Solid Biosciences has announced that its Investigational New Drug (IND) application for SGT-212 has been cleared by the U.S. Food and Drug Administration (FDA).

Solid Biosciences宣布，其针对SGT-212的研究性新药（IND）申请已获得美国食品和药物管理局（FDA）的批准。

FA affects approximately 5,000 individuals in the United States and 15,000 in Europe. It is an inherited, life-threatening disorder caused by defects in the frataxin gene, leading to progressive nervous system and cardiac dysfunction.

FA影响美国约5000人，欧洲约15000人。它是一种遗传性的危及生命的疾病，由frataxin基因缺陷引起，导致进行性神经系统和心脏功能障碍。

Currently, there are no treatments that cure or halt the progression of FA, highlighting the significant unmet need for innovative therapies like SGT-212.

目前，没有治疗方法可以治愈或阻止FA的进展，这突出表明对SGT-212等创新疗法的需求尚未得到满足。

This gene therapy candidate is designed to address Friedreich’s ataxia (FA), a degenerative disease resulting from insufficient levels of the frataxin protein.

该基因治疗候选物旨在解决弗里德里希共济失调（FA），这是一种由frataxin蛋白水平不足引起的退行性疾病。

SGT-212 was developed through a collaboration involving FA212 LLC, the University of Pennsylvania, and Solid Biosciences. The therapy uses a recombinant AAV-based gene replacement approach to deliver full-length human frataxin.

SGT-212是通过FA212 LLC，宾夕法尼亚大学和Solid Biosciences的合作开发的。该疗法使用基于重组AAV的基因替代方法来递送全长人frataxin。

This dual-administration method seeks to restore mitochondrial function in neurons and cardiomyocytes, addressing both neurological and cardiac symptoms of the disease.

这种双重给药方法旨在恢复神经元和心肌细胞的线粒体功能，解决该疾病的神经和心脏症状。

SGT-212 employs a dual route of administration, delivering therapy both intravenously and directly into the cerebellar dentate nuclei via intradentate nuclei (IDN) infusion.

SGT-212采用双重给药途径，通过齿内核（IDN）输注静脉内和直接向小脑齿状核提供治疗。

This approach aims to address both the neurological and cardiac symptoms associated with the disease, offering a more comprehensive treatment strategy.

这种方法旨在解决与疾病相关的神经和心脏症状，提供更全面的治疗策略。

FA is a complex, multisystem condition that presents significant challenges for treatment development. It requires precise delivery of the frataxin protein to avoid cardiac toxicity while also targeting the cerebellum to provide neurological benefits. SGT-212 is the first therapy in development designed to address these challenges through its dual-administration approach, focusing on the dentate nuclei for neurological treatment and cardiac tissue for systemic manifestations..

FA是一种复杂的多系统疾病，对治疗发展提出了重大挑战。它需要精确递送frataxin蛋白以避免心脏毒性，同时还需要靶向小脑以提供神经益处。SGT-212是开发中的第一种疗法，旨在通过其双重给药方法解决这些挑战，重点是用于神经治疗的齿状核和用于全身表现的心脏组织。。

Preclinical studies have shown promising results, demonstrating safe delivery of frataxin to target tissues, improved neurological function, and reversal of cardiac symptoms in animal models. These findings support the dual-route methodology as a potentially effective treatment for FA.

临床前研究显示出有希望的结果，证明frataxin可以安全地传递到靶组织，改善神经功能，并在动物模型中逆转心脏症状。这些发现支持双途径方法作为FA的潜在有效治疗方法。

A Phase 1b clinical trial for SGT-212 is expected to commence in the second half of 2025. The trial will involve ambulatory and non-ambulatory adult patients with FA, assessing safety and tolerability across up to three patient cohorts. Participants will be monitored for five years following treatment..

SGT-212的1b期临床试验预计将于2025年下半年开始。该试验将涉及非卧床和非卧床FA成年患者，评估多达三个患者队列的安全性和耐受性。治疗后将对参与者进行为期五年的监测。。

This milestone reflects years of collaborative research and development involving patient advocacy groups, research institutions, and academic partners, aiming to improve outcomes for individuals living with FA.

这一里程碑反映了多年来涉及患者倡导团体，研究机构和学术合作伙伴的合作研究和开发，旨在改善FA患者的预后。

Source: solidbio.com

来源：solidbio.com