StemCell Therapy

The 2020 award celebrates outstanding research in rare diseases

NEW YORK & OSAKA, Japan--(BUSINESS WIRE)-- Takeda Pharmaceutical Company Limited (Takeda) (TSE:4502/NYSE:TAK) and the New York Academy of Sciences announced today the Winners of the third annual Innovators in Science Award for their excellence in and commitment to innovative science that has significantly advanced the field of rare disease research. Each Winner receives a prize of US $200,000.

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The 2020 Winner of the Senior Scientist Award is Adrian R. Krainer, Ph.D., St. Giles Foundation Professor at Cold Spring Harbor Laboratory. Prof. Krainer is recognized for his outstanding research on the mechanisms and control of RNA splicing, a step in the normal process by which genetic information in DNA is converted into proteins. Prof. Krainer studies splicing defects in patients with spinal muscular atrophy (SMA), a devastating, inherited pediatric neuromuscular disorder caused by loss of motor neurons, resulting in progressive muscle atrophy and eventually, death. Prof. Krainers work culminated notably in the development of the first drug to be approved by global regulatory bodies that can delay and even prevent the onset of an inherited neurodegenerative disorder.

Collectively, rare diseases affect millions of families worldwide, who urgently need and deserve our help. Im extremely honored to receive this recognition for research that my lab and our collaborators carried out to develop the first approved medicine for SMA, said Prof. Krainer. As basic researchers, we are driven by curiosity and get to experience the thrill of discovery; but when the fruits of our research can actually improve patients lives, everything else pales in comparison.

The 2020 Winner of the Early-Career Scientist Award is Jeong Ho Lee, M.D., Ph.D, Associate Professor, Korea Advanced Institute of Science and Technology (KAIST). Prof. Lee is recognized for his research investigating genetic mutations in stem cells in the brain that result in rare developmental brain disorders. He was the first to identify the causes of intractable epilepsies and has identified the genes responsible for several developmental brain disorders, including focal cortical dysplasias, Joubert syndromea disorder characterized by an underdevelopment of the brainstemand hemimegalencephaly, which is the abnormal enlargement of one side of the brain. Prof. Lee also is the Director of the National Creative Research Initiative Center for Brain Somatic Mutations, and Co-founder and Chief Technology Officer of SoVarGen, a biopharmaceutical company aiming to discover novel therapeutics and diagnosis for intractable central nervous system (CNS) diseases caused by low-level somatic mutation.

It is a great honor to be recognized by a jury of such globally respected scientists whom I greatly admire, said Prof. Lee. More importantly, this award validates research into brain somatic mutations as an important area of exploration to help patients suffering from devastating and untreatable neurological disorders.

The 2020 Winners will be honored at the virtual Innovators in Science Award Ceremony and Symposium in October 2020. This event provides an opportunity to engage with leading researchers, clinicians and prominent industry stakeholders from around the world about the latest breakthroughs in the scientific understanding and clinical treatment of genetic, nervous system, metabolic, autoimmune and cardiovascular rare diseases.

At Takeda, patients are our North Star and those with rare diseases are often underserved when it comes to the discovery and development of transformative medicines, said Andrew Plump, M.D., Ph.D., President, Research & Development at Takeda. Insights from the ground-breaking research of scientists like Prof. Krainer and Prof. Lee can lead to pioneering approaches and the development of novel medicines that have the potential to change patients lives. Thats why we are proud to join with the New York Academy of Sciences to broadly share and champion their workand hopefully propel this promising science forward.

Connecting science with the world to help address some of societys most pressing challenges is central to our mission, said Nicholas Dirks, Ph.D., President and CEO, the New York Academy of Sciences. In this third year of the Innovators in Science Award we are privileged to recognize two scientific leaders working to unlock the power of the genome to bring innovations that address the urgent needs of patients worldwide affected by rare diseases.

About the Innovators in Science Award

The Innovators in Science Award grants two prizes of US $200,000 each year: one to an Early-Career Scientist and the other to a well-established Senior Scientist who have distinguished themselves for the creative thinking and impact of their research. The Innovators in Science Award is a limited submission competition in which research universities, academic institutions, government or non-profit institutions, or equivalent from around the globe with a well-established record of scientific excellence are invited to nominate their most promising Early-Career Scientists and their most outstanding Senior Scientists working in one of four selected therapeutic fields of neuroscience, gastroenterology, oncology, and regenerative medicine. Prize Winners are determined by a panel of judges, independently selected by the New York Academy of Sciences, with expertise in these disciplines. The New York Academy of Sciences administers the Award in partnership with Takeda.

For more information please visit the Innovators in Science Award website.

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries. For more information, visit https://www.takeda.com.

About the New York Academy of Sciences

The New York Academy of Sciences is an independent, not-for-profit organization that since 1817 has been committed to advancing science, technology, and society worldwide. With more than 20,000 members in 100 countries around the world, the Academy is creating a global community of science for the benefit of humanity. The Academy's core mission is to advance scientific knowledge, positively impact the major global challenges of society with science-based solutions and increase the number of scientifically informed individuals in society at large. Please visit us online at http://www.nyas.org.

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