Why 12 wise men?
On November 10th, 1919 the headline "Lights All Askew in the Heavens"was published in the New York Times announcing the triumph of Einstein's theory of Relativity.
Einstein's theories shocked the world due to the counterintuitive nature of their results, including the dissolution of absolute time.
Einstein believed his theory could only be understood by the brightest of his time. In the article he mentioned only the 12 Wise Men could comprehend it.
Today, humankind lives a similar scientific breakthrough moment, one that will change the way medicine addresses genetic disease.
Onpattro (Patisiran) is the first pharmaceutical of its kind to be approved at a global level. RNAi's impact on global health is yet at an early stage. Only a FEW can fully grasp its reach.
About RNAi
OUR SPEAKERS
Dr. Phillip A Sharp is an American molecular biologist born in June 1944. In 1969 Sharp received a doctorate in chemistry from the University of Illinois. Dr. Sharp is a world leader of research in molecular biology and biochemistry. Among the numerous grants in his career, Dr. Sharp was awarded the 1993 Nobel Prize for Physiology or Medicine, along with Richard J. Roberts, for his independent discovery that individual genes are often interrupted by long sections of DNA that do not encode protein structure. Dr. Phillip Sharp co-founded Alnylam in 2002 and is currently a member of its Scientific Advisory Board. He is also an Institute Professor and a Professor of Biology at the Massachusetts Institute of Technology and a member of Koch Institute for Integrative Cancer Research.
Dr. Francisco Nunes is the Country Manager for Alnylam Pharmaceuticals in Portugal. Dr. Nunes received his degree in Pharmaceutical Sciences from Universidade de Coimbra and has over 15 years of experience in the pharmaceutical and biotechnology industries.
Dr. John Maraganore is an American biochemist and molecular biologist born in 1962. He received his doctorate in Biochemistry and Molecular Biology from the University of Chicago, which he completed in one year, it was the youngest, fastest Ph.D. at the time. Dr. Maraganore has served as CEO and a Director of Alnylam since its foundation in 2002. He is a member of the Board for Agios Pharmaceuticals and Biotechnology Industry Organization (B10) Board and is a member of the BIO Executive Committee.
Dr. Teresa Coelho is a Portuguese neurologist and coordinator of Unidade Corino de Andrade in Centro Hospitalar do Porto. She is one of the top specialists in hATTR Amyloidosis in Portugal.
EVENT PROGRAM
Time GMT
Subject
Speaker
05:00pm - 05:05pm
Welcome
Francisco Nunes
(Alnylam Country Manager)
05:05pm - 05:20pm
Resilience to make history - the Alnylam case study
John Marganore (Alnylam CEO)
05:20pm - 05:45pm
RNA interference: past, present, and future
Phill Sharp (Nobel Laureate)
05:45pm - 06:00pm
Onpattro: a successful tale of RNAI translation
Teresa Coelho (Porto Neurology Unit Director)
06:00pm - 06:45pm
Structured Discussion
12 Wise Men
06:45pm - 06:50pm
Close
Francisco Nunes
(Alnylam Country Manager)
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ALNYLAM
Alnylam is a top tier, independent, biopharmaceutical company. It was founded in 2002 by a group of distinguished scientific leaders. Alnylam has led the translation of RNAi into an entirely new class of medicines, a major scientific breakthrough built on Nobel Prize winning research. Its pioneering work has delivered the first RNAi pharmaceutical to be approved in the world.
The Vision of Alnylam is to realize the potential of RNAi therapy in transforming the lives of people living with diseases for which there are limited or inadequate treatment options.
hATTR Amyloidosis
Hereditary ATTR (hATTR) Amyloidosis was first described in Portugal by Professor Corino de Andrade in the late 1930s. It is a rare inherited disease that affects approximately 50.000 people worldwide. In its cause is a genetic mutation that results in the misfolding of transthyretin (TTR) proteins. This results in the formation of amyloid fibrils that could deposit in the nerves, heart, and/or gastrointestinal (GI)
tract.
Symptoms can vary from person to person, depending on the organs or tissues affected. They can worsen as the disease progresses. Symptoms may include: Loss of sensation, limb weakness and pain; Alternating episodes of diarrhea, constipation, nausea, and vomiting Heart-related issues.
Hereditary ATTR (hATTR) Amyloidosis is a rapidly progressive, debilitating, and life-threatening disease. It has great impact on patients' quality of life, making them depend on others to perform usual activities.
RNAi
The discovery of RNA Interference (RNAi) has been heralded as a major breakthrough. Its importance was recognized in 2006 with the research being awarded the Nobel Prize in Physiology or Medicine.
RNAi is a natural process of gene silencing that regulates gene expression. It enables the silencing of any gene by targeting messenger RNA (mRNA) fir degradation, preventing the expression of proteins and their function in disease. It is a pioneering approach to treating illness with the ability to target virtually any protein.
MED-PT-TTR02-2000015