| 1 |
Development of new compounds for effective gene editing therapy for an IMPDH1 mutation causing autosomal dominant retinitis pigmentosa |
| Gene Therapy |
Retinitis Pigmentosa |
IMPDH1 |
Hit |
Ulsan University |
RS-2023-00283544 |
|
| 2 |
Development of First.in.Class mutant transcript.selective ASO therapeutic candidates for the treatment of Avellino corneal dystrophy |
| Gene Therapy |
Avellino corneal dystrophy |
Human TGFBIR124H |
Candidate |
MediciBIO Co., LTD |
RS-2023-00282731 |
|
| 3 |
Development of AAV based ocular cell/tissue targeting technology and treatment candidates for the development of gene therapy for X.chromosome.linked retinitis pigmentosa |
| Gene Therapy |
Retinitis Pigmentosa |
RPGR |
Hit |
ENCell |
RS-2023-00258840 |
|
| 4 |
Discovery of Non-clinical Development Candidate Molecule for a Novel Universal Retinitis Pigmentosa Therapeutics |
| Gene Therapy |
Retinitis Pigmentosa |
RORβ |
Candidate |
OliX Pharmaceuticals |
RS-2023-00217014 |
|
| 5 |
Non-clinical development of gene therapy for Retinitis Pigmentosa with an RNA repair mechanism using AAV vector that encodes trans-splicing ribozyme targeting rhodopsin RNA |
| Gene Therapy |
Autosomal dominant Retinitis pigmentosa (adRP) |
P23H |
Preclinical |
Rznomics Inc. |
HN22C0300 |
|
| 6 |
Development of novel gene therapy for PDE6B related inherited retinal disease |
| Gene Therapy |
Genetic Retinal disease |
PDE6B |
Hit |
Asan Medical Center |
HN22C0245 |
|
| 7 |
CRG-02: An AAV-based anti-VEGF gene therapy for angiogenic ocular conditions delivered via intravitreal injection |
| Gene Therapy |
Macular Degeneration |
sVGFR1 |
Preclinical |
CdmoGen |
HN22C0190 |
|
| 8 |
Lead generation of prime editor for genetic variants of Leber congenital amaurosis |
| Gene Therapy |
Leber Congenital Amaurosis |
CEP290 |
Hit |
Seoul National University |
HN21C0917 |
|
