R&D Pipeline

Pipeline (2021-2030)

Neurology - Discovery (36)

Modality Indication Targets Stage Company Project No. Detail
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