- Potential one-time therapy for the treatment of FSHD includes a novel non-cutting dCas protein delivered via a single AAV - - EPI-321 is the only therapy designed to target the epigenetic root cause of the disease - - Company on track for clinical initiation in first half of 2024 - SOUTH SAN FRANCISCO, Calif., Nov.

-用于治疗FSHD的潜在一次性疗法包括通过单一AAV递送的新型非切割dCas蛋白--EPI-321是唯一旨在靶向该疾病的表观遗传根本原因的疗法-公司正在进行临床在2024年上半年开始-南旧金山，加利福尼亚州，11月。

16, 2023 (GLOBE NEWSWIRE) -- Epic Bio, a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD), the most common form of muscular dystrophy in adults.

162023年（GLOBE NEWSWIRE）-一家生物技术公司Epic Bio今天宣布，美国食品和药物管理局（FDA）已授予EPI-321孤儿药名称，用于治疗面肩胛肱型肌营养不良症（FSHD），这是成人最常见的肌营养不良症。

EPI-321 is an investigational therapy being developed as a potential single-dose treatment to suppress abnormal expression of the DUX4 gene. Epic plans to initiate a first-in-human, Phase 1/2 clinical study of EPI-321 in the first half of 2024. The multi-center study will be designed to assess the safety, activity, and preliminary efficacy of EPI-321 in individuals with FSHD.

EPI-321是一种正在开发的研究性疗法，可作为抑制DUX4基因异常表达的潜在单剂量疗法。Epic计划在2024年上半年启动EPI-321的首次人体1/2期临床研究。多中心研究旨在评估EPI-321在FSHD患者中的安全性，活性和初步疗效。

“We are pleased the FDA has recognized the unmet need of those living with FSHD by granting this Orphan Drug Designation, and we believe EPI-321 could serve as an important new therapeutic option for these patients,” said Weston Miller, M.D., chief medical officer of Epic Bio. “We are working diligently to advance EPI-321 toward the clinic, and we look forward to generating meaningful clinical data to inform its future development as a potential new treatment for FSHD.” The FDA has the authority to grant Orphan Drug Designation to therapies to prevent, diagnose or treat a rare disease or condition, defined as those affecting fewer than 200,000 people in the United States.

Weston-Miller博士说：“我们很高兴FDA通过授予这种孤儿药名称，认识到FSHD患者的需求未得到满足，并且我们相信EPI-321可以作为这些患者的重要新治疗选择。”。，Epic Bio的首席医疗官。”我们正在努力将EPI-321推向临床，并期待产生有意义的临床数据，为其作为FSHD潜在新疗法的未来发展提供信息。”FDA有权将孤儿药指定用于预防，诊断或治疗罕见疾病或病症的疗法，定义为影响美国不到20万人的人。

The designation qualifies drug .

该名称符合药物资格。