Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them. However, many approaches still struggle to reach target cells or tissues – either they fail to arrive, or their efficacy is low. In vivo therapies face numerous challenges, but despite these hurdles, 2025 has marked a year of remarkable progress.
Chengdu Easton Biopharmaceuticals Co. Ltd. has divulged sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain, arrhythmia, cough, urinary incontinence and multiple sclerosis.
Neurodawn Pharmaceutical Co. Ltd. has synthesized phosphodiesterase PDE3A inhibitors reported to be useful for the treatment of dementia and cerebrovascular disorders.
Multiple sclerosis (MS) is a chronic immune-mediated disease characterized by the destruction of myelin sheaths, neuroaxonal damage, glial cell activation and formation of demyelinated plaques in the CNS. Since MS is considered a prototypic antigen-specific autoimmune disease, restoring immune tolerance to self-antigens is being explored as a therapeutic strategy.
U.S. policy, China’s strategic rise, blockbuster deals and AI dominated South Korea’s biotechnology industry this year, with U.S. tariffs and the Biosecure Act’s hitch onto 2026 legislation serving as major topics of speculation.
U.S. policy, China’s strategic rise, blockbuster deals and AI dominated South Korea’s biotechnology industry this year, with U.S. tariffs and the Biosecure Act’s hitch onto 2026 legislation serving as major topics of speculation.
Neushen Therapeutics Inc. has disclosed N-acetyl-β-D-glucosaminidase (O-GlcNAcase; OGA) inhibitors reported to be useful for the treatment of Alzheimer’s disease.
Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them. However, many approaches still struggle to reach target cells or tissues – either they fail to arrive, or their efficacy is low. In vivo therapies face numerous challenges, but despite these hurdles, 2025 has marked a year of remarkable progress.
Gene therapy has had its commercial struggles in the past year. The cost to patients is in the millions and fewer are stepping forward for treatment than companies would like. While development continues in this game-changing field, some have struggled with regulatory authorities during development while others have just stepped away altogether.
Shionogi & Co. Ltd. will acquire global rights to U.S. FDA approved amyotrophic lateral sclerosis therapy edaravone through a $2.5 billion acquisition deal with Tanabe Pharma Corp. Under the terms, Tanabe will form a new entity harboring both oral and intravenous (I.V.) infusion formulations of edaravone that are marketed in the U.S. as Radicava ORS and I.V. Radicava.
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