pgn-edo51是基于其自有平台开发的一款多肽-寡核苷酸偶联药物，旨在通过跳过dmd基因中的外显子51来进行治疗。 这是一项开放标签MAD临床试验，旨在评估PGN-EDO51在三组卧床和非卧床的DMD男性少年/青年患者中的疗效和安全性。

PGN-EDO51: 一种DMD exon 51调节剂、肌营养不良蛋白表达刺激剂、RNA干扰药物，由PepGen Inc. (PepGen Inc.)公司最早进行研发，目前全球最高研发状态为暂停，作用机制: DMD exon 51调节剂(DMD 外显子51调节剂),肌营养不良蛋白表达刺激剂,RNA干扰，治疗领域: 神经系统疾病,遗传 …

2023年4月25日 · PepGen’s EDO cell-penetrating peptide technology is engineered to optimize tissue delivery and cellular uptake of therapeutic oligonucleotides. PGN-EDO51 is being evaluated for the treatment of DMD amenable to exon 51 skipping.

2024年7月31日 · PGN-EDO51, PepGen's lead clinical candidate for the treatment of Duchenne muscular dystrophy (DMD), utilizes the Company's proprietary Enhanced Delivery Oligonucleotide (EDO) technology to deliver a therapeutic phosphorodiamidate morpholino oligomer (PMO) that is designed to target the root cause of this devastating disease.

2024年12月6日 · PGN-EDO51是PepGen重点研发的一款多肽-寡核苷酸偶联药物，目前正处于临床二期研究阶段。 其中核心组成部分EDO肽，凭借其卓越的递送能力，实现了对寡核苷酸的有效运输。

2022年9月28日 · PGN-EDO51, PepGen’s lead clinical candidate for the treatment of Duchenne muscular dystrophy (DMD), utilizes the company’s proprietary Enhanced Delivery Oligonucleotide (EDO) technology to ...

2023年10月1日 · PGN-EDO51 is PepGen's clinical candidate for treatment of DMD amenable to exon 51 skipping. A Phase 1 single-ascending dose study in healthy adult male volunteers (HV) evaluated safety, tolerability, pharmacokinetics (plasma and muscle), and pharmacodynamics (exon skipping) of PGN-EDO51.

2 天之前 · The application of 4 doses of PGN-EDO51 over 3 months was able to show evidence of exon skipping (2.15%) along with a 0.70% increase in adjusted dystrophy and 0.26% increase in unadjusted dystrophin levels compared to baseline. PGN-EDO51 is PepGen’s investigational clinical candidate for the treatment of DMD amenable to exon 51 skipping.

PGN-EDO51是基于PepGen自有的增强型递送寡核苷酸（EDO）平台开发的一款多肽-寡核苷酸偶联药物，旨在通过跳过 DMD基因 中的外显子51来进行治疗。 Ⅰ期临床 为一项单剂量递增试验，旨在评估PGN-EDO51在32名健康成年男性中的安全性和耐受性。

2024年3月13日 · PGN-EDO51 is designed to skip exon 51 of the dystrophin transcript, an established therapeutic target for approximately 13% of DMD patients, thereby aiming to restore the open reading frame and enabling the production of a …