Nature Medicine: Dr. Nabil G. Seidah's Work at the Heart of One of the Eleven Pivotal Clinical Studies of 2024

Nature Medicine: Dr. Nabil G. Seidah's Work at the Heart of One of the Eleven Pivotal Clinical Studies of 2024

The Montreal Clinical Research Institute (IRCM) is pleased to announce that the work of its leading researcher Dr. Nabil G. Seidah, is playing a key role in one of the eleven clinical studies that will shape medicine in 2024, according to the highly respected journal Nature Medicine.

In an effort to identify the clinical studies with the greatest therapeutic potential for 2024, Nature Medicine asked leading researchers to name the best clinical trials for 2024, and the most likely to bring concrete medical advances. Among the nominated clinical trials is research directly stemming from Dr. Nabil G. Seidah's major discovery of the role of the proprotein convertase PCSK9. They selection was made by Amit Khera, Vice President of Genomic Medicine at Verve Therapeutics, cardiologist at Brigham and Women's Hospital and lecturer at Harvard Medical School.

“ If there were still any doubts concerning the importance of Nabil Seidah's work on modern medicine, and its positive impact on human health, to be part of this prestigious list is yet another proof," enthused Dr. Jean-François Côté, President and Scientific Director of the IRCM.

At the heart of this exceptional work
Approximately one person out of 300 is born with heterozygous familial hypercholesterolemia. Heterozygous familial hypercholesterolemia is one of the most common inherited genetic disorders. The disease is caused by mutations in the three genes LDLR, APOB or PCSK9. The latter codes for a PCSK9 protein that leads to the degradation of low-density lipoprotein (LDL) receptors, a type of lipoprotein found in the blood. Although statins can reduce the risk of cardiovascular disease in these patients, most fail to achieve optimal LDL cholesterol levels with chronic treatment.

The heart-1 trial, the first of its kind in the world in terms of approach and technique, has the potential to demonstrate the proof-of-concept of PCSK9-targeted base-editing therapeutic approaches for sustainably lowering LDL cholesterol.

About Dr. Nabil G. Seidah
Dr. Seidah is known worldwide as the scientist responsible for the discovery and cloning of seven of the nine known secretory serine proteases belonging to the proprotein convertases. Proprotein convertases are essential for the regulation of the neural and endocrine systems, and for the proper functioning of many peripheral organs, including the liver, the pancreas, the pituitary gland and the heart, as well as the cardiometabolic and immune systems. Proprotein convertases have been identified as therapeutic targets for viral infections, inflammation, hypercholesterolemia and cancer. Seidah’s work, which has already resulted in groundbreaking cholesterol drugs, therefore holds infinite potential for the future development of innovative therapies.

Recently, the Université de Montréal highlighted the fact that Nabil G.Seidah was  among the 2023 list of the world's most cited scientists, according to Clarivate Analytics' Web of Science ranking.

With more than 820 peer-reviewed manuscripts, Dr. Seidah is the most recognized protease expert in Canada, and sixth worldwide. As the genetic and biopharmaceutical research fields continue to develop , Dr. Seidah's discoveries will translate into improved therapies and personalized preventive treatments for age-related diseases.

Through his career spanning almost 50 years, Dr. Seidah has received numerous awards and distinctions. He has been a member of the Ordre du Québec since 1997, and of the Order of Canada since 1999. He has held a Tier 1 Chair in Precursor Proteolysis since 2003, and is the recipient of the prestigious 2018 Lefoulon DeLalande (Institut de France) and Akira Endo awards, for his research and innovations in cardiovascular disease, as well as the 2021 Kuwait Prize for Applied Medical Sciences, among others. In 2023 he received the J. Allyn Taylor Award for his studies leading to revolutionary biotherapies in cardiovascular diseases.

Dr. Seidah is Full Professor at the IRCM, Director of the Biochemical Neuroendocrinology Research Unit at the IRCM's Centre de santé cardiométabolique, and Full Professor in the Department of Medicine at the Université de Montréal (UdeM).

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