Precision BioSciences獲FDA孤兒藥資格認定用於治療杜氏肌營養不良症

英為財情

2025/07/18

Investing.com -- Precision BioSciences (NASDAQ:DTIL)已獲得美國食品藥品監督管理局(FDA)對其靶向杜氏肌營養不良症的核酸酶治療的孤兒藥資格認定。該認定授予了該公司的腺相關病毒9型遞送的DMD19-20L.431和DMD35-36L.457核酸酶，這些核酸酶正在開發用於治療杜氏肌營養不良症。孤兒藥資格認定授予用於治療、診斷或預防在美國影響不足...

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height: 30px; margin: 0; padding: 0; border-radius: 50%; float: left;}\n.head .h-content { margin: 0; padding: 0 0 0 9px; float: left;}\n.head .h-name {font-size: 13px; color: #eee; margin: 0;}\n.head .h-time {font-size: 12.5px; color: #7E829C; margin: 0;}\n.small {font-size: 12.5px; display: inline-block; transform: scale(0.9); -webkit-transform: scale(0.9); transform-origin: left; -webkit-transform-origin: left;}\n.smaller {font-size: 12.5px; display: inline-block; transform: scale(0.8); -webkit-transform: scale(0.8); transform-origin: left; -webkit-transform-origin: left;}\n.bt-text {font-size: 12px;margin: 1.5em 0 0 0}\n.bt-text p {margin: 0}\n</style>\n</head>\n<body>\n<div class=\"wrapper\">\n<header>\n<h2 class=\"title\">\nPrecision BioSciences獲FDA孤兒藥資格認定用於治療杜氏肌營養不良症\n</h2>\n<h4 class=\"meta\">\n<p class=\"head\">\n<strong class=\"h-name small\">英為財情</strong><span class=\"h-time small\">2025-07-18 00:11</span>\n</p>\n</h4>\n</header>\n<article>\n<div>\n<p>Investing.com -- Precision BioSciences (NASDAQ:DTIL)已獲得美國食品藥品監督管理局(FDA)對其靶向杜氏肌營養不良症的核酸酶治療的孤兒藥資格認定。\n該認定授予了該公司的腺相關病毒9型遞送的DMD19-20L.431和DMD35-36L.457核酸酶，這些核酸酶正在開發用於治療杜氏肌營養不良症。\n孤兒藥資格認定授予用於治療、診斷或預防在美國影響不足...</p>\n\n<a href=\"https://cn.investing.com/news/stock-market-news/article-93CH-2890912\">網頁鏈接</a>\n\n</div>\n\n<div class=\"bt-text\">\n\n\n<p> 來源：<a href=\"https://cn.investing.com/news/stock-market-news/article-93CH-2890912\">英為財情</a></p>\n<p>為提升您的閱讀體驗，我們對本頁面進行了排版優化</p>\n\n\n</div>\n</article>\n</div>\n</body>\n</html>\n","isBrief":false,"type":0,"news_type":1,"symbol":"DTIL","symbol_name":"Precision Biosciences Inc","start_time":0,"source_url":"https://cn.investing.com/news/stock-market-news/article-93CH-2890912","article_id":"2552343604","we_media_id":null,"thumbnails":[],"rights":{"source":"investing_news","url":"https://cn.investing.com/news/stock-market-news/article-93CH-2890912","rn_cache_url":null,"directOrigin":true},"url":"https://stock-news.laohu8.com/highlight/detail?id=2552343604","pubTimestamp":1752768660,"columns":[],"sourceInfo":{"source_id":"investing_news","name":"英为财情新闻"},"weMediaInfo":null,"summary":"Investing.com -- Precision BioSciences 已獲得美國食品藥品監督管理局對其靶向杜氏肌營養不良症的核酸酶治療的孤兒藥資格認定。該認定授予了該公司的腺相關病毒9型遞送的DMD19-20L.431和DMD35-36L.457核酸酶，這些核酸酶正在開發用於治療杜氏肌營養不良症。杜氏肌營養不良症是一種罕見的遺傳性疾病，特徵為肌肉逐漸退化和無力，這是由於一種稱為肌營養不良蛋白的蛋白質發生改變，該蛋白質有助於保持肌肉細胞的完整性。","collect":0,"end_time":0,"defaultTopTitle":"investing.com","property":[],"viewcount":null,"language":"zh","relate_stocks":{"DTIL":"Precision Biosciences Inc","BK4139":"生物科技"},"translate_title":"Precision BioSciences receives FDA orphan drug designation for the treatment of Duchenne muscular dystrophy","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"DTIL":1},"content_text":"Investing.com -- Precision BioSciences (NASDAQ:DTIL)已獲得美國食品藥品監督管理局(FDA)對其靶向杜氏肌營養不良症的核酸酶治療的孤兒藥資格認定。\n該認定授予了該公司的腺相關病毒9型遞送的DMD19-20L.431和DMD35-36L.457核酸酶，這些核酸酶正在開發用於治療杜氏肌營養不良症。\n孤兒藥資格認定授予用於治療、診斷或預防在美國影響不足20萬人的罕見疾病的藥物和生物製品。這一地位為藥物開發商提供了某些優勢，包括合格臨床試驗的税收抵免和潛在的市場獨佔權。\n杜氏肌營養不良症是一種罕見的遺傳性疾病，特徵為肌肉逐漸退化和無力，這是由於一種稱為肌營養不良蛋白的蛋白質發生改變，該蛋白質有助於保持肌肉細胞的完整性。\n\n本文由人工智能協助翻譯。更多信息，請參見我們的使用條款。","kind":"highlight","is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"1","news_tag":"policyRegulatory","news_rank":0,"symbols":[],"gpt_button":0,"need_auth":false,"code":"91000000","status":"200"}}}