| 1 |
Development of a Potent Dual-acting Gene Therapy for Spinal Muscular Atrophy |
| Gene & Nucleic acids |
SMA |
SMN2, SMN1 |
Hit |
GenKOre |
RS-2025-25454812 |
|
| 2 |
Discovery of lead compounds targeting TDP43 for treatment of Amyotrophic lateral sclerosis (ALS) |
| Small molecules |
ALS |
TDP43 |
Hit |
KIST |
RS-2025-25453059 |
|
| 3 |
Development of first-in-class tuberous sclerosis brain disease therapeutic targeting Rheb GTPase |
| Small molecules |
Tuberous Sclerosis |
Rheb |
Hit |
K-medihub/New drug development center |
RS-2025-02213662 |
|
| 4 |
Discovery of PIP5Kgamma inhibitor for treatment of Parkinson's disease |
| Small molecules |
Parkinson's disease |
PIP5Kγ |
Hit |
K.MEDI hub |
RS-2023-00283494 |
|
| 5 |
Development of gene.editing therapeutics for Huntington’s disease with CAG repeat expansion |
| Gene & Nucleic acids |
Huntington's disease |
HTT gene(CAG.repeat) |
Hit |
GenKOre |
RS-2023-00259174 |
|
| 6 |
Development of therapeutic agent for Parkinson's disease targeting alpha synuclein aggregates using autophagy targeting chimera (AUTOTAC) |
| TPD |
Parkinson's disease |
α-Syn |
Hit |
AUTOTAC Bio Inc. |
RS-2022-00166787 |
|
| 7 |
Developing biomedicine to overcome neuropathic pain by GCH1 expression down-regulation |
| Gene & Nucleic acids |
Neuropatic Pain |
GCH1 |
Hit |
Yonsei University |
HN22C0307 |
|
| 8 |
Development of an exosome-mediated intracellular therapeutic protein delivery system for Parkinson's disease |
| Other-Bio. |
Parkinson's disease |
Parkin, PGC-1α |
Hit |
Sungkyunkwan University |
HN21C1266 |
|
| 9 |
Development of a lead compound for preventing Parkinson’s disease using small compounds with inhibitory mechanism of initial alpha-synuclein aggregation |
| Small molecules |
Parkinson's disease |
α-synuclein |
Hit |
Hanyang University |
HN21C1258 |
|
| 10 |
Development of lead compounds as antidepressant having both α2 adrenergic antagonist and NDRI activities |
| Small molecules |
Major depressive disorder |
A2 adrenergic, NDRI |
Hit |
Kyung Hee University |
HN21C1076 |
|
| 11 |
AB103: Development of a Lead Gene Therapy Candidate for Parkinson’s Disease Based on α-Synuclein Aggregation Inhibition |
| Gene & Nucleic acids |
Parkinson's Disease |
α-synuclein |
Lead |
ABrain Inc. |
RS-2026-25504113 |
|
| 12 |
Identification of Lead Candidates for Tissue-Specific Gene Editing Therapeutics for DM1 |
| Gene & Nucleic acids |
DM1 |
DMPK |
Lead |
NES Biotechnology |
RS-2025-25455070 |
|
| 13 |
Development of a lead compound for CT contrast agents in the quantitative diagnosis of acute cerebral infarction and establishment of personalized diagnostic and therapeutic strategies for stroke patients |
| Small molecules |
Ischemic stroke |
Fibrin within the thrombus |
Lead |
Seoul National University R&DB Foundation |
RS-2025-02223267 |
|
| 14 |
Lead optimization of Tau-ATTEC as a therapeutic agent for progressive supranuclear palsy |
| TPD |
Taupathy |
Tau |
Lead |
Korea Research Institute of Chemical Technology |
RS-2025-02223246 |
|
| 15 |
Development of Lead Compounds for Parkinson’s Disease Treatment by Regulating Adult Neurogenesis through ERRTargeted Small Molecules |
| Small molecules |
Parkinson’s disease |
Estrogen-related receptor gamma |
Lead |
CHA university |
RS-2024-00463446 |
|
| 16 |
Development of Parkinson’s Disease Therapeutics through Degradation of Pathological α-Synuclein Protein Using the SPiDEM Platform |
| TPD |
Parkinson’s disease |
α-synuclein |
Lead |
PRAZER THERAPEUTICS INC. |
RS-2024-00445011 |
|
| 17 |
Discovery of Lead Candidates for Parkinson’s disease targeting novel TAS2R GPCR |
| Small molecules |
Parkinson's disease |
TAS2R |
Lead |
DIGMBIO |
RS-2023-00219269 |
|
| 18 |
Discovery of a novel lead compound for chronic pain treatments using a TRPV1 binding peptides design and manufacture platform |
| rProtein |
Chronic Pain |
TRPV1 |
Lead |
Gachon University |
RS-2022-00167087 |
|
| 19 |
The lead selection study of TLR2 targeted antisense oligonucleotide
using PNA as a therapeutic agent for Parkinson’s Disease (PD) |
| Gene & Nucleic acids |
Parkinson's Disease |
TLR2 |
Lead |
SEASUN THERAPEUTICS, Inc. |
HN22C0411 |
|
| 20 |
Discovery of a lead compound utilizing exon skipping platform for the treatment of
amyotrophic lateral sclerosis |
| Gene & Nucleic acids |
Amyotrophic Lateral Sclerosis |
STMN2 |
Lead |
OliPass corporation |
HN22C0017 |
|
| 21 |
Lead identification of ASK1 inhibitor as a therapeutic agent for Parkinson’s disease |
| Small molecules |
Parkinson's disease |
ASK1 |
Lead |
Korea Research Institute of Chemical Technology |
HN21C1139 |
|
| 22 |
Discovery of Lead Compounds for the Treatment of Progressive Supranuclear Palsy by Controlling 4R Tauopathy |
| Small molecules |
Progressive supranuclear palsy, 4R tauopathies |
4R tau oligomer |
Lead |
Korea Institute of Science and Technology |
HN21C0315 |
|
| 23 |
Development of treatment for neurodevelopmental disorder (Rett syndrome) based on RNA replacement technology using the next-generation efficacy evaluation method |
| Gene & Nucleic acids |
Rett syndrome |
MeCP2 |
Lead |
Rznomics Inc. |
HN21C1329 |
|
| 24 |
Development of Preclinical Candidates Targeting Novel TAS2R GPCR for Parkinson's disease |
| Small molecules |
Parkinson's disease |
TAS2R GPCR |
Candidate |
DigmBio |
RS-2025-25453249 |
|
| 25 |
Nrf2-targeting small molecule candidate development for parkinson's disease |
| Small molecules |
Parkinson's Disease |
Keap1-Nrf2 axis |
Candidate |
SPARKBiopharma |
RS-2025-25449417 |
|
| 26 |
Development of structurally modified microbial small molecule of multi-MOA for Parkinson's disease |
| Small molecules |
Parkinson's disease |
FKBP (FK506-binding protein) |
Candidate |
MolGenBio Co., Ltd. |
RS-2025-25444385 |
|
| 27 |
Development of a novel small molecule targeting ASK1 for the treatment of Parkinson’s disease |
| Small molecules |
Parkinson's disease |
ASK1 |
Candidate |
Naason Science Inc. |
RS-2025-02213938 |
|
| 28 |
AAK1 Inhibition for the Treatment of Neuropathic Pain: Identification and Evaluation of Novel Candidates |
| Small molecules |
Pain |
AAK1 |
Candidate |
CIMPLRX Co. Ltd. |
RS-2025-02213667 |
|
| 29 |
Development of Candidate Compounds for Therapeutic Intervention in Ischemic Brain Injury: Targeting MMP-9 Inhibition to Prevent Blood-Brain Barrier Disruption, Neuronal Degeneration, and Neuroinflammatory Responses. |
| Small molecules |
Ishemic brain injury |
MMP-9 |
Candidate |
Zincure Corp |
RS-2025-02182973 |
|
| 30 |
Preclinical development of an anti-neuroinflammatory drug for the treatment of Parkinson’s disease |
| TPD |
Parkinson's disease |
Neuroinflammation |
Candidate |
Aevisbio, Inc |
RS-2024-00338017 |
|
| 31 |
Development of AAV.based regenerative medicine expressing HGF gene (AAV9.HGF) for the treatment of ALS disease |
| Gene & Nucleic acids |
Amyotrophic Lateral Sclerosis |
HGF/c.Met signaling pathway |
Candidate |
Helixmith Co.,Ltd. |
RS-2023-00283601 |
|
| 32 |
Development of therapeutic antibody for the treatment of myasthenia gravis |
| Antibody |
Myasthenia gravis |
Disclosure after confidentiality agreement |
Candidate |
Medytox Inc. |
RS-2023-00283006 |
|
| 33 |
Development of ASO therapeutics to treat childhood intractable epilepsy |
| Gene & Nucleic acids |
Pediatric epilepsy |
MTOR |
Candidate |
Sovargen Co., Ltd. |
RS-2023-00282544 |
|
| 34 |
Discovery the preclinical candidate for amyotrophic lateral sclerosis (ALS) based on the activation of lysosomes using small-molecular proteins |
| rProtein |
Amyotrophic lateral sclerosis |
Zn2+ |
Candidate |
ZINCURE |
RS-2023-00218770 |
|
| 35 |
Investigation of proper candidates for in vivo direct lineage reprogramming based SCI gene therapy development |
| Gene & Nucleic acids |
Spinal cord injury |
astrocyte |
Candidate |
Stand Up Therapeutics |
RS-2022-00167083 |
|
| 36 |
Development of small molecule LRRK2 inhibitor for Parkinson’s disease treatment |
| Small molecules |
Parkinson's disease |
LRRK2 |
Candidate |
Whan In Pharm. Co., Ltd. |
HN21C0655 |
|