Epicrispr披露了FSHD的遗传疗法EPI-321,2025年初临床试验针对D4Z4区域。 Epicrispr reveals epigenetic therapy EPI-321 for FSHD, targeting D4Z4 region in early 2025 clinical trials.

在ESGCT大会上,Epicrispr生物技术公司展示了其用于治疗面额骨肌缩症 (FSHD) 的EPI-321治疗的表观遗传编辑平台和数据. Epicrispr Biotechnologies showcased its epigenetic editing platform and data for its therapy EPI-321 aimed at treating facioscapulohumeral muscular dystrophy (FSHD) at the ESGCT Congress. 新的表观遗传调节器可以在75天内使已被静止的基因重新激活到近100%的表达. The new epigenetic modulator can reactivate a silenced gene to nearly 100% expression for over 75 days. Epicrispr计划在2025年初开始EPI-321临床试验,针对染色体4的D4Z4区域,以抑制有害的DUX4基因表达方式。 Epicrispr plans to start clinical trials for EPI-321 in early 2025, targeting the D4Z4 region on chromosome 4 to inhibit harmful DUX4 gene expression.