To determine if long noncoding RNA (lncRNA) LINC01018 has a tumor-suppressing role in hepatocellular carcinoma (HCC), scientists at the Theodor Bilharz Research Institute and the American University in Cairo developed a therapeutic modality to treat mice with experimental disease.
Preclinical data on a novel T-cell therapy based on engineered autologous regulatory T cells (GNTI-122, EngTregs) for the potential treatment of type 1 diabetes (T1D) were presented by Gentibio Inc.
Increasing evidence exists regarding estrogen receptor β (ERβ) playing a protective role in Alzheimer’s disease (AD) and its loss resulting in progressive neural cell body degeneration.
Researchers from Meiragtx Ltd. presented preclinical data on a new and optimized gene therapy based on a mini ATP7B gene for the potential treatment of Wilson’s disease (WD), a rare (1:30,000) autosomal recessive genetic disease caused by loss-of-function mutations in ATP7B, leading to a pathologically high amount of copper in the liver and brain.
Kriya Therapeutics Inc. has presented preclinical data on their novel candidate, KT-A261, an adeno-associated viral vector serotype 2 (AAV2) encoded to express adalimumab, administered as a single intravitreal injection to improve ocular concentrations of adalimumab, for the potential treatment of noninfectious uveitis (NIU).
Glaucoma is an eye disease that damages the optic nerve, with the main cause being ocular hypertension due to high resistance to the outflow of aqueous humor.
New and updated preclinical data presented at the European Society of Gene and Cell Therapy 29th Congress in Edinburgh, Scotland, by: Cellectis, Chameleon, Gentibio, M6P, Vor Biopharma, Voyager Therapeutics.
Researchers from Enanta Pharmaceuticals Inc. presented preclinical data for the small molecule non-nucleoside respiratory syncytial virus (RSV) L-protein inhibitor, EDP-323, being developed for the treatment of RSV infection.
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