Laureates of the King Faisal Prize in Medicine, Professor Jerry Mendell, and in Science, Professor Howard Chang, awarded

During its 46th session, the King Faisal Prize recognized other eminent figures in the field of Islamic studies and service to Islam

Professor Howard Chang, winner of the 2024 King Faisal Prize in Science

Professor Jerry Mendell, winner of the 2024 King Faisal Prize in Medicine

Riyadh, Saudi Arabia, April 22, 2024 (GLOBE NEWSWIRE) — The 46th session of the King Faisal Prize on April 22 recognized exceptional achievements in gene therapy for neuromuscular diseases and groundbreaking RNA discoveries. Professor Jerry Mendell received the Medicine Prize for his pioneering contributions to the screening, early diagnosis and treatment of neuromuscular disorders, in particular spinal muscular atrophy (SMA) And Duchenne muscular dystrophy (DMD). Professor Howard Chang received the Science Prize for revealing the intrinsic role of long non-coding RNAs in gene regulation and function, a previously overlooked area of ​​research.

Through innovative approaches to gene therapy, Professor Mendell, Director of the Gene Therapy Center at Nationwide Children’s Hospital and Curran Peters Chair in Pediatric Research, has revolutionized treatment strategies and provided hope to countless patients around the world.

Spinal muscular atrophy (SMA), once considered a major genetic cause of infant mortality, has seen a paradigm shift in treatment thanks to the pioneering efforts of Professor Mendell. By using gene therapy to deliver crucial genes to affected cells, he has significantly improved patient outcomes, leading to the first-ever FDA-approved gene therapy treatment for pediatric SMA patients in 2019 (Zolgensma®).

In addition to his work at SMA, Professor Mendell’s research has expanded into Duchenne muscular dystrophy and muscular dystrophy of the limbs, demonstrating the versatility and efficacy of gene therapy in addressing a spectrum of neuromuscular disorders. Genetic mutations in patients with Duchenne muscular dystrophy (DMD) hinder the production of dystrophin, a crucial protein for muscle health. While initial symptoms mainly affect skeletal muscle, DMD progresses to impact cardiac and respiratory functions. Gene therapy offers a solution by tackling this genetic defect, allowing the body to produce dystrophin and halt muscle degeneration. In June 2023, the US Food and Drug Administration approved the first treatment for pediatric DMD patients aged 4 to 5 years old, using a breakthrough gene therapy co-developed by Professor Mendell and Professor Louise Rodino-Klapac, a former postdoctoral researcher in Mendell’s laboratory. . This therapy involves a single injection, delivering a microdystrophin gene within an adeno-associated virus serotype (AAVrh74), facilitating the transfer of missing or corrected genes into cells.

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He has played an important role in clinical studies of various forms of Limb-Girdle Muscular Dystrophy (LGMD), characterized by muscle weakness in the shoulders, upper arms, hips and thighs. One study focused primarily on LGMD2B, showing that a single injection of a gene therapy vector effectively repaired damaged muscle fibers, alleviated degeneration and improved muscle function.

Professor Mendell has a portfolio of more than 400 published articles, a testament to his prolific contributions to the field. In recognition of his achievements, he was honored in 2021 with election to the National Academy of Medicine. The American Society of Gene and Cell Therapy awarded him the prestigious Translational Science Award named in his honor. Additionally, in 2017, Science Magazine recognized his groundbreaking work in SMA gene therapy with the esteemed Breakthrough Achievement Award. His honors also include numerous other prizes and invitations to deliver honorary lectures at esteemed institutions.

In his acceptance speech at the ceremony, Professor Mendell said: “The principles of the award, which reflect King Faisal’s intention to alleviate human suffering, are consistent with my own views and lifelong achievements. I did everything I could do improve the quality and prolong the lives of patients suffering from neuromuscular diseases.”

At the frontier of molecular biology, Professor Howard Chang, a physician-scientist and professor of dermatology and genetics, and the Virginia and DK Ludwig Professor of Cancer Research at Stanford University, have been awarded the King Faisal Prize for Science in Biology. Professor Chang’s elucidation of the role of long non-coding RNAs (lncRNAs) in gene regulation has revolutionized our understanding of cellular processes and disease mechanisms.

Through innovative genome-wide methodologies, Professor Chang has uncovered the intricate interplay between lncRNAs and gene expression, shedding light on fundamental biological processes underlying development, cancer and aging. Although they do not code for proteins, lncRNAs are crucial for controlling the timing and amount of protein production, and influence the overall function and behavior of cells.

A remarkable feat of organization unfolds within each human cell: 2 meters of DNA are densely packed into a nucleus just 10 microns in size. This tight packaging makes most of the DNA inaccessible, except for the active DNA elements that are crucial for cell function and reading. Identifying these accessible elements reveals valuable insights into the cellular ‘software’. His groundbreaking techniques are in the field of mapping chromatin – the substance that makes up chromosomes, consisting of DNA and associated proteins that determine genome structure and gene expression. This has provided unprecedented insights into the regulatory mechanisms that determine gene expression, paving the way for new therapeutic interventions and precision medicine approaches. One technique is the Assay of Transposase Accessible Chromatin, which uses the Tn5 transposase enzyme to efficiently copy and paste DNA. This revolutionary technique has delivered a million-fold improvement in sensitivity and a hundred-fold improvement in mapping speed for regulatory DNA – the epigenome – in human cells.

As founder of the RNA Medicine Program at Stanford University, Professor Chang continues to lead efforts to translate RNA science into tangible clinical applications, unlocking the promise of RNA-based therapies in the treatment of a wide range of diseases promoted. Prof. Chang’s awards include the NAS Award for Molecular Biology, Outstanding Investigator Award from the National Cancer Institute, Paul Marks Prize for Cancer Research, Judson Daland Prize from the American Philosophical Society, and the Vilcek Prize for Creative Promise. His work was honored by the journal Cell as a Landmark paper of the past 40 years and by Science as “Insight of the decade.”

In his acceptance speech at the ceremony, Professor Chang said: “Our research asked a fundamental question: how do cells decide when and where to turn on different genes? How are these decisions adopted over time? Our studies led us to a new class of RNAs, called long non-coding RNAs, that help cells remember their cell fate. Our understanding of the gene switches has led to an understanding of how inherited genetic differences cause diseases, especially immune diseases. This insight also helped tackle mutations that occur in cancer.”

In addition to honoring advances in medicine and science, the King Faisal Prize also recognized exemplary contributions in the field of Islamic studies and service to Islam. Professor Wael Hallaq, ranked among the top 500 scholars in the field of Islamic Studies worldwide, received the Islamic Studies Award for his groundbreaking contributions in guiding the development of Islamic law and challenging the narrative of the conclusion of the gate of ijtihad – the interpretation of Islamic texts – that gained fame. reforming accepted paradigms in the field.

The Japan Muslim Association and Mr. Mohammad El Sammak were honored for their exemplary leadership in serving Islam and humanity. The Japan Muslim Association is a pioneering Islamic organization for Japanese Muslims, known for its historic translation of the Holy Quran into Japanese and its advocacy of Muslim causes in Japan. And Mr. Mohammad El Sammak, a pioneer in dialogue and building bridges between different religions, has had a lasting impact on interfaith dialogue for fifty years. He was recognized for his effective contributions to conferences examining Islam’s relationship with other beliefs, and for his leadership in institutions dedicated to tolerance and peace.

The King Faisal Prize for Arabic Language and Literature for 2024 on the topic “Non-Arab Institutions and Their Efforts to Promote Arabic” was withheld because nominated works did not meet the criteria of the award.

Since 1979, the King Faisal Prize has awarded 295 laureates in five different categories who have made distinguished contributions to various sciences and causes. Each prize winner will receive a sum of $200,000; a 24-carat gold medal weighing 200 grams, and a certificate with the name of the laureate and a summary of their work that qualified them for the award.

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CONTACT: Maysa Shawwa King Faisal Foundation [email protected]