Intellia治疗公司以CRISPR为基础的新的治疗方法,使林业发展局能够快速地治疗一种罕见、致命的疾病。 Intellia Therapeutics' new CRISPR-based treatment gets FDA fast-track for a rare, deadly disease.
Intellia Therapeutics 因其新的基因编辑疗法 nexiguran ziclumeran 而获得 FDA 的特殊指定，用于治疗一种罕见的、危及生命的疾病，称为遗传性转甲状腺素蛋白淀粉样变性伴多发性神经病。 Intellia Therapeutics has received a special FDA designation for its new gene-editing treatment, nexiguran ziclumeran, for a rare, life-threatening disease called hereditary transthyretin amyloidosis with polyneuropathy. 这种基于CRISPR的治疗旨在通过针对导致该疾病的基因来阻止这一疾病。 This CRISPR-based therapy aims to stop the disease by targeting the gene that causes it. 林业发展局的指定将有助于加快批准程序,有可能使这种创新治疗更快地提供给病人。 The FDA's designation will help speed up the approval process, potentially bringing this innovative treatment to patients sooner.