顶点研究员保罗·内古列斯库博士因在囊性纤维化药物的开创性研究和发现方面荣获2025年加拿大盖尔德纳国际奖-动脉网

Vertex Researcher, Paul Negulescu Ph.D., Receives the 2025 Canada Gairdner International Award for Pioneering Research and Discovery of Medicines for Cystic Fibrosis

福泰制药等信源发布 2025-04-11 17:35

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BOSTON

波士顿

--(BUSINESS WIRE)--Apr. 11, 2025--

--（商业资讯）--2025年4月11日--

Vertex Pharmaceuticals Incorporated

顶点制药公司

(Nasdaq: VRTX) today announced that

(Nasdaq: VRTX) 今天宣布

Paul Negulescu

保罗·内古莱斯库

, Ph.D. Senior Vice President,

，博士，高级副总裁，

Vertex

顶点

has been awarded the 2025

已被授予2025年

Canada Gairdner International

加拿大盖尔德纳国际奖

Award “for pioneering research into the cellular and molecular mechanisms underlying the genetic disease cystic fibrosis, leading to the development of transformative drug therapies based on these mechanisms, thereby improving and saving countless lives.” Negulescu shares the award with

因“对遗传性疾病囊性纤维化的细胞和分子机制进行开创性研究，促成基于这些机制的变革性药物疗法的开发，从而改善并拯救了无数生命”而获奖。内古列斯库与

Michael J. Welsh

迈克尔·J·威尔士

, M.D.,

，医学博士，

University of Iowa

爱荷华大学

。

“For more than 20 years, Paul and the team of dedicated

“二十多年来，保罗和一支专注的团队”

Vertex

顶点

researchers have focused on discovering and developing breakthrough therapies for people living with cystic fibrosis (CF). Paul’s outstanding vision, leadership, determination and collaborative nature has resulted in what was once thought impossible — the discovery of the first-ever protein folding corrector medicines that treat the underlying cause of CF and have forever transformed the course of this disease,” said .

研究人员专注于为囊性纤维化（CF）患者发现和开发突破性疗法。保罗卓越的远见、领导力、决心和协作精神促成了曾经被认为不可能的事情 —— 发现了首个蛋白质折叠矫正药物，这些药物针对囊性纤维化的根本原因进行治疗，并永久改变了这种疾病的进程。”

David Altshuler

大卫·阿尔特舒勒

, M.D., Ph.D., Executive Vice President,

医学博士，哲学博士，执行副总裁，

Global Research

全球研究

, and Chief Scientific Officer at

，以及首席科学官在

Vertex

顶点

。

“I am honored and humbled by this award that recognizes not only me, but also the dedication, creativity and scientific excellence of the thousands of people across

“我因这个奖项而感到荣幸和谦卑，它不仅认可了我，也认可了成千上万名人员的奉献、创造力和科学卓越性，他们遍布于

Vertex

顶点

, and in the CF community, who have been steadfast in their commitment to bringing these medicines to people with cystic fibrosis around the world,” said Negulescu.

“，以及囊性纤维化社区，他们一直坚定地致力于将这些药物带给世界各地的囊性纤维化患者，”内古莱斯库说。

The gene responsible for CF was discovered in 1989, but until the work of

1989年发现了导致囊性纤维化的基因，但直到

Vertex

顶点

scientists led by Negulescu, treatments for CF addressed only its symptoms rather than the underlying cause of disease. Today, Vertex’s five approved medicines treat CF by restoring function of the defective CFTR protein. The most recently approved medicine is a triple combination therapy that has the potential to treat more than 90% of people with CF with any of more than 300 different mutations, with once-daily dosing.

在内古莱斯库的带领下，囊性纤维化（CF）的治疗仅针对其症状，而非疾病的根本原因。如今，Vertex公司获批的五种药物通过恢复有缺陷的CFTR蛋白功能来治疗CF。最新获批的药物是一种三联组合疗法，有望通过每日一次的剂量，治疗超过90%的CF患者，这些患者携带300多种不同突变中的任何一种。

The research team continues its relentless efforts to discover and develop even more effective therapies as well as novel approaches for the approximately 10% of people who are not expected to respond to existing therapies, such as a messenger ribonucleic acid (mRNA) approach currently in clinical development..

研究团队继续不懈努力，发现和开发更有效的疗法以及全新方法，以惠及预计约 10% 对现有疗法无反应的患者，例如目前正处于临床开发阶段的信使核糖核酸 (mRNA) 方法。

Through the

通过

Canada Gairdner International

加拿大盖尔德纳国际奖

Award, the

奖项，这个

Gairdner Foundation

盖尔德纳基金会

, celebrates the world’s most creative and accomplished researchers whose unique scientific contributions have increased the understanding of human biology and disease, and improved the health and wellbeing of people around the world.

，表彰世界上最具创意和成就的研究人员，他们独特的科学贡献增进了对人类生物学和疾病的理解，并改善了世界各地人民的健康和福祉。

The Canada Gairdner International

加拿大盖尔德纳国际奖

Award is one of the most preeminent science awards in

奖项是最重要的科学奖项之一

Canada

加拿大

and is widely recognized as one of the top scientific prizes in the world.

并被广泛认为是世界上最高的科学奖之一。

About Cystic Fibrosis

关于囊性纤维化

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 94,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the .

囊性纤维化 (CF) 是一种罕见的、缩短寿命的遗传性疾病，全球有超过 94,000 人受到影响。CF 是一种进行性的多器官疾病，会影响肺、肝、胰腺、胃肠道、鼻窦、汗腺和生殖道。CF 是由某些突变导致的 CFTR 蛋白缺陷和/或缺失引起的。

CFTR

gene. Children must inherit two defective

基因。儿童必须继承两个有缺陷的

CFTR

genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of

基因——每个父母各一个——患有囊性纤维化，这些基因突变可以通过基因测试来识别。虽然有许多不同类型的

CFTR

囊性纤维化跨膜电导调节因子

mutations that can cause the disease, the vast majority of people with CF have at least one

可能导致这种疾病的突变，绝大多数CF患者至少有一个

F508del

mutation.

突变。

CFTR

mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients.

突变通过导致CFTR蛋白缺陷或引起细胞表面CFTR蛋白的短缺或缺失而引发囊性纤维化。CFTR蛋白的功能缺陷和/或缺失会导致多个器官中盐和水的流动不畅。在肺部，这会引起异常粘稠、粘性的黏液积聚，导致慢性肺部感染和进行性肺损伤，最终导致许多患者死亡。

The median age of death is in the 30s, but with treatment, projected survival is improving..

死亡的中位年龄在30多岁，但通过治疗，预计生存率正在提高。

Today

今天

Vertex

顶点

CF medicines are treating over 68,000 people with CF across 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy.

囊性纤维化药物正在六大洲的60个国家治疗超过68,000名囊性纤维化患者。这占符合CFTR调节剂治疗条件的已确诊患者的三分之二。

About

关于

Vertex

顶点

Vertex

顶点

is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas.

是一家全球生物技术公司，致力于投资科学创新，为患有严重疾病和病症的人群开发变革性药物。该公司已获批的疗法涵盖囊性纤维化、镰状细胞病、输血依赖型β-地中海贫血和急性疼痛，并继续在这些领域推进临床和研究项目。

。

Vertex

顶点

also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1..

此外，还拥有一系列针对其他严重疾病的强大临床研究疗法管道，这些疾病包括神经性疼痛、APOL1介导的肾病、IgA肾病、原发性膜性肾病、常染色体显性多囊肾病、1型糖尿病和1型肌强直性营养不良，对于这些疾病，公司对因果人类生物学有深入的理解。

Vertex

顶点

was founded in 1989 and has its global headquarters in

成立于1989年，其全球总部位于

Boston

波士顿

, with international headquarters in

，国际总部位于

London

伦敦

. Additionally, the company has research and development sites and commercial offices in