PGN-EDO51: Preclinical Data Supporting the Development of an Enhanced Delivery Oligonucleotide (EDO) for the Treatment of Duchenne Muscular Dystrophy (DMD)

被引：2
|
作者：
Holland, Ashling [1 ]
Godfrey, Caroline [1 ]
Lonkar, Pallavi [1 ]
Svenstrup, Niels [1 ]
Larkindale, Jane [1 ]
McArthur, James [1 ]
Bracegirdle, Sonia [1 ]
Goyal, Jaya [1 ]
机构：
[1] PepGen Inc, Cambridge, MA USA
来源：
NEUROLOGY | 2023年 / 100卷 / 17期
关键词：
D O I：
10.1212/WNL.0000000000203724
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
P3-8.002
引用
收藏
页数：3
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  • [1] PGN-EDO51, an Enhanced Delivery Oligonucleotide (EDO) for the Treatment of Duchenne Muscular Dystrophy (DMD): Results of a Phase 1 Study in Healthy Volunteers
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  • [4] Single- and repeat-dose nonclinical data for PGN-EDO51 demonstrate potential for the treatment of Duchenne muscular dystrophy (DMD)
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    NEUROMUSCULAR DISORDERS, 2023, 33 : S103 - S103
  • [5] CONNECT-EDO51: Trial designs to support the development of PGN-EDO51 for Duchenne Muscular dystrophy amenable to exon 51 skipping
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  • [6] Phase 1 study of PGN-EDO51 demonstrates tolerability, delivery and high levels of exon skipping for treatment of Duchenne muscular dystrophy (DMD)
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  • [9] CONNECT2-EDO51: a Phase 2 placebo-controlled study to evaluate PGN-EDO51 safety and efficacy in people with Duchenne amenable to exon 51 skipping
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  • [10] CONNECT1-EDO51: a 12-week open-label Phase 2 study to evaluate PGN-EDO51 safety and efficacy in people with Duchenne amenable to exon 51 skipping
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