R&D Pipeline

R&D Pipeline Pipeline (2021-2030)

Pipeline (2021-2030)

Ophthalmology - Gene & Nucleic acids (14)

Modality Indication Targets Stage Company Project No. Detail
1 Development of STING1 Signal Regulating Lipid Nanoparticle Treatment for Sjogren Syndrome Using Conjunctive Organoid Model
Gene & Nucleic acids Severe ocular surface inflammation and dry eye disease associated with Sjögren syndrome STING (Stimulator of Interferon Genes) Hit Yonsei University RS-2026-25503159
2 Development of AI platform-based AAV engineering and gene therapy for choroideremia
Gene & Nucleic acids Choroideremia CHM gene Hit Aavatar Therapeutics RS-2026-25502043
3 Clinical Development and Commercialization of NG101: A Gene Therapy for Wet Age-Related Macular Degeneration
Gene & Nucleic acids wet-AMD VEGF Phase 2 Neuracle Genetics RS-2025-02213615
4 AAV Gene Therapy for Degenerative Retinal Diseases
Gene & Nucleic acids Dry-AMD(Age-related Macular Degeneration), Inherited retinal disease Nkx3.2 Candidate ICM CO., LTD. RS-2024-00344469
5 Development of Retina-Specific AAV and Gene-Editing Therapeutics for Leber Congenital Amaurosis type 10 based on the TaRGET System
Gene & Nucleic acids Leber’s congenital amaurosis 10 CEP290 Lead GenKOre RS-2024-00339507
6 Development of new compounds for effective gene editing therapy for an IMPDH1 mutation causing autosomal dominant retinitis pigmentosa
Gene & Nucleic acids Retinitis Pigmentosa IMPDH1 Hit Ulsan University RS-2023-00283544
7 Development of First.in.Class mutant transcript.selective ASO therapeutic candidates for the treatment of Avellino corneal dystrophy
Gene & Nucleic acids Avellino corneal dystrophy Human TGFBIR124H Candidate MediciBIO Co., LTD RS-2023-00282731
8 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 & Nucleic acids Retinitis Pigmentosa RPGR Hit ENCell RS-2023-00258840
9 Discovery of Non-clinical Development Candidate Molecule for a Novel Universal Retinitis Pigmentosa Therapeutics
Gene & Nucleic acids Retinitis Pigmentosa RORβ Candidate OliX Pharmaceuticals RS-2023-00217014
10 Development of gene therapeutics targeting EYS-related Retinitis Pigmentosa
Gene & Nucleic acids Retinitis pigmentosa EYS c.4957dupA Hit Sungkyunkwan University HN22C0571
11 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 & Nucleic acids Autosomal dominant Retinitis pigmentosa (adRP) P23H Preclinical Rznomics Inc. HN22C0300
12 Development of novel gene therapy for PDE6B related inherited retinal disease
Gene & Nucleic acids Genetic Retinal disease PDE6B Hit Asan Medical Center HN22C0245
13 CRG-02: An AAV-based anti-VEGF gene therapy for angiogenic ocular conditions delivered via intravitreal injection
Gene & Nucleic acids Macular Degeneration sVGFR1 Preclinical CdmoGen HN22C0190
14 Lead generation of prime editor for genetic variants of Leber congenital amaurosis
Gene & Nucleic acids Leber Congenital Amaurosis CEP290 Hit Seoul National University HN21C0917
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