R&D Pipeline

R&D Pipeline Pipeline (2021-2030)

Pipeline (2021-2030)

Neurology - Gene & Nucleic acids (16)

Modality Indication Targets Stage Company Project No. Detail
1 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
2 Phase 1 Clinical Development to Evaluate the Safety and Efficacy of IPS101A Gene Therapy Product for Severe Parkinson's Disease
Gene & Nucleic acids Parkinson's Disease Nurr1, Foxa2 Phase 1 Innopeutics Corporation RS-2026-25501504
3 Identification of Lead Candidates for Tissue-Specific Gene Editing Therapeutics for DM1
Gene & Nucleic acids DM1 DMPK Lead NES Biotechnology RS-2025-25455070
4 Development of a Potent Dual-acting Gene Therapy for Spinal Muscular Atrophy
Gene & Nucleic acids SMA SMN2, SMN1 Hit GenKOre RS-2025-25454812
5 Pre-clinical study of ASO drug for treating Parkinson's disease
Gene & Nucleic acids Parkinson's disease Undisclosed Preclinical HLB Neurotobe RS-2025-02223597
6 Non-clinical study for developing a gene therapy of spinal cord injury using cell fate conversion
Gene & Nucleic acids Spinal Cord Injury, SCI Non-neuronal cells (Astrocytic scar, Glial scar, fibroblasts and etc.） Preclinical Stand Up Therapeutics RS-2025-02173013
7 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
8 Development of ASO therapeutics to treat childhood intractable epilepsy
Gene & Nucleic acids Pediatric epilepsy MTOR Candidate Sovargen Co., Ltd. RS-2023-00282544
9 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
10 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
11 Pre-clinical development of AAV-CRISPR-based PMP22 normalization gene therapy for Charcot-Marie-Tooth 1A
Gene & Nucleic acids Charcot-Marie-Tooth disease type 1A PMP22 Preclinical ToolGen, Inc HN22C0431
12 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
13 Developing biomedicine to overcome neuropathic pain by GCH1 expression down-regulation
Gene & Nucleic acids Neuropatic Pain GCH1 Hit Yonsei University HN22C0307
14 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
15 Development of eIF4E ASO treating childhood intractable epilepsy as a first-in-class drug and establishment of R&D environment for RNA therapeutics
Gene & Nucleic acids FCDII Refractory epilepsy eIF4E Preclinical SoVarGen Co., Ltd. HN21C0617
16 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
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