天鹰治疗公司出席2025年亨廷顿舞蹈症青年组织国际大会-动脉网

Skyhawk Therapeutics Presents at Huntington's Disease Youth Organization HDYO International Congress 2025

CISION 等信源发布 2025-03-15 18:07



可切换为仅中文







Skyhawk presents today on its novel SKY-0515 small molecule RNA splicing modulator targeting

天鹰今天介绍了其新型SKY-0515小分子RNA剪接调节剂，靶向

Huntington's

亨廷顿氏

Disease to members of the

疾病对成员的

Huntington's

亨廷顿病

Disease Youth Organization, at the annual HYDO International Congress in

疾病青年组织，年度HYDO国际大会在

Prague, Czech Republic

捷克共和国布拉格

。

BOSTON

波士顿

，

March 15, 2025

2025年3月15日

/PRNewswire/ --

Skyhawk Therapeutics, Inc.,

天鹰治疗公司，

a clinical-stage biotechnology company developing novel small molecule therapies designed to modulate critical RNA targets, today presents on its novel SKY-0515 small molecule RNA splicing modulator targeting

一家临床阶段的生物技术公司，致力于开发新型小分子疗法以调节关键的RNA靶点，今天介绍了其新型SKY-0515小分子RNA剪接调节剂，该调节剂针对

Huntington's

亨廷顿氏舞蹈症

Disease to members of the

疾病对于成员们来说

Huntington's

亨廷顿氏舞蹈症

Disease Youth Organization (HDYO), at the biannual HYDO International Congress in

疾病青年组织 (HDYO)，在每两年一次的HYDO国际大会上

Prague, Czech Republic

捷克共和国布拉格市

。

继续阅读

SKY-0515 is Skyhawk's investigational small molecule RNA splicing modifier developed through the company's novel RNA-splicing platform. SKY-0515 is designed to reduce both HTT protein and PMS1 protein, an additional key driver of somatic CAG repeat expansion and HD pathology.

SKY-0515 是 Skyhawk 公司通过其新型 RNA 剪接平台开发的在研小分子 RNA 剪接修饰剂。SKY-0515 旨在减少 HTT 蛋白和 PMS1 蛋白，PMS1 蛋白是体细胞 CAG 重复扩增和 HD 病理学的另一个关键驱动因素。

Skyhawk Presents at Huntington's Disease Youth Organization International Congress 2025

天鹰在2025年亨廷顿舞蹈症青年组织国际大会上发表演讲

Post this

发布这个

'We are excited to present on our Huntingtin-lowering clinical trials at the HDYO International Congress this month,' said Sergey Paushkin, Chief Scientific Officer and Head of R&D at Skyhawk, 'with positive results including SKY-0515's average HTT mRNA reduction of 72% at a daily oral dose of 9mg in trials thus far, and the drug's predicted suppression of the PMS1 protein, SKY-0515, if approved, could make a meaningful difference in .

“我们很高兴能够在本月的HDYO国际大会上展示我们的亨廷顿降低临床试验结果，”Skyhawk公司的首席科学官兼研发主管谢尔盖·保什金表示，“目前试验取得了积极成果，包括SKY-0515在每日口服9毫克剂量下，HTT mRNA平均降低了72%，并且该药物预计能够抑制PMS1蛋白。如果获得批准，SKY-0515可能会带来重要的改变。”

Huntington's

亨廷顿氏舞蹈症

patients' lives.'

患者的生命。

Huntington's

亨廷顿氏舞蹈病

disease is a rare hereditary neurodegenerative disease affecting over 40,000 patients in

疾病是一种罕见的遗传性神经退行性疾病，影响着超过40,000名患者在

the United States

美国

and many more worldwide. There are no approved treatments that can reverse or slow its course of progression.

还有许多其他疾病。目前尚无可以逆转或减缓其进展的治疗方法。

'We are pleased with the speed at which we've conducted our studies thus far and are focused on this patient community in great need', said

“到目前为止，我们对研究进展的速度感到满意，并且我们专注于这个非常需要帮助的患者群体，”

Bill Haney

比尔·哈尼

, Chief Executive Officer at Skyhawk. 'SKY-0515's compelling results thus far demonstrate the immense potential of our platform to target indications for which there are no approved disease modifying therapies.'

Skyhawk首席执行官表示：“SKY-0515迄今为止的令人信服的结果表明了我们的平台在针对尚无获批疾病修饰疗法的适应症方面具有巨大的潜力。”

About Skyhawk Therapeutics

关于Skyhawk Therapeutics

Skyhawk Therapeutics is a clinical-stage biotechnology company focused on the discovery and development of novel small molecule therapies designed to modulate critical RNA targets and revolutionize patient treatment for some of the world's most intractable diseases. Skyhawk's discovery expertise is rooted in its proprietary drug discovery platform, which assesses, identifies, and tests RNA splicing targets and small molecules across a broad range of therapeutic areas and disease states.

Skyhawk Therapeutics是一家临床阶段的生物技术公司，专注于发现和开发新型小分子疗法，旨在调控关键的RNA靶点，并彻底改变一些世界上最难治疾病患者的治疗方式。Skyhawk的发现专长植根于其专有的药物发现平台，该平台评估、识别并测试跨广泛治疗领域和疾病状态的RNA剪接靶点和小分子。

Skyhawk has built collaborations with multiple pharma partners that leverage Skyhawk's novel platform across disease areas including neurodegenerative disease, autoimmune disease, and oncology. For more information visit .

Skyhawk 已与多家制药合作伙伴建立了合作，利用 Skyhawk 的新型平台跨越包括神经退行性疾病、自身免疫疾病和肿瘤学在内的疾病领域。欲了解更多信息，请访问。

www.skyhawktx.com

。

Skyhawk Contact

天鹰座联系

Maura McCarthy

毛拉·麦卡锡

maura@skyhawktx.com

毛拉@天鹰tx.com

SOURCE Skyhawk Therapeutics

来源：天鹰治疗学

WANT YOUR COMPANY'S NEWS