complex as an active ingredient. The present invention is research for developing a technology for targeting target cells to be treated, to fundamentally treat retinal diseases, specifically age-related macular disease (AMD), and Cas9 expressed under the human VMD2 promoter was constructed to induce retinal pigment epithelium (RPE)-specific expression, and it was confirmed that pVMD2-Cas9 was specifically expressed and operated in RPE cells in both in-vivo mouse and in-vitro human retinal organism models. By using this, gene editing (correction) restricted to RPE cells is possible, which is expected to be greatly used in the fundamental treatment of various hereditary and non-hereditary eye diseases related to RPE cells.
Inventors:
LEE JUNWON (KR)
BYEON SUK HO (KR)
CUI GANG (KR)
PARK JIN KYU (KR)
BYEON SUK HO (KR)
CUI GANG (KR)
PARK JIN KYU (KR)
Application Number:
PCT/KR2023/095023
Publication Date:
December 28, 2023
Filing Date:
June 22, 2023
Export Citation:
Assignee:
UNIV YONSEI IACF (KR)
International Classes:
C12N15/63; A61K48/00; A61P27/02; C12N9/22; C12N15/113; C12N15/86
Domestic Patent References:
| WO2015195621A1 | 2015-12-23 |
Foreign References:
| KR20200036912A | 2020-04-07 | |||
| KR101961332B1 | 2019-03-22 | |||
| KR20220022107A | 2022-02-24 | |||
| KR102264829B1 | 2021-06-14 |
Attorney, Agent or Firm:
PADO IP LAW PLLC (KR)
Download PDF: