| 1 |
Development of AAV.based regenerative medicine expressing HGF gene (AAV9.HGF) for the treatment of ALS disease |
| Gene Therapy |
Amyotrophic Lateral Sclerosis |
HGF/c.Met signaling pathway |
Candidate |
Helixmith Co.,Ltd. |
RS-2023-00283601 |
|
| 2 |
Development of ASO therapeutics to treat childhood intractable epilepsy |
| Gene Therapy |
Pediatric epilepsy |
MTOR |
Candidate |
Sovargen Co., Ltd. |
RS-2023-00282544 |
|
| 3 |
Development of gene.editing therapeutics for Huntington’s disease with CAG repeat expansion |
| Gene Therapy |
Huntington's disease |
HTT gene(CAG.repeat) |
Hit |
GenKOre |
RS-2023-00259174 |
|
| 4 |
Investigation of proper candidates for in vivo direct lineage reprogramming based SCI gene therapy development |
| Gene Therapy |
Spinal cord injury |
astrocyte |
Candidate |
Stand Up Therapeutics |
RS-2022-00167083 |
|
| 5 |
Pre-clinical development of AAV-CRISPR-based PMP22 normalization gene therapy for Charcot-Marie-Tooth 1A |
| Gene Therapy |
Charcot-Marie-Tooth disease type 1A |
PMP22 |
Preclinical |
ToolGen, Inc |
HN22C0431 |
|
| 6 |
Developing biomedicine to overcome neuropathic pain by GCH1 expression down-regulation |
| Gene Therapy |
Neuropatic Pain |
GCH1 |
Hit |
Yonsei University |
HN22C0307 |
|
| 7 |
Development of eIF4E ASO treating childhood intractable epilepsy as a first-in-class drug and establishment of R&D environment for RNA therapeutics |
| Gene Therapy |
FCDII Refractory epilepsy |
eIF4E |
Preclinical |
SoVarGen Co., Ltd. |
HN21C0617 |
|
| 8 |
Development of treatment for neurodevelopmental disorder (Rett syndrome) based on RNA replacement technology using the next-generation efficacy evaluation method |
| Gene Therapy |
Rett syndrome |
MeCP2 |
Lead |
Rznomics Inc. |
HN21C1329 |
|
