DMD: Charting the Path for New Therapeutics and Better Care

EVENT RECAP
September 9, 2021
Events

Dear colleagues and friends,

Thank you for participating in our global webinar, “Duchenne Muscular Dystrophy: Charting the Path for New Therapeutics and Better Care,” which was co-organized with Parent Project Muscular Dystrophy (PPMD) and CureDuchenne. We are delighted that more than 1500 registered from 36 countries worldwide, coming together to raise awareness of this devastating rare disease, and to foster collaborations towards better medicines for patients.

For those who registered but couldn’t make it, or who couldn’t stay through the entire webinar, we have prepared a replay link. Please click HERE to watch the on-demand video.

At WuXi AppTec, we firmly believe in a future where “every drug can be made and every disease can be treated”. That future is only possible if we all work together.

Thank you again, and we hope to see you at our next episode of WuXi AppTec Rare Disease webinar series.

AGENDA

8:00 AM

Welcome

Hui Cai, VP and Head of Content, WuXi AppTec

Pat Furlong, Founding President & CEO, Parent Project Muscular Dystrophy

Debra Miller, CEO & Founder, CureDuchenne

Filippo Buccella, Founder, Parent Project Italy APS

Huigu Chen, President, Shanghai Jiai Myopathy Care Center, China

Tali Kaplan, Co-Founder & CEO, Little Steps Association for Patients with Duchenne & Becker Muscular Dystrophy, Israel

Sherena Loh, Director, Muscular Dystrophy Association (Singapore)

8:15 AM

Clinical Research and Practices

Kevin Flanigan, Director, Center for Gene Therapy, Professor, Pediatrics & Neurology, Nationwide Children’s Hospital

Stanley Nelson, Professor of Human Genetics, Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine at UCLA

Richard Parad, Associate Professor of Pediatrics, Harvard Medical School, Department of Pediatric Newborn Medicine, Brigham and Women’s Hospital

Moderator: Hawken Miller, Features Writer, BioNews

8:45 AM

A Conversation between Pat Furlong, Founding President & CEO, Parent Project Muscular Dystrophy and Debra Miller, CEO & Founder, CureDuchenne

8:55 AM

Patient Story - The Nilson Family

9:00 AM

What can we do to bring medicines to DMD patients around the world faster?

Michael Binks, VP & Head of Clinical Research in the Rare Disease Research Unit at Pfizer

Paula Clemens, Professor & Vice Chair, Neurology, University of Pittsburgh School of Medicine

Nathalie Goemans, Professor, Neuromuscular Reference Centre for Children, Department of Pediatric Neurology, University Hospitals Leuven, Belgium

Moderator: Timothy Franson, Principal, Faegre Drinker Consulting and formerly VP Global Regulatory Affairs, Eli Lilly & Co and Chief Medical Officer of YourEncore

9:30 AM

New Targets. New Modalities. New Directions.

Rhonda Bassel-Duby, Professor, Department of Molecular Biology, University of Texas Southwestern Medical Center

Ashish Dugar, SVP & Global Head of Medical Affairs, Dyne Therapeutics

Jane Larkindale, VP, Clinical Sciences, PepGen

Art Levin, CSO, Avidity Biosciences

Stuart Peltz, Founder & CEO, PTC Therapeutics

Moderator: Richard Soll, Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec

10:20 AM

10-Year Vision

Michael Binks, VP & Head of Clinical Research in the Rare Disease Research Unit at Pfizer

Yi Dai, Associate Professor, Department of Neurology, Peking Union Medical College Hospital, China

Xihua Li, Director, Department of Neuromuscular Disease, Children’s Hospital of Fudan University, China

Stanley Nelson, Professor of Human Genetics, Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine at UCLA

Richard Parad, Associate Professor of Pediatrics, Harvard Medical School, Department of Pediatric Newborn Medicine, Brigham and Women’s Hospital

Stacey Tay, Senior Consultant, Department of Paediatrics, Khoo Teck Puat – National University Children’s Medical Institute, National University Hospital, Singapore

10:30 AM

Closing

Hui Cai, VP and Head of Content, WuXi AppTec

SPEAKERS

Hui Cai

VP and Head of Content, WuXi AppTec

Dr. Hui Cai joined WuXi AppTec in 2009 as Vice President of Business Development, and is currently Vice President and Head of Content Division.

Prior to WuXi, Dr. Cai spent 10 years at Johnson & Johnson Pharmaceutical Research and Development leading multiple drug discovery programs in the therapeutic areas of inflammation and autoimmune diseases. She is a co-author and co-inventor to over 50 scientific publications and issued or pending patents. Dr. Cai is a Councilor of the American Chemical Society (ACS), a member of BayHelix, and a member of the UCSD Alumni Board. In her past capacity, she served as a Commissioner at the City of San Diego Science and Technology Commission, Chair of SABPA, and President of SDCA. Dr. Cai received her BS and MS in Chemistry from Peking University, PhD from The Scripps Research Institute, and MBA from UCSD Rady School of Management as a DLA Piper – Athena Scholar.

Pat Furlong

Founding President & CEO, Parent Project Muscular Dystrophy

Pat Furlong is the founding president and chief executive officer of Parent Project Muscular Dystrophy (PPMD), the largest nonprofit organization in the United States focused on Duchenne muscular dystrophy. After her two sons were diagnosed with Duchenne, Ms. Furlong joined other parents to found PPMD in 1994. The mission of PPMD is to END Duchenne. PPMD invests in research and has leveraged >500 M, advancing every possible opportunity to accelerate therapy development. PPMD is focused on three major goals: impact, influence and change policy.

Since 2000, PPMD has influenced policy – Children’s Health Act, MD CARE Act, Congressionally Directed Medical Research Program (CDMRP), Written Guidance for FDA in 2016 and currently developing draft Guidance to include gene therapy. With the goal of ensuring every single person diagnosed with duchenne or becker have access to optimal care, opportunities to participate in clinical trials and access to therapy.

Ms. Furlong has served on the boards of the Genetic Alliance and the National Organization for Rare Disorders, the National Institutes of Health’s Muscular Dystrophy Coordinating Committee, the Institute of Medicine’s Committee on Pediatric Studies Conducted Under BPCA and PREA, and the U.S. Food and Drug Administration’s Molecular and Clinical Genetics Panel. For her leadership in changing the course of Duchenne, Ms. Furlong was named among the top 50 orphan drug thought leaders by the World Orphan Drug Congress in 2014. She received the 2012 Global Genes | RARE Champion of Hope Advocacy Award and the 2008 Research America Advocacy Award. She serves on the Board of the ARM foundation and on the Advisory Board of Orphan Disease Center, U. Penn.

Debra Miller

CEO & Founder, CureDuchenne

Debra Miller is CEO and Founder of CureDuchenne. She, and her husband Paul, started the organization in 2002 after their only son, Hawken, was diagnosed with Duchenne. Debra and her team have brought many firsts to the Duchenne community, including a Duchenne-focused venture philanthropy model, an annual nationwide community education event tour (virtual for now), a Duchenne Physical Therapy Certification program and an open access Biobank. Debra proudly serves as the Patient Representative on TREAT-NMD’s Executive Committee and reviews Duchenne applications for the Department of Defense’s Congressionally Directed Medical Research Program. A native Californian, Debra graduated from Mariner’s High School in Huntington Beach and earned a Bachelor of Arts in Communication Studies from the University of California Los Angeles. Prior to CureDuchenne, Debra had a career in publishing with positions at IDG Communications, Cahners Publishing, Ziff-Davis Publishing, and Scholastic Publishing. She also worked in management at PC Magazine and was an independent stock trader.

Filippo Buccella

Founder, Parent Project Italy APS

As the father of a young man affected by Duchenne, Filippo, in 1995, founded Parent Project, an association to advocate in Italy for Duchenne and Becker patients. Filippo always believed that Parent Project’s work could open a new path for many other similar diseases and conditions. In his past role as president of Parent Project in Italy, he established good working relationships with many other organizations worldwide. This network proved invaluable when he was on the executive committee of the TREAT-NMD Alliance and the United Parent Project Muscular Dystrophy. Filippo is keen to continue his work to amplify the voice of patients as key stakeholders and collaborate with patient organizations to represent their views. Filippo is also a pharmacist. His knowledge of the mechanisms of action of drugs and drug development is beneficial for translating complicated topics for families and patients. Since the beginning, he has been working with patient registries. He set up a national DMD/BMD patient registry in Italy, instrumental in conducting clinical trials.

Huigu Chen

President, Shanghai Jiai Myopathy Care Center, China

Tali Kaplan

Co-Founder & CEO, Little Steps Association for Patients with Duchenne & Becker Muscular Dystrophy, Israel

Tali Kaplan is co-founder and CEO of Little Steps, an Israeli NGO founded in 2010 by parents of children with Duchenne and Becker muscular dystrophy. Tali is the proud mother of Ido, a 13-year-old boy that was diagnosed with Duchenne when he was only 1 year old.

Little Steps mission is to improve the life quality of patients with DMD/BMD and extend their life expectancy.

Ido's diagnosis of a rare and incurable disease led Tali to leave her previous job as an economist in high-tech companies and establish the association together with Ziva Goldschmidt, the mother of Adi who also suffers from the disease.

The decisions Tali and Ziva had taken was to enrich the knowledge of the families about the disease, establish contacts with Duchenne organizations around the world to learn and share knowledge, and to set up a support system by social workers who will help families from the first phase of diagnosis and throughout dealing with the disease challenges.
At the same time, Tali was working to build a registry of the patients as a platform to bring research to Israel, made connect with researchers to raise funds to find a cure.

Tali is a member of the Board of Directors of the Coalition for Rare Diseases in Israel and in international forums for promoting the treatment and research.
Tali believes that collaborations in Israel and around the world are the key to advancing knowledge and treating the disease.

Sherena Loh

Director, Muscular Dystrophy Association (Singapore)

Sherena Loh is a Co-Founder and Director of Muscular Dystrophy Association (Singapore). She is an ardent champion for the mobility challenged. She is a wheelchair-user because she has muscular dystrophy.

Sherena was once told that she would not live beyond 25 years old. But her heart proved stronger than her muscles. She has been volunteering with disability organisations and collaborating with government agencies for more than two decades, transforming the quality of life for people who are differently-abled.

Sherena holds a Bachelor of Social Science from Edith Cowan University, Australia, as well as a Diploma of Family Life Education and a Diploma in Business Administration. She is the author of Shaped For A Purpose.

Kevin Flanigan

Director, Center for Gene Therapy, Professor, Pediatrics & Neurology, Nationwide Children’s Hospital

Kevin Flanigan, M.D., is the Director of The Center for Gene Therapy at Nationwide Children’s Hospital in Columbus, Ohio, where he holds the Robert F. & Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research. He directs the NCH Neuromuscular Program as well as Nationwide Children’s NIAMS P50-funded Center for Research Translation (CORT) in Muscular Dystrophy Therapeutic Development. He trained in Neurology & Neuromuscular Medicine at Johns Hopkins University, pursued a post-doctoral fellowship in Human Molecular Biology and Genetics at the University of Utah, and currently holds appointments as Professor of Pediatrics and Neurology at the Ohio State University. His research focuses on genotype/phenotype correlations in the muscular dystrophies, with a goal of understanding molecular mechanisms that lead to amelioration of symptoms in order to identify new therapeutic pathways. He has served as sponsor-investigator on trials of AAV gene replacement therapy for mucopolysaccharidosis types 3A and 3B, as well as a surrogate gene therapy for Duchenne muscular dystrophy (DMD). His laboratory works primarily on AAV-based approaches to gene therapy, including viral-vector based exon skipping and gene replacement, including a U7snRNA therapy for DMD now in clinical trial.

Stanley Nelson

Professor of Human Genetics, Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine at UCLA

Stanley F. Nelson, MD is a physician scientist who develops and implements genomic tools to facilitate mutation discovery, improve molecular diagnostics for rare diseases, and performs translational research related to Duchenne muscular dystrophy. His work has led to the identification of dozens of gene mutations causing human disease and novel approaches to diagnosis. He graduated from Duke University School of Medicine in 1987, and subsequently trained in Pediatric Hematology-Oncology at UCSF. He completed postdoctoral fellowship in 1993 at Stanford University in genomics with Dr. Patrick Brown. He is Professor of Human Genetics, Pediatrics, Neurology and Pathology and Laboratory Medicine at the David Geffen School of Medicine at UCLA where he has been faculty since 1993. He established the first genomics core at UCLA in 1996. He is Founder in 2007 and Co-Director of the Center for Duchenne Muscular Dystrophy which is a multi-investigator program encompassing graduate and undergraduate education, basic and translational research, clinical care, and clinical trials for Duchenne at UCLA. In 2011, he founded the Clinical Genomics Center, with others, which develops and implements clinical genomic testing and established clinical exome sequencing at UCLA. He is a PI since 2014 of a Clinical Site of the Undiagnosed Diseases Network, at UCLA which performs discovery of new rare diseases and implementing improvements in transcriptome analysis to augment whole genome sequencing. Since, 2019 he is Director of the California Center for Rare Diseases.

Richard Parad

Associate Professor of Pediatrics, Harvard Medical School, Department of Pediatric Newborn Medicine, Brigham and Women’s Hospital

Dr. Parad is an attending neonatologist, dual boarded in Neonatal-Perinatal Medicine and Pediatric Pulmonology, and an Associate Professor of Pediatrics at Harvard Medical School. He also received training in clinical trial design at the Harvard School of Public Health. He is an NIH-funded translational scientist with expertise in neonatal respiratory drug development, newborn screening and genomic medicine. He is currently Director of the Department of Pediatric Newborn Medicine’s Neonatal Genomic Medicine Program.

Hawken Miller

Features Writer, BioNews

Hawken Miller is a writer, journalist, producer, and Duchenne muscular dystrophy advocate. He is currently a features writer for BioNews, where he tells stories about people making a difference in the rare disease community. A former intern on The Washington Post’s video game and esports desk, Launcher, Hawken helps produce their weekly live streams. Hawken is also a research producer at Paragon Creative Agency, where he produces two Call of Duty League YouTube shows. He is a spokesperson and speaker for the research-and-care-focused organization, CureDuchenne, founded by his parents, Paul and Debra Miller. Hawken was diagnosed with Duchenne at 5 years old in 2002 but hasn’t let that stop him from becoming an Eagle Scout, graduating with honors from the University of Southern California in 2019, or working and interning at prestigious news organizations like The Sacramento Bee, KTLA 5 News, and the Orange County Register. When he isn’t working, Hawken enjoys being active in his church community, livestreaming on his Twitch channel, TheHawkLive, and playing with his six-year-old rescue pit bull-border collie mix, Patch.

Michael Binks

VP & Head of Clinical Research in the Rare Disease Research Unit at Pfizer

Michael is the VP and head of Clinical Research in the Rare Disease Research Unit at Pfizer, based in Cambridge, MA. He has 2 decades of experience in the discovery, translation and early clinical development of therapeutics of multiple modalities in rare and common disease. For the past 7 years he has been involved in drug development programs in Duchenne muscular dystrophy, most recently as the program lead for DMD gene therapy. Michael is a UK trained physician (MRCP) and an accredited specialist in Rheumatology.

Paula Clemens

Professor & Vice Chair, Neurology, University of Pittsburgh School of Medicine

Paula Clemens is a clinician and scientist at the University of Pittsburgh. She has expertise in the care of adults with neuromuscular diseases such as Duchenne and Becker muscular dystrophy, myotonic dystrophy, spinal muscular atrophy and late-onset Pompe disease. Her primary research focus is translational approaches that advance the understanding and treatment of genetic muscle disorders, including Duchenne and Becker muscular dystrophy and late-onset Pompe disease. In addition to her university-based practice and research efforts, she directs the Neurology Division at the VA Pittsburgh Healthcare System.

She has been a member of CINRG and a site PI since its founding in 2000. She has been Medical Director since 2010.

Nathalie Goemans

Professor, Neuromuscular Reference Centre for Children, Department of Pediatric Neurology, University Hospitals Leuven, Belgium

Nathalie Goemans is a paediatrician and child neurologist, with certification in rehabilitation medicine, affiliated to the KUL University of Leuven, currently emeritus since November 2020. She was a staff member since 1987 at the University Hospitals Leuven, Belgium, as head of clinic within the department of Pediatrics (Child Neurology) and head of the Neuromuscular Reference Centre for Children at the University Hospitals Leuven, and assistant professor at the Faculty of Medicine, University of Leuven, Belgium. She is a consultant at DVC Heder, Antwerp, Belgium, a rehabilitation centre with residential setting for neuromuscular patients.

She is actively involved in national and international research networks, with a particular interest in the multidisciplinary aspects and the dissemination of standards of care in neuromuscular disorders and the development of international registries. Research activities involve translational and clinical research in various neuromuscular disorders, the development of new therapies for Duchenne muscular dystrophy and spinal muscular atrophies and the research and development of outcome measures to assess interventions in these diseases.

Timothy Franson

Principal, Faegre Drinker Consulting and formerly VP Global Regulatory Affairs, Eli Lilly & Co and Chief Medical Officer of YourEncore

Dr. Tim Franson serves as Principal at Faegre Drinker Consulting since 2019. He was Senior Vice President at Baker and Daniels Consulting (later Faegre Baker Daniels Consulting) from 2009-2014, then served as chief medical officer at YourEncore from 2014 to 2019. He also served as VP of global regulatory affairs at Lilly from 2003 to 2008, retiring from that position after 21 years at Lilly. From 1997 to 2003, he served as Lilly’s VP of clinical research and regulatory affairs.

Franson provides clinical and regulatory guidance for drug and biologics development (including strategy, planning, training and issues management), post-approval advice (REM's, labeling and compliance) and due diligence. During his time at Lilly, Tim was directly responsible for the company’s FDA submissions (NDAs and supplements), which involved more than 20 major submission reviews and approvals. He also played a leadership role in regulatory compliance and policy matters.

Tim has worked to advance the objectives of various rare disease communities. He sat on the steering committee for the Duchenne Patient Community Draft Guidance submitted to the FDA, served as an advisor to the ALS Community Draft Guidance to the FDA, and advised the Lupus community in the externally led FDA patient-focused drug development (PFDD) workshop, as well as serving as moderator for the 2019 FDA externally facilitated PFDD public meeting for the Niemann Pick Type C disease community. He also served on the NIH-NCATS Treatment of Rare Diseases (TRND) review panel.

Tim has been a leader in many legislative and regulatory initiatives impacting the pharmaceutical and health and biosciences industries. He was co-chair of the joint FDA-industry working group addressing clinical aspects of the FDA Modernization Act of 1997, including the Prescription Drug User Fee Act (PDUFA) renewal. From 2000 to 2003, he co-chaired industry FDA committees for PDUFA-3 renewal and has testified, on behalf of industry stakeholders, at several congressional hearings.

Franson is past President of the US Pharmacopeial (USP) Convention (2010-2015) and immediate past Board Chair of the Critical Path Institute, as well as a Board member of Paratek Pharmaceuticals, Cidara Therapeutics, the AMPATH Development Board for the IU Global Health Program, and the Executive Leadership Board for the University of Iowa College of Pharmacy. He serves as Adjunct Professor of Medicine at Indiana University School of Medicine.
Tim has authored more than 50 articles in the fields of regulatory affairs, infectious disease, epidemiology, pharmacoeconomics and antibiotic utilization.

Rhonda Bassel-Duby

Professor, Department of Molecular Biology, University of Texas Southwestern Medical Center

Rhonda Bassel-Duby, Ph.D. is a Professor of Molecular Biology, and an Associate Director of the Hamon Center for Regenerative Science and Medicine at the University of Texas Southwestern Medical Center. She is also a Principal Investigator of the Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center at UT Southwestern, recently awarded by the NIH.

Dr. Bassel-Duby grew up in Montreal Canada, and received her Ph.D. degree in Biochemistry from McGill University in Montreal, Canada. She trained at Harvard Medical School and University of Texas Southwestern Medical Center.

Over the past two decades, her research focused on the molecular mechanisms governing cardiac and skeletal muscle growth, development and disease.
Recently, she has been involved in using CRISPR/Cas9-mediated genomic editing to correct mutations in Duchenne muscular dystrophy.

Over the years, she has mentored many graduate students and postdoctoral fellows, and has co-authored over 190 publications in peer-reviewed journals.

She is a member of numerous institutional committees, including being the Chairperson of the ARC Advisory Committee plus a member of the Stem Cell Research Oversight Committee.

Additionally, she has served on NASA Musculoskeletal Biology Review Panels, NIH Study Sections and has been a Chairperson of an American Heart Association Review Consortium Committee. She also served on the American Heart Association Advisory Research Committee.

Fun fact: She loves to dance with her NIA (Neuromuscular Integrative Action) dance group (virtually these days) and do virtual yoga and Pilates.

Ashish Dugar

SVP & Global Head of Medical Affairs, Dyne Therapeutics

Dr. Dugar brings to Dyne over 20 years of broad experience across medical affairs, clinical and commercial development, and real-world evidence (RWE) generation. Prior to joining Dyne, Dr. Dugar served as vice president and global head of medical affairs at Sarepta Therapeutics, Inc., where he built the company’s medical affairs organization. Previously, he led all commercial and market access efforts for Intra-Cellular Therapies, Inc. as vice president and head of commercial development. Dr. Dugar joined Intra-Cellular Therapies following his tenure at Roche, where he was global head of clinical development science and innovation and global head of the external development group, including work in oligonucleotides. Previously, he spent 13 years at Pfizer Inc. in various roles, most recently serving as vice president, global head of clinical development and medical affairs of the biosimilars business unit. Dr. Dugar began his career at the National Institutes of Health. He completed a pharmacoeconomics and outcomes research fellowship at the University of Michigan School of Public Health. Dr. Dugar earned an MBA from The Pennsylvania State University and a Ph.D. in pharmacology from The Pennsylvania State University College of Medicine.

Jane Larkindale

VP, Clinical Sciences, PepGen

Jane Larkindale is the Vice President of Clinical Science at PepGen, a company developing enhanced delivery oligonucleotide therapeutics with lead programs in Duchenne muscular dystrophy and myotonic dystrophy. She has dedicated the past 15 years of her career to accelerating therapy development for rare diseases, with a focus on neuromuscular diseases. She launched and ran international consortia and programs focused on data standardization and aggregation, and use of that data to support regulatory acceptance of disease models, outcome assessments and biomarkers to accelerate drug development. She conceived of and subsequently led the Rare Disease Cures Accelerator, Data and Analytics Platform and multiple disease specific consortia at the Critical Path Institute, as well as programs at the Friedreich’s Ataxia Research Alliance and the Muscular Dystrophy Association, all with the goal of accelerating rare disease drug development. These programs have built community consensus on best practices for drug development, and formal regulatory endorsement of several tools is underway. She launched and ran MDA Venture Philanthropy in 2007, which supported and guided 21 drug development projects through pre-clinical and early clinical phases of development, leaving MDA as the Vice President for Research in 2014. She went on to form NMD Consulting to support research and clinical strategy to accelerate drug development and worked with a multitude of for-profit and not-for-profit clients. She is a molecular biologist by training, having completed her D.Phil. (Ph.D.) in the Department of Plant Sciences at Oxford University, which she attended on a Rhodes Scholarship.

Art Levin

CSO, Avidity Biosciences

Art Levin is a key opinion leader in the RNA therapeutics field. He serves as the Chief Scientific Officer at Avidity Biosciences Inc. Previously, he held Senior levels positions at mRiragen, Santaris Pharma, and Ionis Pharmaceuticals. He played key roles in developing numerous oligonucleotides, including the first approved antisense drugs and the first microRNA-targeted therapeutic in clinical trials. He has three decades of experience in drug development from discovery through drug registration. Dr. Levin has published over 60 scientific articles and several of the most cited reviews in the field. He is on the scientific advisory boards of multiple institutions. Dr. Levin received a doctorate in toxicology from the University of Rochester.

Stuart Peltz

Founder & CEO, PTC Therapeutics

Dr. Peltz founded PTC Therapeutics in 1998 and has served as CEO and a member of the Board of Directors since the company’s inception. Under his leadership, PTC has grown from a research organization into a global, publicly traded, fully-integrated biotech company with multiple approved products. PTC’s hallmark is its deep commitment to science, employing multiple technology platforms to drive a robust discovery pipeline in support of Patients with rare disorders around the world.

Prior to founding PTC, Dr. Peltz was a Professor in the Department of Molecular Genetics & Microbiology at the Robert Wood Johnson Medical School, Rutgers University. Dr. Peltz is a recognized scientific leader in RNA biology in the area of post-transcriptional control processes involving mRNA turnover and translation, with more than 30 years of research and over 100 publications in the field. Dr. Peltz was elected as a Fellow of the American Academy for the Advancement of Science in 2010. He was recognized as PharmaVOICE’s 100 Most Inspiring People in 2009 and received the Dr. Sol J. Barer Award for Vision, Innovation and Leadership in 2014.

Dr. Peltz received a Ph.D. from the McArdle Laboratory for Cancer Research at the University of Wisconsin.

Richard Soll

Head of Boston Office and Senior Advisor of Strategic Initiatives, WuXi AppTec

Dr. Richard Soll is presently Senior Advisor, Strategic Initiatives for the Research Service Division at WuXi AppTec and Head of the WuXi office in Boston, Massachusetts. He has held various leadership roles at WuXi as Senior Vice President including head of the medicinal chemistry unit known as the International Discovery Service Unit, business development, and corporate alliances.

Dr. Soll’s contributions led to the discovery of the HCV NS5A inhibitor elbasvir (a component of Zepatier®) in the Merck-WuXi partnership, the JAK2 inhibitor fedratinib at TargeGen which formed the basis of the $7B partnership between Celgene and Impact Biomedicines, and more than 10 other clinical stage drugs throughout his career.

Dr. Soll is currently a board member at Simcha Therapeutics, an board observer at the Accelerator Life Science Parnters, and an advisory board member with the Blavatnik Center for Drug Discovery at Tel Aviv University, the Children’s Tumor Foundation, and the Pistoia Alliance.

Dr. Soll was CSO / VP of R&D at TargeGen and was VP of Chemistry at 3-Dimensional Pharmaceuticals. He started his career at Ayerst Research Labs and was trained as a synthetic chemist at Dartmouth and Harvard.

Yi Dai

Associate Professor, Department of Neurology, Peking Union Medical College Hospital, China

Xihua Li

Director, Department of Neuromuscular Disease, Children’s Hospital of Fudan University, China

Stacey Tay

Senior Consultant, Department of Paediatrics, Khoo Teck Puat - National University Children's Medical Institute, National University Hospital, Singapore

A/Prof Stacey Tay is a senior consultant paediatric neurologist with a special interest in neurogenetics, neuromuscular disorders and neurodegenerative disorders of childhood. She obtained her basic MBBS medical degree with honours in 1994, and went on to obtain her postgraduate paediatric degrees in 1997. She won the NUH Young Doctor’s Award in 2001 for her research in Duchenne Muscular Dystrophy. She was on a clinical and research Child Neurology fellowship from 2002–2004 at Columbia University, New York, and worked as a fellow in the Children’s Hospital of New York.

She anchors multidisciplinary neuromuscular and neurogenetic clinics in NUH and works with community partners like Ronald McDonald House Charities and Muscular Dystrophy Association Singapore to bridge hospital and community care.

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