Vertex Researcher, Paul Negulescu Ph.D., Receives the 2025 Canada Gairdner International Award for Pioneering Research and Discovery of Medicines for Cystic Fibrosis
Vertex Pharmaceuticals (Nasdaq: VRTX) announced that Paul Negulescu, Ph.D., Senior Vice President, has been awarded the prestigious 2025 Canada Gairdner International Award for his groundbreaking research in cystic fibrosis (CF). The award recognizes his pioneering work in understanding CF's cellular and molecular mechanisms, leading to transformative drug therapies.
Under Negulescu's leadership, Vertex developed the first-ever protein folding corrector medicines that treat CF's underlying cause. The company's most recent achievement is a triple combination therapy that can treat over 90% of CF patients with more than 300 different mutations through once-daily dosing.
Vertex currently has five approved CF medicines that restore function of the defective CFTR protein. The research team continues to develop new therapies, including an mRNA approach in clinical development, targeting the remaining 10% of patients who don't respond to existing treatments.
Vertex Pharmaceuticals (Nasdaq: VRTX) ha annunciato che Paul Negulescu, Ph.D., Vicepresidente Senior, è stato premiato con il prestigioso Premio Internazionale Canada Gairdner 2025 per la sua ricerca innovativa sulla fibrosi cistica (FC). Questo riconoscimento evidenzia il suo lavoro pionieristico nella comprensione dei meccanismi cellulari e molecolari della FC, che ha portato a terapie farmacologiche trasformative.
Sotto la guida di Negulescu, Vertex ha sviluppato i primi medicinali correttori del ripiegamento proteico che trattano la causa sottostante della FC. Il recente traguardo dell'azienda è una terapia combinata tripla che può trattare oltre il 90% dei pazienti con FC con più di 300 diverse mutazioni attraverso una somministrazione giornaliera.
Attualmente, Vertex ha cinque medicinali approvati per la FC che ripristinano la funzione della proteina CFTR difettosa. Il team di ricerca continua a sviluppare nuove terapie, inclusa un'approccio mRNA in fase di sviluppo clinico, mirato al restante 10% dei pazienti che non rispondono ai trattamenti esistenti.
Vertex Pharmaceuticals (Nasdaq: VRTX) anunció que Paul Negulescu, Ph.D., Vicepresidente Senior, ha sido galardonado con el prestigioso Premio Internacional Canadá Gairdner 2025 por su investigación innovadora en fibrosis quística (FQ). El premio reconoce su trabajo pionero en la comprensión de los mecanismos celulares y moleculares de la FQ, lo que ha llevado a terapias farmacológicas transformadoras.
Bajo el liderazgo de Negulescu, Vertex desarrolló los primeros medicamentos correctores de plegamiento proteico que tratan la causa subyacente de la FQ. El logro más reciente de la empresa es una terapia combinada triple que puede tratar a más del 90% de los pacientes con FQ con más de 300 mutaciones diferentes mediante una dosis diaria.
Actualmente, Vertex tiene cinco medicamentos aprobados para la FQ que restauran la función de la proteína CFTR defectuosa. El equipo de investigación continúa desarrollando nuevas terapias, incluyendo un enfoque de ARNm en desarrollo clínico, dirigido al 10% restante de los pacientes que no responden a los tratamientos existentes.
Vertex Pharmaceuticals (Nasdaq: VRTX)는 Paul Negulescu, Ph.D., 수석 부사장이 섬유 낭종 (CF)에 대한 획기적인 연구로 2025 캐나다 가이던상을 수상했다고 발표했습니다. 이 상은 CF의 세포 및 분자 메커니즘을 이해하는 그의 선구적인 작업을 인정하며, 이는 변혁적인 약물 치료로 이어졌습니다.
Negulescu의 지도 아래, Vertex는 CF의 근본 원인을 치료하는 최초의 단백질 접힘 교정 약물을 개발했습니다. 회사의 최근 성과는 하루 한 번 복용으로 300개 이상의 다양한 돌연변이를 가진 90% 이상의 CF 환자를 치료할 수 있는 삼중 조합 요법입니다.
Vertex는 현재 결함이 있는 CFTR 단백질의 기능을 복원하는 5개의 승인된 CF 약물을 보유하고 있습니다. 연구팀은 기존 치료에 반응하지 않는 나머지 10%의 환자를 목표로 하는 임상 개발 중인 mRNA 접근 방식을 포함하여 새로운 치료법을 계속 개발하고 있습니다.
Vertex Pharmaceuticals (Nasdaq: VRTX) a annoncé que Paul Negulescu, Ph.D., Vice-président senior, a été récompensé par le prestigieux Prix International Canada Gairdner 2025 pour ses recherches novatrices sur la fibrose kystique (FK). Ce prix reconnaît son travail pionnier dans la compréhension des mécanismes cellulaires et moléculaires de la FK, ce qui a conduit à des thérapies médicamenteuses transformantes.
Sous la direction de Negulescu, Vertex a développé les premiers médicaments correcteurs de repliement des protéines qui traitent la cause sous-jacente de la FK. Le dernier succès de l'entreprise est une thérapie combinée triple qui peut traiter plus de 90% des patients atteints de FK avec plus de 300 mutations différentes grâce à une posologie quotidienne.
Vertex dispose actuellement de cinq médicaments approuvés pour la FK qui restaurent la fonction de la protéine CFTR défectueuse. L'équipe de recherche continue de développer de nouvelles thérapies, y compris une approche par ARNm en développement clinique, visant les 10% restants de patients qui ne répondent pas aux traitements existants.
Vertex Pharmaceuticals (Nasdaq: VRTX) gab bekannt, dass Paul Negulescu, Ph.D., Senior Vice President, mit dem renommierten 2025 Canada Gairdner International Award für seine bahnbrechende Forschung zur Mukoviszidose (CF) ausgezeichnet wurde. Der Preis würdigt seine Pionierarbeit zum Verständnis der zellulären und molekularen Mechanismen von CF, die zu transformierenden Medikamententherapien geführt hat.
Unter Negulescus Leitung entwickelte Vertex die ersten jemals zugelassenen Medikamente zur Korrektur des Protein-Faltens, die die zugrunde liegende Ursache von CF behandeln. Der jüngste Erfolg des Unternehmens ist eine dreifache Kombinationstherapie, die über 90% der CF-Patienten mit mehr als 300 verschiedenen Mutationen durch eine tägliche Dosis behandeln kann.
Vertex hat derzeit fünf zugelassene CF-Medikamente, die die Funktion des defekten CFTR-Proteins wiederherstellen. Das Forschungsteam entwickelt weiterhin neue Therapien, einschließlich eines mRNA-Ansatzes in der klinischen Entwicklung, der auf die verbleibenden 10% der Patienten abzielt, die nicht auf bestehende Behandlungen ansprechen.
- Development of revolutionary CF treatments that address root cause rather than just symptoms
- Current therapies can treat over 90% of CF patients with 300+ mutations
- Portfolio of 5 approved CF medicines
- Ongoing development of mRNA therapy for remaining 10% of patients
- 10% of CF patients still cannot be treated with current therapies
“For more than 20 years, Paul and the team of dedicated Vertex 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 David Altshuler, M.D., Ph.D., Executive Vice President, Global Research, and Chief Scientific Officer at Vertex.
“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 Vertex, 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.
The gene responsible for CF was discovered in 1989, but until the work of Vertex 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
Through the Canada Gairdner International Award, the Gairdner Foundation, 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. The Canada Gairdner International Award is one of the most preeminent science awards in
About Cystic Fibrosis
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 CFTR gene. Children must inherit two defective 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 CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. 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. The median age of death is in the 30s, but with treatment, projected survival is improving.
Today Vertex 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.
About Vertex
Vertex 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. Vertex 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.
Vertex was founded in 1989 and has its global headquarters in
(VRTX-GEN)
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FAQ
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