SKY-0515 is a small molecule RNA splicing modifier developed through the company's novel SKYSTAR® platform. SKY-0515's Phase 1 study will include healthy volunteers before targeting Huntington's patients. Huntington's disease is a fatal neurological disease with no curative therapies.

SKY-0515是通过公司新颖的SKYSTAR®平台开发的小分子RNA剪接修饰剂。SKY-0515的第一阶段研究将包括健康志愿者，然后针对亨廷顿病人。亨廷顿舞蹈病是一种致命的神经系统疾病，没有治愈的疗法。

WALTHAM, Mass., Oct. 23, 2023 /PRNewswire/ -- Skyhawk Therapeutics, Inc., a clinical-stage biotechnology company developing novel small molecule RNA splicing modifiers, today announced the Australian Human Research Ethic Committee (HREC) approval by Australian regulators for a clinical trial for Skyhawk's SKY-0515 small molecule candidate targeting Huntington's disease (HD).

马萨诸塞州沃尔瑟姆，2023年10月23日/PRNewswire/-Skyhawk Therapeutics，Inc。是一家开发新型小分子RNA剪接修饰剂的临床阶段生物技术公司，今天宣布澳大利亚人类研究伦理委员会（HREC）批准Skyhawk的SKY-0515小分子候选靶向亨廷顿舞蹈病（HD）的临床试验。

Huntington's is caused by a toxic 'CAG' repeat in the huntingtin (HTT) gene, causing mutated huntingtin protein. SKY-0515 is designed to modify the RNA expression of HTT, reducing the production of mutated protein that leads to disease progression in Huntington's patients..

亨廷顿舞蹈病是由亨廷顿蛋白（HTT）基因中的毒性'CAG'重复引起的，导致亨廷顿蛋白突变。SKY-0515旨在修饰HTT的RNA表达，减少导致亨廷顿舞蹈病患者疾病进展的突变蛋白的产生。。

'SKY-0515 provides exciting potential for Huntington's disease patients and leads a growing pipeline of RNA-targeting therapies developed by our SKYSTAR® platform,' said Bill Haney, CEO and Co-Founder of Skyhawk Therapeutics. 'Huntington's patients presently have few therapeutic options and none that reverse or slow disease progression.

“Skyhawk Therapeutics首席执行官兼联合创始人Bill Haney说，”SKY-0515为亨廷顿舞蹈病患者提供了令人兴奋的潜力，并领导了由我们的SKYSTAR®平台开发的越来越多的RNA靶向治疗方案亨廷顿舞蹈病患者目前几乎没有治疗选择，也没有逆转或减缓疾病进展的选择。

SKY-0515 has demonstrated strong activity in our preclinical models which we believe will translate to patient benefit.'.

SKY-0515在我们的临床前模型中表现出强烈的活性，我们相信这将转化为患者的益处。

There are approximately 30,000 symptomatic Huntington's disease patients in the US alone and many more that are pre-symptomatic. There are currently no approved drugs that reverse or slow disease progression for Huntington's patients. SKY-0515 is a small molecule which directly targets HTT RNA, is dosed orally and offers brain penetration and distribution in peripheral tissues which may provide important benefits to patients.

仅在美国就有大约30000名有症状的亨廷顿舞蹈病患者，还有更多的是有症状的亨廷顿舞蹈病患者。目前还没有批准的药物可以逆转或减缓亨廷顿舞蹈病患者的疾病进展。SKY-0515是一种直接靶向HTT RNA的小分子，口服给药并在外周组织中提供脑渗透和分布，这可能为患者提供重要益处。

It is the first Skyhawk-developed drug to enter clinical trials..

它是第一个进入临床试验的Skyhawk开发药物。。

Skyhawk Therapeutics' SKY-0515 Phase 1 clinical trial is a first-in-human, multi-part, randomized, double-blind, single and multiple ascending dose study designed to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamic activity of SKY-0515 monotherapy in healthy volunteers and, subsequently, Huntington's patients.

Skyhawk Therapeutics的SKY-0515 1期临床试验是首次人体，多部分，随机，双盲，单次和多次递增剂量研究，旨在研究SKY-0515的安全性，耐受性，药代动力学和药效学活性在健康志愿者和随后的亨廷顿病患者中进行单一疗法。

Additional information about the trial can be found at: Australian New Zealand Clinical Trials Registry (https://www.anzctr.org.au)..

有关该试验的更多信息，请访问：澳大利亚新西兰临床试验注册处(https://www.anzctr.org.au)..

About SKY-0515Skyhawk Therapeutics is developing a small molecule RNA splicing modifier for potential treatment of Huntington's disease as part of the company's novel SKYSTAR® platform. SKY-0515 is designed to reduce the production of the mutated huntingtin protein which results in disease progression.

关于SKY-0515Skyhawk Therapeutics正在开发一种小分子RNA剪接修饰剂，用于治疗亨廷顿舞蹈病，作为该公司新型SKYSTAR®平台的一部分。SKY-0515旨在减少导致疾病进展的突变亨廷顿蛋白的产生。

SKY-0515's predicted PK allows for daily, oral dosing, and the molecule is designed for brain penetration and distribution in peripheral tissue..

SKY-0515预测的PK允许每日口服给药，并且该分子设计用于脑渗透和分布在外周组织中。。

About Huntington's diseaseHuntington's disease is a rare, inherited disease that causes the progressive degeneration of nerve cells in the brain. Huntington's disease has a wide impact on a person's functional abilities and usually results in movement, cognitive and psychiatric disorders. Huntington's disease symptoms can develop at any time, but they often first appear when people are in their 30s or 40s.

关于亨廷顿舞蹈病亨廷顿舞蹈病是一种罕见的遗传性疾病，可导致大脑神经细胞的进行性退化。亨廷顿舞蹈病对一个人的功能能力有广泛的影响，通常会导致运动，认知和精神疾病。亨廷顿舞蹈病的症状可以随时发展，但通常在人们30多岁或40多岁时首次出现。

If the condition develops before age 20, it's called juvenile Huntington's disease. When Huntington's develops early symptoms are somewhat different and the disease may progress faster. Medications are available to help manage the symptoms of Huntington's disease, but there is no cure for Huntington's disease, and currently no approved drugs that delay the disease onset or slow disease progression..

如果病情在20岁以前发展，则称为青少年亨廷顿舞蹈病。当亨廷顿舞蹈病发展时，早期症状有所不同，疾病可能进展得更快。药物可用于帮助控制亨廷顿舞蹈病的症状，但亨廷顿舞蹈病尚无治愈方法，目前尚无批准的可延缓疾病发作或减缓疾病进展的药物。。

About SkyhawkSkyhawk develops and commercializes therapies using its novel SKYSTAR® platform, building small molecules that modify RNA expression to provide breakthrough treatments for patients. Skyhawk has productive collaborations across a broad range of disease areas, from neurodegenerative disease to oncology..

关于SkyhawkSkyhawk使用其新颖的SKYSTAR®平台开发和商业化疗法，构建修饰RNA表达的小分子，为患者提供突破性治疗。Skyhawk在从神经退行性疾病到肿瘤学的各个疾病领域都有着丰富的合作。。

For more information visit:www.skyhawktx.comhttps://www.linkedin.com/company/skyhawk-therapeutics/ https://twitter.com/Skyhawk_Tx

For more information visit:www.skyhawktx.comhttps://www.linkedin.com/company/skyhawk-therapeutics/ https://twitter.com/Skyhawk_Tx

SKYHAWK MEDIA CONTACT:Maura McCarthy [email protected]

SKYHAWK媒体联系人：Maura McCarthy[电子邮件保护]

SOURCE Skyhawk Therapeutics

源Skyhawk疗法