Modalis Therapeutics Corporation
TTSE:488330.00
JPY
+1.00
(+3.45%)
Modalis Therapeutics Corporation
TSE:4883
(Tokyo Stock Exchange)
Recent
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30.00
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ROIC.AI
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- Business
- Modalis Therapeutics Corporation (4883.T) engages in the research, development, and commercialization of epigenome editing-based gene therapies targeting serious genetic disorders using its proprietary CRISPR-GNDM platform technology, which enables precise control of gene expression without altering DNA sequences. The company's core pipeline includes in-house candidates such as MDL-101 for LAMA2-related congenital muscular dystrophy (LAMA2-CMD), MDL-201 (GNDM-UTRN) for Duchenne muscular dystrophy (DMD), MDL-103 for facioscapulohumeral muscular dystrophy (FSHD), MDL-202 for myotonic dystrophy type 1 (DM1), MDL-105 for dilated cardiomyopathy (DCM) due to TTN mutations, MDL-104 for tauopathy, MDL-206 for Angelman syndrome, and MDL-207 for Dravet syndrome; it also pursues collaborative pipelines for additional undisclosed indications. Products leverage AAV-based delivery systems to modulate epigenetics at specific loci, addressing muscle disorders, cardiomyopathies, and central nervous system diseases. Founded in January 2016 and headquartered in Tokyo, Japan, with a U.S. subsidiary Modalis Therapeutics Inc. in Waltham, Massachusetts, the company was formerly known as EdiGENE Corporation before renaming in August 2019. It operates primarily in Japan and the United States, targeting rare genetic diseases affecting muscle, heart, and neurological functions in pediatric and adult patients. Recent developments include the addition of MDL-105 to its in-house pipeline for DCM, expanding the portfolio to five in-house programs alongside three collaborative efforts; entry into a strategic partnership with Ginkgo Bioworks in July 2024 to integrate CRISPR-GNDM into Ginkgo's Technology Network for broader applications in cell and gene therapies; and a joint research agreement with JCR Pharmaceuticals Co., Ltd. for central nervous system drug delivery technologies. The company reported operational highlights and third-quarter 2025 financial results in November 2025, alongside expectations of funding from Evolution Capital Management LLC totaling approximately ¥3.072 million. These initiatives underscore Modalis' focus on advancing preclinical programs toward IND-enabling studies and forging alliances to accelerate platform scalability.