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World’s Premier Precision Medicine Conference (PMWC 2019) Opens 10th Annual Meeting in California’s Silicon Valley; Awards Given to Four Major Contributors to Advance of Precision Medicine

The Precision Medicine World Conference (PMWC 2019) opened its tenth annual meeting ("Celebrating 10 Years of Precision Medicine Innovation) Sunday evening, January 20, in Santa Clara, Caifornia, with an awards ceremony honoring four distinguished contributors to the advance of precision medicine. ( Live tweets from the conference can be accessed on Twitter using @JohRONeill1 or #pmwc19 ). The award recipients were Carl June, MD, PhD, (Director, Center for Cellular Immunotherapies, University of Pennsylvania) for developing CAR-T therapy (the world’s first gene-based cancer therapy); Feng Zhang, PhD, (Professor, Neuroscience, MIT/Broad) for spearheading the development of optogenetics and CRISPR; George Yancopoulos, MD, PhD, (President & CSO & Director, Regeneron Pharmaceuticals) for developing foundational technologies designed to invent groundbreaking therapies; and Sharon Terry (President, Genetic Alliance) for initiating the movement to build systems for individuals to access and share health data. The master of ceremonies for the awards event was Dawn Berry (Co-Founder & President, Luna DNA) and she opened the evening by welcoming everyone and then introduced event organize Tal Behar (Co-Founder & President, PMWC, LLC) to make some opening remarks. Behar first expressed her thanks the Program Co-Chairs Nancy Davidson, MD (Director Clinical Research Division, Fred Hutchinson Cancer Research Center), and India Hook-Barnard, PhD (Director Research Strategy, UCSF), and then introduced Program Chairman William Dalton, MD, PhD (former President, CEO & Center Director of the Moffitt Cancer Center) to kick off the awards ceremony. Dr. Dalton said that he believed that the conference is “an outstanding event,” and that he is immensely impressed by the breadth and depth of those who participate in the conference, and that at the end of presentations, he never had to ask if there were any questions. Ari Baras (VP & Head, Regeneron Genetics Center, then stepped to the podium to introduce the winner of this year’s Pioneer Award—Regeneron’s Dr. George Yancopoulos. Dr. Baras first described the PMWC as “a phenomenal event,” and then said that his friend and mentor Dr Yancopoulos, after 30 years of precision medicine efforts and prolific inventing, could be thought of as the “Mount Rushmore of Biotech.” He lauded the efforts that Dr. Yancopoulos had spearheaded to support education and said that he had “a one in a million mind, that he “dreams big,” and that he is “committed to the long game.” Then he said, “Let’s all dream big” and work with “passion to make a difference for patients.”

Dr. Yancoloupos then stepped forward to accept his award, noting that it was a little “embarrassing” and really represented the accomplishments of an entire company. He said that there is nothing harder than inventing something completely from scratch because the biology is so daunting, and yet Regeneron has done that seven different times. He said that early on they had “bet it all on genetics,” and that “DNA is the basis of life.” He said it takes an “incredible team and an enormous number of people to build and do all the work.” He closed by saying that after 30 years, what he is proudest of is that Regeneron is still at “the bleeding edge of innovation.” We are just at the start, he said, of getting important medicines to patients who really need them.

Dr. Yancopoulos has been the Chief Scientific Officer at Regeneron Pharmaceuticals, Inc., since January 1998 and its President since December 16, 2016.
Dr. Yancopoulos joined Dr. Schleifer in 1989 as founding scientist of Regeneron, and together they built and have managed the Company since then. In 2004, Dr. Yancopoulos was elected to be a member of the National Academy of Sciences. Dr. Yancopoulos, together with key members of his team, is a principal inventor and/or developer of the six FDA-approved drugs the Company has developed, EYLEA® (aflibercept) Injection, PRALUENT® (alirocumab) Injection, DUPIXENT® (dupilumab) Injection, KEVZARA® (sarilumab) Injection, ZALTRAP® (ziv-aflibercept) Injection for Intravenous Infusion, and ARCALYST® (riloacept) Injection for Subcutaneous Use, as well as of its foundation technologies, including the TRAP technology, VelociGene®, and VelocImmune®. As one of the few members of the National Academy of Sciences from industry and as an author of a substantial number of scientific publications, Dr. Yancopoulos has a distinguished record of scientific expertise. In 1997 survey by the Institute for Scientific Information, he was listed among the 11 most highly cited scientists and was the only non-academic scientist in that group. He received his PhD in Biochemistry and Molecular Biophysics and his MD from Columbia University.

LUMINARY AWARD WINNER CARL JUNE

Next, pediatric oncologist Ilan Kirsch, MD (Senior VP, Translational Medicine, Adaptive Technologies) introduced Luminary Award winner Dr. Carl June. Dr. Kirsch described Dr. June as a brilliant immunologist with fundamental insights, who sought to make a realty of adaptive cell transfer for the treatment of lymphoid malignancies. He said that Dr. June’s work led, on August 30, 2017, to the first FDA approval of CAR T cell therapy for relapsed lymphoblastic leukemia. He then noted the moving story of how Dr. June had been included among Time’s 2018 list of the world’s top 100 most influential people. The publication included an article written Emily Whitehead, who, as a 12-year-old girl with relapsed ALL, received the first CAR T cell therapy. In the Time article, Emily Whitehead thanked Dr. June for saving her life (http://time.com/collection/most-influential-people-2018/5238121/carl-june).

Dr. June then came forward to accept his award and make a few remarks. He said that Churchill had once said, “all dogs look up to us, all cats look down on us, only pigs look us straight in the eye.” Now, he said, with the Nobel Prize going to Jim Allison, we can look pigs straight in the eye. CAR T cells are “the first living drug,” he said, and he saluted the brilliant work of a colleague who led the efforts to move the revolutionary therapy through clinical trials, culminating in the first-ever FDA approval of a gene therapy for patients. Now, Dr. June said the goal is reduce the cost of goods, and to bring the power of the therapy to bear on solid tumors, in addition to blood cancers. He also emphasized the importance of collaboration and the use of orthogonal tools.

Dr. June is a pioneer in precision medicine and immunotherapy of human immunodeficiency virus (HIV) and cancer. He was the driving force behind the first FDA-approved gene therapy – Kymriah, chimeric antigen receptor (CAR) T cells to treat B-cell acute lymphoblastic leukemia.

Dr. June studies mechanisms of lymphocyte activation that relate to immune tolerance and adoptive immunotherapy for cancer and chronic infection. In 2011, his research team published findings detailing a new therapy in which patients with refractory and relapsed chronic lymphocytic leukemia were treated with genetically engineered versions of their own T cells, CAR-Ts.

The treatment has also now been used with promising results to treat children with refractory acute lymphoblastic leukemia. Dr. June’s work led to the development and commercialization of tisagenlecleucel, the first FDA-approved gene therapy. In the 1980s, his lab discovered the CD28 molecule as the major control switch for T cells.

He has published more than 350 manuscripts and is the recipient of numerous prizes and honors, including election to the Institute of Medicine in 2012 and the American Academy of Arts and Sciences in 2014, the William B Coley award, the Richard V Smalley Memorial Award from the Society for Immunotherapy of Cancer, the AACR-CRI Lloyd J. Old Award in Cancer Immunology, the Philadelphia Award, the Taubman Prize for Excellence in Translational Medical Science, the Paul Ehrlich and Ludwig Darmstaedter Prize, the Novartis Prize in Immunology, the Karl Landsteiner Memorial award, the Debrecen Award and a lifetime achievement award from the Leukemia and Lymphoma Society. June received his Bachelor of Science in Biology from the United States Naval Academy and his MD from Baylor College of Medicine.

LUMINARY AWARD WINNER FENG ZHANG

Dr. Dalton returned to the podium to introduce Luminary Award winner Dr. Feng Zhang. He noted how young Dr. Zhang is to have already done so much. Dr. Zhang has both continuously improved and refined CRISPR-Cas9 gene editing technology, while also developing a light-based measuring approach called “optogenetics.” In 2010, Dr. Zhang’s combination of CRISPR and optogenetic was named “method of the year” by Nature Methods. Dr. Zhang had earned his PhD just a year earlier, in 2010. In 2011, he published a seminal paper on CRISPR in Science, and has followed that with another key CRISPR paper published in Science just last month.

Dr. Zhang stepped forward and first expressed his thanks for what he termed a “great honor.” He then expressed thanks to his entire research team, his collaborators, and to his mentors. He said that when he was much younger, he never thought he would go into biology. He didn’t enjoy having to memorize things and he really disliked having to dissect smelly, formaldehyde-preserved frogs. Certainly not for him, he thought.

But then came a transformative event. While in high school in Iowa, he took a volunteer position in a gene therapy lab. And that’s when everything changed. His mentor there, Dr. Jon Levy, was a passionate, inspirational scientist, and he convinced Zhang that biology could provide avenues to the understanding of disease.
This demonstrates, Dr, Zhang said, the power of teachers to change lives, and he moved from his profound gratitude to Dr. Levy, to thank all educators for what they do to change lives for the better and for good.

He moved on to note that CRISPR is the basis of an immune system in bacteria, and that it is by understanding how CRISPR worked in bacteria that we have been able to adapt it for gene editing purposes. This is an example of how useful the study of basic diversity can be in enabling rapid technology advances. He closed by noting the importance of making tools widely available and also of ensuring that the tools are used responsibly and in ethical ways.

Dr. Zhang’s lab at MIT is focused on using synthetic biology to develop technologies for genome and epigenome engineering to study neurobiology. Dr. Zhang played a central role in the development of both CRISPR technology and optogenetics, a biological technique that uses light to control cells in living tissue, usually neurons. Zhang’s group optimized the Cas9 system in human cells starting in 2011. They then compared their RNA expression approach with a design based on the Doudna / Charpentier chimeric RNA for use in human cells and established features of the guide necessary for Cas9 to function effectively in mammalian cells.

His lab’s diagnostic nucleic acid detection protocol based on CRISPR, called SHERLOCK (Specific High sensitivity Enzymatic Reporter UnLOCKing) is able to detect and distinguish strains of viruses and bacteria present in as low as attomolar (10−18 M) concentration.

Dr. Zhang is a recipient of the NIH Director’s Pioneer Award and is a 2012 Searle Scholar. He was named one of MIT Technology Review’s 35 most innovative people in the world under age 35, in 2013. His work on optogenetics and CRISPR was recognized by many awards, including the 2011 Perl-UNC Prize, the 2014 Alan T. Waterman Award, the National Science Foundation’s highest honor that annually recognizes an outstanding researcher under the age of 35; and the 2014 Gabbay Award, the 2014 Young Investigator Award from the Society for Neuroscience. In 2018, Zhang was elected as a Fellow of the American Academy of Arts and Sciences, and a member of the National Academy of Sciences.

Zhang received an AB in Chemistry and Physics from Harvard University in 2004 and his PhD in chemical and biological engineering from Stanford University in 2009.

LUMINARY AWARD WINNER SHARON TERRY

Randy Scott, PhD (Chairman & CEO, Invitae Corporation) then came to the podium to introduce Luminary Award winner Sharon Terry. He began by noting what an extraordinary story Sharon Terry’s is. She had actually been studying theology, and her husband Patrick was in the construction business, when life threw them a cruel curveball. Both their young children were diagnosed with the genetic disease PXE (pseudoxanthoma elasticum), which can cause blindness. Sharon very quickly took decisive and positive action by forming the PXE Foundation to empower research into the disease and to speed the development of possible treatments or cures. Such was her involvement that she helped in the eventual cloning of the disease gene and was a co-inventor on the patent for the gene. Sharon went on to found the Genetic Alliance in order to centralize efforts to help families with a wide range of rare diseases and she has been an author on 150 peer-reviewed papers. Dr. Scott added that he had attended a great number of meetings with Sharon at which everyone would introduce themselves to the group by stating their position and background. When it came time for Sharon, with all her herculean accomplishments, to introduce herself, she would say, “I’m Sharon Terry. I’m just a mom.”

Sharon came forward to accept her award and she said first that she was accepting it “on behalf of the people who suffer,” and she thanked Dr. Scott, noting that he had been one of the first in industry who had worked with them. She spoke of the “diagnostic odyssey” she had endured with her children and she emphasized the importance of “bringing people truly to the center.”

Sharon works to provide ordinary people the tools to take charge of their health and to further biomedical research through her leadership of Genetic Alliance. In 1994, she cofounded PXE International, a research advocacy organization for the genetic condition pseudoxanthoma elasticum (PXE), when her two children were diagnosed with the rare condition. In 2000, with her husband, Patrick, she co-discovered the ABCC6 gene and patented it to ensure its ethical stewardship. She subsequently developed a diagnostic test and conducts clinical trials.

Sharon is the author of 150 peer-reviewed papers. In her focus at the forefront of consumer participation in genetic research, services, and policy, she serves in leadership roles on many international and national organizations, including the Precision Medicine Initiative Cohort Advisory Panel; Accelerating Medicines Partnership; the Cures Acceleration Network Review Board; and the Advisory Council, National Center for Accelerating Translation Science, NIH, among many others.

She serves on the editorial boards and is active on several journals, including Genome, Genetic Testing, and Molecular Biomarkers, and Clinical and Translational Science. She led the coalition that was instrumental in the passage of the Genetic Information Nondiscrimination Act.

Among numerous awards and honors, she received the Research! America Distinguished Organization Advocacy Award and is an inaugural member of Disruptive Women in Health Care; in 2009 an Ashoka Fellowship and the Clinical Research Forum and Foundation’s Annual Award for Leadership in Public Advocacy.

Sharon was named one of FDA’s “30 Heroes for the Thirtieth Anniversary of the Orphan Drug Act” in 2013. She was a member of the Blue Ribbon Panel’s Working Group on Enhanced Data Sharing for the Cancer Moonshot. She was named a National Associate of the National Research Council, National Academies of Engineering, Sciences, and Medicine for her extraordinary service.

She received the Health 2.0 Health Activist award in 2016. In 2017, she co-founded the People Centered Research Foundation. She received her education at Assumption College and Stony Brook University and was awarded an Honorary Doctorate from Iona College.

After Sharon’s remarks, MC Dawn Berry closed this moving opening session of PMWC 2019.

CONFERENCE THEME, CO-HOSTS, & RECORD ATTENDANCE

This year’s conference theme is “How Do We Accelerate Precision Medicine and Deliver on Its Promises?” The conference co-hosts are Stanford Health Care/Stanford Medicine, UCSF, Johns Hopkins, Duke University School of Medicine, and the University of Michigan.

A record 1,800 attendees are expected to hear over 400 presentations from world leaders in the many different fields that are impacted by precision medicine.
The meeting runs from today (January 20) through Wednesday (January 23).

BioQuick News is proud to be a Media Partner of PMWC 2019.

by Michael D. O’Neill, Editor & Publisher, BioQuick News (logophile2000@yahoo.com)

TWITTER: Live tweets from the conference can be accessed using @JohRONeill1 or #pmwc19 ).

[PMWC 2019] [PMWC 2019 Awards]