Coming back in 2019!
Boston, MA, USA

 

2018 Speakers

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Michael Binks
Vice President & Head of Rare Disease Clinical Research, Worldwide Research & Development Verified Date 21/06/2018 Local Job Title
Pfizer

Day One

Monday, October 29

08.40 | Gene Therapy in Duchenne Muscular Dystrophy

Brian Kaspar
Chief Scientific Officer
Avexis

Dr. Kaspar is Scientific Founder and Chief Scientific Officer at AveXis, Inc., a clinical-stage gene therapy company focused on the treatment of neurological diseases. He is a former Endowed Chair and Professor of Pediatrics at The Ohio State University. His work on gene therapy for spinal muscular atrophy was Science's People's Choice Award for 2017 Breakthrough of the Year. Dr. Kaspar.  He is a Fellow of the American Association for the Advancement of Science (FAAAS) with over 100 peer reviewed manuscripts and book chapters.  He serves on NIH Review Panels, a number of ASGCT committees, as well as serves on the editorial board of Molecular Therapy.  

Day One

Monday, October 29

09.10 | Translation of Gene Therapeutics in Neurological & Neuromuscular Diseases

Rajeev Sivasankaran
Head Rare Diseases
Novartis

Rajeev Sivasankaran PhD, currently leads the Rare Diseases group in the Neuroscience Division at Novartis Institutes of Biomedical Research. His group is responsible for initiating and establishing drug discovery programs focused on genetically defined targets in orphan neurodegenerative diseases. He has led multiple target validation efforts and screening campaigns at NIBR. He has also advanced rare disease programs from high throughput screening to the clinic. Dr Sivasankaran completed his doctoral work in molecular biology at the University of Zurich and his post-doctoral work at Children’s Hospital / Harvard Medical School.  

Day Two

Tuesday, October 30

11.30 | Developing a Potential Therapy for an Orphan Neuromuscular Disease

Brian Bonk
Head of External Innovation & Rare Diseases
Sanofi

Brian is the Head of External Innovation for the Rare Diseases Therapeutic Area at Sanofi. In this role, he and his teams are responsible for working with senior leadership to define an overall portfolio strategy and execute on the external activities, with accountability for identification and securing of external innovation solutions from around the globe that meet the needs of the R&D portfolio. Prior to this role, Brian was a member of the Sanofi Sunrise team, where his responsibilities spanned a range of activities related to building and maintaining a vibrant portfolio of investments, including Warp Drive Bio and Thermalin. Prior to joining Sanofi in 2014, Brian served as an advisor to a wide array of life science organizations, along with venture capital groups, where he contributed to multiple teams reaching key R&D and financing milestones. From 2009-2013, Brian was the Vice President of R&D at Satori Pharmaceuticals. Before Satori, Brian worked at Pfizer, rising to the level of Senior Director. Brian and his teams have been involved in the discovery of more than 20 development candidates, including Draxxin™, Convenia™, Cerina™ and Slentrol™ Brian earned his bachelor’s degree from Colgate University in 1989. Following a year as a Fulbright Fellow in Dortmund, Germany, he initiated his doctoral work at the Massachusetts Institute of Technology, receiving his doctorate in chemistry in 1994.  

Day Two

Tuesday, October 30

17.00 | Panel/Round table: Looking into Payment Models to Ensure Commercial Success when Bringing Neuromuscular Therapies to Market

10.10 | Panel Discussion: Exploring the Benefits and Challenges of Combining “Next Generation” & Existing Therapeutics

Daniel Cohen
Chief Executive Officer
Pharnext

More than 30 years' experience in academia, biotech and big pharma companies. Professor of Medical Genetics, founder of modern genetics, genomics and pharmacogenomics. Former CSO of Genset (acquired by Serono) and VP Genetics Worldwide of Serono Genetics Institute. Co-founder of Millennium Pharma, Genethon and the CEPH research institute. Author of 150 peer-reviewed publications.  

Day Two

Tuesday, October 30

09.10 | Showcasing the Natural History of PXT 3003, a Pleodrug for CMT1A

Salvador Rico
Vice President, Clinical Development
Audentes

Sal Rico is Vice President of Clinical Development at Audentes Therapeutics. He is a clinical pharmacologist with 13 years of clinical research experience in the pharmaceutical industry, contract research organizations and academia. He has participated in the design, conduct, and analysis of phase 1-4 clinical trials in multiple therapeutic areas including rare diseases, hematology/oncology, transfusion medicine, psychiatry, sleep medicine, cardiovascular medicine, infectious diseases, pain, allergy, and dermatology. Prior to joining Audentes he led the clinical development teams at Cerus Corporation and Transcept Pharmaceuticals, and was an investigator at the Centre for Drug Research, Hospital de la Santa Creu i Sant Pau in Barcelona, Spain. His clinical research and development experience includes drugs, biological products and medical devices, such as the INTERCEPT Blood System®, INTERMEZZO®, HIBOR®, CHAMPIX®, PROLIA® and KERYDIN®, among others. Dr. Rico was awarded a Doctor of Medicine and Surgery degree from the National Autonomous University of Mexico and holds a Masters in Science and a Ph.D. (Summa Cum Laude) in Pharmacology from the Universitat Autonoma de Barcelona. Additionally, he has received postdoctoral training in clinical pharmacology, transfusion medicine (National Autonomous University of Mexico), and pharmaceutical medicine (Universitat Autonoma de Barcelona).    

Day Two

Tuesday, October 30

10.10 | Panel Discussion: Exploring the Benefits and Challenges of Combining “Next Generation” & Existing Therapeutics

09.40 | Gene Therapy Clinical Development Program for X-Linked Myotubular Myopathy (XLMTM), a Rare, Severe Neuromuscular Disease

Nikolai Naryshkin
Vice President
PTC Therapeutics

Dr. Naryshkin is Vice President of Biology and has been involved in discovery and development of small molecule modulators of RNA-mediated processes for over 16 years. Dr. Naryshkin’s current work is focused on discovery and development of small molecule splicing modifiers as oral therapeutics for neuromuscular disorders. Two of the most advanced programs are SMN2 splicing modifiers for treatment of spinal muscular atrophy (in collaboration with Roche and the SMA Foundation) and IKBKAP splicing modifiers for treatment of familial dysautonomia (in collaboration with the Slaugenhaupt lab at Massachusetts General Hospital). Dr. Naryshkin received his Ph.D. in bioorganic chemistry from Moscow State University in Moscow, Russia, and completed postdoctoral training in molecular biology at Rutgers University.  

Day Two

Tuesday, October 30

12.00 | Evolving Therapeutic Approaches for Treatment of Neuromuscular Disorders

Gregory Petsko
Chief Scientific Officer
MeiraGTx

Gregory A. Petsko, D. Phil. is the Arthur J. Mahon Professor of Neurology and Neuroscience at Weill Cornell Medical College in New York City, where he is also Director of the Helen and Robert Appel Alzheimer’s Disease Research Institute. He received his BA from Princeton University, summa cum laude, in 1970, and his D. Phil. from Oxford University (which he attended as a Rhodes Scholar) in Molecular Biophysics in 1973.  He was Professor of Chemistry at MIT from 1978 until 1990, when he moved to Brandeis University as Gyula and Katica Tauber Professor of Biochemistry and Chemistry, Director of the Rosenstiel Basic Medical Sciences Research Center, and Chair of the Department of Biochemistry. He moved to Weill Cornell Medical College in April 2012. His awards include the Siddhu Award and the Martin J. Buerger Award, both from the American Crystallographic Association (35 years apart), the Pfizer Award in Enzyme Chemistry of the American Chemical Society (for development of methods to visualize reaction intermediates in three dimensions at atomic resolution), the Lynen Medal for pioneering contributions to the study of protein dynamics, the McKnight Endowment for Neuroscience Brain Disorders Award, a Guggenheim Fellowship, and in 1991 the Max Planck Prize, shared with Professor Roger Goody of Heidelberg for their joint work on the molecular origins of Ras-dependent human cancers. He is an elected Fellow of the American Association for the Advancement of Science. He has been elected to the National Academy of Sciences, the National Academy of Medicine, the American Academy of Arts and Sciences, and the American Philosophical Society.  He has an honorary Doctor of Laws from Dalhousie University.  He is Past-President both of the American Society for Biochemistry and Molecular Biology and of the International Union of Biochemistry and Molecular Biology and past Chair of the Medical Sciences Section of the AAAS. He has been involved in founding several publicly-traded biotechnology companies (Arqule [ARQL], Ironwood Pharmaceuticals [IRWD] and Denali Therapeutics [DNLI]) and is Chief Scientist at Meira GTx, a gene therapy company.  His research interests focus on the development of methods to treat age-related neurodegenerative diseases, including ALS (Lou Gehrig’s), Alzheimer’s and Parkinson’s diseases, and the development of better models for brain disorders including brain cancers. His public lectures on the aging of the population and its implications for human health have attracted a wide audience in person and on the Internet (one of his TED talks, for example, has been viewed almost a million times).  For many years he has also written a widely-read and much reprinted column on science and society, the first ten years of which have recently appeared in book form.  He admits, however, that the columns guest-written by his two dogs, Mink and Clifford, are much more popular than those he writes himself.

Workshop B

Wednesday, 31 October

12.00 | Exploring New Approaches to The Treatment of Neuromuscular Diseases Starting from Genetic & Biochemical Studies in a Model Organism

Eva Chin
Director
Cytokinetics

Experienced Director of Pharmacology with a demonstrated history of working in the biotechnology industry. Skilled in Physiology, Pharmacology, cell and Molecular Biology, Life Sciences, Entrepreneurship, and Pharmaceutical Research & Development. Strong research professional with a Certificate of Entrepreneurship focused in INNoVATE Program from The Johns Hopkins University - Carey Business School.

 

Day Two

Tuesday, October 30

12.30 | Fast Skeletal Muscle Troponin Activators as Treatments for Neuromuscular Diseases

10.10 | Panel Discussion: Exploring the Benefits and Challenges of Combining “Next Generation” & Existing Therapeutics

Chikwendu Ibebunjo
Senior Scientist
Novartis

Chikwendu Ibebunjo is a Senior Investigator at the Novartis Institutes for Biomedical Research where his research is focused on establishing and applying translational preclinical models in drug discovery for skeletal muscle diseases including sarcopenia, cachexia and neuromuscular diseases.  Prior to Novartis, Chikwendu was at Pfizer Inc. where he led studies to demonstrate the efficacy of anti-myostatin antibodies in models of various skeletal muscle diseases. Chikwendu received a Ph.D. from the University of Cambridge, England, and a D.V.M. from the University of Nigeria, Nsukka, Nigeria.  

Day One

Monday, October 29

11.30 | Improving the Translational Validity of Animal Models of Disease

Kim Long
Senior Scientist
Scholar Rock

Kimberly joined Scholar Rock in 2015 where she leads the preclinical research efforts involving skeletal muscle and SRK-015, an inhibitor of myostatin activation currently in clinical development. Prior to Scholar Rock, Kimberly was a member of preclinical pharmacology teams at Pfizer and Ironwood. She holds a PhD from Stanford University and a BA from Northwestern University.  

Day One

Monday, October 29

11.00  | Selective Inhibition of Myostatin Activation is Beneficial in Mouse Models of SMA Therapy

Sanjay Rakhade
Senior Director,
Medical Sarepta

Sanjay Rakhade MD, PhD is a physician-scientist and currently serves as Senior Medical Director, at Sarepta Therapeutics.  Sanjay is a neurologist by training, and oversees the clinical trials for exon-skipping therapies in clinical development for DMD at Sarepta Therapeutics. He was previously at Sanofi Genzyme, where he had roles of increasing responsibility over a period of 6 years, most recently as Global Medical Lead, Multiple Sclerosis and Neurology.  Sanjay has experience across multiple phases of clinical development, and across a spectrum of therapies and disorders, including pivotal Phase 3 studies for alemtuzumab (Lemtrada®) for multiple sclerosis, and eliglustat tartrate (Cerdelga®) for Gaucher disease.  In addition, Sanjay has worked on multiple Phase 1 and 2 programs in MS, and enzyme replacement therapies in lysosomal storage diseases. Sanjay has published his research in several leading neurology journals including Nature Reviews Neurology, Annals of Neurology, and Journal of Neuroscience etc. Prior to Sanofi Genzyme, Sanjay was on faculty at the Department of Neurology Harvard Medical School and Children’s Hospital, Boston.  Sanjay received his MD from Grant Medical College, University of Bombay, his PhD in Molecular Medicine and Genetics from Wayne State University School of Medicine, and medical training in neurology at Children’s Hospital of Michigan and Detroit Medical Center. He also did a fellowship in pediatric neurology at Children’s Hospital, Boston and Harvard Medical School.

Day One

Monday, October 29

16.30 | Clinical Development for Rare Disease Therapies: Making Trials Compelling for Key Stakeholders

Meg Wood
Director of Development
CureDuchenne

CureDuchenne is a leading patient advocacy group supporting the development landscape for a meaningful treatment for Duchenne. As Director of Business Development, Meg spearheads new initiatives to improve how DMD treatment and research entities interact with the patient community. Alongside CureDuchenne scientific leadership, Meg manages the CureDuchenne Venture portfolio and investment priorities. Prior to joining CureDuchenne, Meg helped launched the Oncology Impact Fund at MPM Capital, a unique venture product designed in partnership with UBS Europe. Meg received MPH and MSW degrees from Boston University after completing her undergraduate work at College of Charleston.  

Day One

Monday, October 29

16.00 | Building a Robust Strategy to Overcome Limited Patient Access to Clinical Trials

Fred Cohen
Chief Medical Officer
Strongbridge Bio

Fred joined Strongbridge in 2015 and has held roles of increasing responsibility, including senior vice president, global research and development, and vice president, clinical research and development. Fred is an endocrinologist by training with more than 20 years of drug and business development experience, most recently focused in development and commercialization of rare disease and specialty products. Prior to joining Strongbridge, Fred provided strategic and operational counsel to life science companies, actively supporting their development and licensing functions. Prior to that, he served as executive director, clinical pipeline, at Aptalis Pharma, where he was responsible for innovation strategy as well as building and advancing the company’s specialty pharma pipeline. He has also held research and development positions with Johnson & Johnson and Eli Lilly & Company. Fred holds an MD from Pennsylvania State University College of Medicine and an AB in biology from Franklin and Marshall College  

Day Two

Tuesday, October 30

08.40 | Overview of the Investigational Landscape in Neuromuscular Drugs

Jinsy Andrews
Director of Neuromuscular Clinical Trials
Columbia University

Jinsy Andrews, MD, MSc Assistant Professor of Neurology ; Director of Neuromuscular Clinical Trials; Jinsy A. Andrews, MD, MSc is an Assistant Professor of Neurology, in the Division of Neuromuscular Medicine and serves as the Director of Neuromuscular Clinical Trials.  

Workshop A

Wednesday, 31 October

08.00 | Evolving landscape of neuromuscular clinical trials

Lale Welsh
Chief Executive Officer
Neuromuscular Disease Foundation

I started out in film and spent most of my career in Silicon Valley. I retired in 2006 and started my own foundation for the arts. In that process I learned so much about the challenges of running a nonprofit and how much more difficult it is to run a successful nonprofit than a for-profit business. Because of that, I ended up creating a niche for myself. People ask me to help them run their nonprofits. I parachute in, take a diagnostic look at their problems, set up a business plan, do fundraising, and get them on their way. This organization contacted me to come in. The fit was that I happened to speak Persian. The founding family at that time believed this was a Jewish Persian disease and they thought that was important. Since I joined we’ve learned it’s prevalent in many different demographics. This is my fourth year with this organization.  

Day Two

Tuesday, October 30

16.00 | Rethinking Nonprofits & Patient Advocacy

Joanne Donovan
Chief Medical Officer, Senior Vice President, Clinical Development
Catabasis Pharmaceuticals

Joanne M. Donovan, M.D., Ph.D., has served as our Chief Medical Officer since July 2011. Since 1989, she has worked as a staff physician at the VA Boston Healthcare System, where she was formerly Chief of Gastroenterology. Dr. Donovan has held an appointment at Harvard Medical School since 1990, most recently as associate clinical professor of medicine. From 1998 to July 2011, Dr. Donovan served in positions of increasing responsibility, ultimately as vice president of clinical development, at Genzyme, a biotechnology company, which she joined through its acquisition of GelTex. Dr. Donovan holds a Ph.D. in medical engineering and medical physics from the Massachusetts Institute of Technology, an M.D. from Harvard Medical School and an S.B. from the Massachusetts Institute of Technology. She completed residency

Day Two

Tuesday, October 30

17.00 | Panel/Round table: Looking into Payment Models to Ensure Commercial Success when Bringing Neuromuscular Therapies to Market

Alon Ben-Noon
Chief Executive Officer
NeuroSense Therapeutics

Life Sciences Executive ; Leading NeuroSense Therapeutics, a Biotech startup which accelerates research,development and therapy for ALS (Amyotrophic Lateral Sclerosis). Alon Ben-Noon is an experienced project manager, specifically in the pharmaceutical industry, in which he has successfully managed many projects in leading companies, such as Teva Pharmaceuticals and Perrigo, including drug development  projects within Innovative R&D, operational projects and marketing projects, while working in a global environment, with many multi-disciplinary interfaces. Alon holds a BSc in Industrial Engineering and Management, and an MBA from Ben-Gurion University of the Negev.  

Day Two

Tuesday, October 30

16.30 | ALS Drug Re-purposing - Upsides & Downsides of the Business Model

Niren Murthy
Department of Bioengineering
University of California, Berkley

Dr. Niren Murthy is a professor in the Department of Bioengineering at the University of California at Berkeley. Dr. Murthy’s scientific career has focused on the molecular design and synthesis of new materials for drug delivery and molecular imaging, The Murthy laboratory developed the hydrocyanines in 2009, which are now one of the most commonly used probes for imaging reactive oxygen species and commercially available from multiple sources. The Murthy laboratory has developed several new nanoparticulate technologies for drug delivery, such as the polyketals and CRISPR-Gold, which have been used by numerous laboratories to enhance the delivery of small molecules and proteins. Dr. Murthy received the NSF CAREER award in 2006, and the 2009 Society for Biomaterials Young Investigator Award.

Day Two

Tuesday, October 30

14.00 | In vivo Delivery of Cas9 Ribonucleoprotein & Donor DNA with Gold Nanoparticles

Matthew Pletcher
Head of Rare Diseases - Discovery
Roche

Mathew Pletcher currently serves as the Head of Rare Disease - Discovery for Roche, directing early drug development and research activities for rare neuromuscular, neurodevelopmental, and ophthamological diseases including spinal muscular atrophy, Duchenne muscular dystrophy, myotonic dystrophy, and Angelman Syndrome. Mathew received a Ph.D. in human genetics from the Johns Hopkins School of Medicine and conducted a post-doctoral fellowship at the Genomics Institute of the Novartis Research Foundation, publishing the first haplotype map of mouse inbred strains. After establishing his own laboratory at the Scripps Research Institute in Florida and acting as the director of its Genetics and Genomics Core Facility, Mathew joined Pfizer Global Research and Development, eventually serving as Director of Medical Genetics for Pfizer's Rare Disease Research Unit.  Most recently, Mathew served as the Chief Science Officer for Autism Speaks, the world's largest advocacy and research organization for autism.   As part of this role, he led MSSNG, a collaboration between Autism Speaks, Google, and the Hospital for Sick Children, to make available in an open-access database over 10,000 whole genome sequences from families with autism.  He has also founded the RDH12 Fund for Sight, a non-profit organization devoted to the development of a gene therapy for a genetic form of Leber Congenital Amaurosis, a congenital blinding disorder, is a board member of Odylia Therapeutics, a non-profit enterprise focused on developing gene therapies for ultra-rare diseases, and holds an adjunct appointment with the University of North Carolina.

Day One

Monday, October 29

13:30 | Shooting the Messanger - Targeting RNA for Treatment of Rare Disease

Juan Chavez
Experimental Medicine
Biogen

Day Two

Tuesday, October 30

14.30 | Nusinersen for Spinal Muscular Atrophy