Speakers

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Aileen Healy
Global Head, Rare Diseases
Pfizer/WRD
Aileen Healy is the Global Head for External Science and Innovation representing Rare Diseases at Pfizer. In this capacity, Aileen works to identify science that forms the basis of innovative therapies for patients with rare diseases. Aileen was Vice President of Preclinical Development at Cydan, a Cambridge-based rare disease accelerator. Aileen began work in the rare disease space at Seaside Therapeutics as Vice President of Research, where she focused on developing treatments to improve the course of neurodevelopmental disorders. Aileen has over 20 years of experience in the biotech field, including leadership roles at Momenta Pharmaceuticals and Millennium Pharmaceuticals. Aileen began her career as an Assistant Professor at Boston University School of Medicine. Aileen holds patents and has published in peer-reviewed journals on a wide range of topics including rare diseases. Aileen received her Ph.D. from Tufts University and postdoctoral training from the MIT.
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Alison Silva
CEO & President
Critical Outcome Technologies Inc., The Orphan Group
Alison Silva is the CEO & President at Critical Outcome Technologies, Inc. a clinical stage biotech company identifying and developing targeted therapeutics utilizing proprietary machine learning technology, with a focus in oncology and has served as a member of the Board of Directors since 2015. She was the co‐founder, Executive Vice President and COO at Synlogic, Inc. where she led strategy, process and operational aspects of engineered therapeutic probiotics. Ms. Silva is also a co‐founder of The Orphan Group.
Alison was COO at SLA Pharma, a biotech company, where she headed U.S. corporate and clinical operations for orphan drug candidates. In 2007, Ms. Silva became VP of Drug Development at Marina Biotech.
Ms. Silva began her career in drug development in clinical operations at Pfizer, Massachusetts General Hospital, and the University of Massachusetts and holds a Bachelor’s degree from Clark University and a Master’s degree from Clark University and UMass Medical Center.
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Alvin Shih
CEO
Enzyvant
Alvin is a physician executive and drug developer who is passionate about bringing together people, ideas, and resources to help patients and families affected by rare diseases. Alvin is the CEO of Enzyvant, a development-stage biotechnology company focused on advancing transformative therapies for patients with rare diseases. Prior to joining Enzyvant, Alvin was the head of R&D at Retrophin, where he led the development of drugs for renal, metabolic, and neurological diseases. Earlier, Alvin helped establish the Pfizer Rare Disease Research Unit in Cambridge MA, and he started his career as a healthcare consultant for LEK Consulting and McKinsey & Company. Alvin holds an MD from the University of Alabama, an MBA from the Kellogg School of Management at Northwestern University, and an undergraduate degree from Vanderbilt University. He completed his residency training in Internal Medicine at the Massachusetts General Hospital in Boston.
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Art Pappas
Managing Partner
Pappas Capital
Art has over 30 years’ experience as a pharmaceutical executive and life science venture capital investor and founded Pappas Capital in 1994. Prior to Pappas Capital, Art held senior leadership positions at Glaxo Holdings, Abbott International, Merrell Dow Pharmaceuticals and the Dow Chemical Company. Art is currently a director for Aura Biosciences and OrphoMed. He also serves on several non-profit boards including the North Carolina Biotechnology Center and The Wistar Institute. He is a decorated Vietnam veteran, having served in the US Army 101st Airborne Division. Art received a Bachelor’s in Science in Biology from Ohio State University and an MBA in Finance from Xavier University.
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Bharatt Chowrira
President & Chief of Business & Strategy
PureTech Health
Bharatt Chowrira is President and Chief of Business and Strategy at PureTech Health. He brings over 23 years of experience in the biopharma industry, combining a unique blend of research and development, corporate development, operations, financing, public offering, mergers and acquisitions, legal, IP, and licensing expertise. Dr. Chowrira most recently was the President of Synlogic and the Chief Operations Officer of Auspex Pharmaceuticals, later acquired by Teva Pharmaceuticals. He was President and Chief Executive Officer of Addex Therapeutics, a biotechnology company and held various leadership and management positions at Nekatar Therapeutics, Merck & Co., Sirna Therapeutics, and Ribozyme Pharmaceuticals. Dr. Chowrira has a J.D. from the University of Denver’s Sturm College of Law, a Ph.D. in Molecular Biology from the University of Vermont College of Medicine, an M.S. in Molecular Biology from Illinois State University and a B.S. in Microbiology from the UAS, Bangalore, India.
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Brad Margus
Co-Founder & CEO, Founder
Cerevance, A-T Children's Project
Brad Margus is co-founder and CEO of Cerevance, a new drug discovery company focused on brain diseases with sites in Massachusetts and the UK. In 2013, Margus started Genome Bridge, a non-profit subsidiary of the Broad Institute of Harvard and M.I.T., to build a computational platform for sharing genomic and clinical data. He co-founded Envoy Therapeutics, serving as CEO prior to its acquisition by Takeda in 2012. Mr. Margus also co-founded Perlegen Sciences and served as its Vice Chairman and CEO.
For the last 20 years, Margus has worked as founder and volunteer President of the A-T Children’s Project, a research based non-profit focused on ataxia telangiectasia or “A-T” that two of his sons have. A-T causes neurodegeneration, cancer and immune system problems.
Margus also currently serves on the Boards of Arvinas and Presage Biosciences and Second Genome. He is a member of National Institutes of Health committees: NCATS Sciences Advisory Council and the CAN Review Board.
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Bruno Gagnon
Acting VP, Clinical Operations
Eloxx Pharmaceuticals
Bruno is an opinion leader and influencer in the field of Clinical Trial Execution with an emphasis on rare and ultra-rare diseases. He currently serves as Vice President of Clinical Operations for small biopharma companies developing orphan drugs. Prior roles have included Vice President of Clinical Operations at BioMarin Pharmaceutical, Head of ClinOps at Roche Molecular and other Bay Area biotechs. Over his 25-year career, functions under his leadership have included Global Clinical Trial Management, Patient Advocacy, Medical Writing, Outsourcing and Contracts, Supply Chain Management, Clinical Data Management, Clinical Systems, Document Management and Clinical Training. Bruno is trained as a pharmacist with degrees from Laval University and University of Montreal, both in Quebec, Canada. Prior positions have included heading the Clinical Operations group at Roche Molecular Systems and FibroGen. He worked 6 years at Chiron Corporation in positions of increasing responsibilities.
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Camilla Simpson
Senior VP
BioMarin
Camilla V Simpson, SVP and Head of Product Development joined BioMarin in 2014. She is responsible for Corporate and R&D Governance, Program Leadership, R&D Project Management and Portfolio Strategy and Business Analytics. Prior to this role, Camilla held the position of Group Vice President, Head of Global Regulatory Affairs 2014 – 2017. Camilla has 22 years in industry and prior to BioMarin spent 12 years at Shire Pharmaceuticals where after multiple roles of increasing responsibility held the position of Vice President Regulatory Affairs; Early Development and Business Development. Before joining Shire, Camilla spent 7 years at Abbott Laboratories. Camilla has significant experience in global drug development and registration, due diligence and M&A plus leadership of globally based teams, talent and change management. She has operated at the senior leadership level for 11 years. Camilla holds a BSc from University College Galway, Ireland, BSc Hons from Kingston University, UK and an MSc with distinction from the University of London.
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Camille Samuels
Managing Partner
Venrock
Camille Samuels is a Partner at Venrock, a venture capital firm originally founded by the Rockefeller family in the 1960’s. She focuses on healthcare investing, particularly in biotech, medical devices, and consumer medicine. Camille currently serves on the board of Spirox and Unity, having recently stepped off the board of RegenXBIO (RGNX). Prior to Venrock, Cami was a Managing Director at Versant Ventures for a decade where she led investments in Kythera (KYTH -- acquired by Allergan), Novacardia (acquired by Merck), and ParAllele (acquired by Affymetrix), among others, and was a board observer at several companies including Genomic Health (GHDX), Fluidigm (FLDM), and Syrrx (acquired by Takeda). Before Versant, she was responsible for business development at Tularik and worked in corporate development and strategic marketing at Genzyme and Millennium Predictive Medicine. She also worked at LEK Consulting. Cami studied Biology at Duke and received an MBA from Harvard.
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Chris Adams
PhD, MBA
Founder and Chief Executive Officer
Cydan
Chris has more than 20 years’ experience in ensuring patient impact drives all aspects of the business side of orphan drug development, including business strategy, commercial planning, licensing and mergers and acquisitions.
Before co-founding Cydan in 2013, Chris was president of CMA Biotech Consulting, which primarily served development stage orphan drug companies. He also was chief business officer of FoldRx Pharmaceuticals Inc., where he played an instrumental role in the company being acquired by Pfizer in 2010. He also served as senior vice president of business development for ViaCell Inc., VP of business development for Transkaryotic Therapies Inc., and director of business development for the pharmaceutical division of Ciba-Geigy Limited, in Basel, Switzerland.
He holds a PhD in organic chemistry and a diploma in organic chemistry and biochemistry from the University of Zurich, and an MBA from INSEAD of Fontainebleau, France.
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Dan Bobear
President and Founder
The Patient Experience Project
Dan cofounded the Patient Experience Project (PEP), a marketing and communications agency, with a mission to deliver the best patient experience. PEP’s approach in collaborating with patients and caregivers to co-create solutions for unmet needs has earned it national recognition.
Dan has 25 years of experience in developing strategic solutions for the pharma/ biotech sector. Known for his authentic and results-oriented leadership style, Dan has led both large and small teams of marketing and communications professionals to help achieve success.

Dan collaborates with his internal team and with clients to ensure that patient-focused insights drive brand strategy and shape programs that change what it means—and how it feels—to talk about healthcare. Dan has hosted an annual Rare Disease Day at the Movies.
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Dan Faga
Chief Business Officer
Spark Therapeutics
Dan Faga is the Chief Business Officer of Spark Therapeutics. He has 15 years of biopharma industry experience. Prior to joining Spark Therapeutics, Dan was a managing director at Centerview Partners, where he served as a founding member of Centerview's healthcare advisory practice. Primarily focused in the life sciences sector, Dan has extensive expertise in corporate strategy, mergers and acquisitions, and partnering, and he has advised on more than $50 billion in biopharma transactions. Prior to Centerview, Dan worked at Merrill Lynch in its healthcare investment banking group and as a management consultant in the life sciences practice at PRTM.

Dan earned a B.S. in Engineering from Cornell University and an M.B.A. in Healthcare Management from the Wharton School of the University of Pennsylvania.
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Daniel Levine
Host of RARECast
Principal Levine Media Group
Daniel S. Levine is an award-winning business journalist who has reported on the life sciences, economic development, and business policy issues throughout his 25-year career. He founded Levine Media Group in 2013 to provide strategic communications to life sciences companies. He is host of The Bio Report and RARECast podcasts, a senior fellow at the Center for Medicine in the Public Interest, and a member of the advisory board of the California Biotechnology Council.
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Graeme Martin
Principal
Hatchbox Bioconsulting
Graeme Martin has more than 35 years of multinational Pharma and Biotech R&D experience in Europe, USA and Japan. Currently an independent consultant, he was for 12 years President & CEO of Palo Alto based Takeda Ventures, Inc., where he oversaw strategic investments into 31 early stage companies representing >$100mm under management, serving on the Board of 16 of these companies. Prior to this, he held senior management positions at Telik, Inc., and from 1996-2001, was at Roche Palo Alto, where he was VP of CNS Research USA. Before moving to the USA, Dr. Martin was a senior research manager at GlaxoWellcome and Wellcome Research Laboratories in the UK. He has been involved in three IND submissions and ultimately, the launch of Zomig®, marketed worldwide for the acute treatment of migraine. He received his Bachelor of Science degree in Pharmacology from the University of Bath, UK and his doctorate from University College, London, UK.
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Joe Nolan
President
Dohmen Life Science Services
Joe Nolan is a corporate professional with a proven track record of driving results for over 28 years within the pharmaceutical industry (from large/multi-billion pharma, to start ups, and growth agent to mid-size pharma). Joe has demonstrated success in a range of activities including market access, global licensing, third party alliances, new product development, product launches, orphan disease, specialty pharmacy and strategic business planning. He has therapeutic experience in CNS, GI and Cardiovascular. Joe has Board experience on Illinois Biotechnology Industry Organization (IBIO Vice Chair Human Health), Illinois Science and Math Academy (ISMA), and Naurex Inc.
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John Crowley
Chairman and CEO
Amicus Therapeutics
John F. Crowley is Chairman and CEO of Amicus Therapeutics. John's involvement with biotechnology stems from the 1998 diagnosis of two of his children with Pompe disease—a severe and often fatal neuromuscular disorder. In his drive to find a cure for them, he left his position at Bristol-Myers Squibb and became an entrepreneur as the Co-founder, President and CEO of Novazyme Pharmaceuticals, a biotech start-up conducting research on a new experimental treatment for Pompe disease (which he credits as ultimately saving his children's lives). In 2001, Novazyme was acquired by Genzyme Corporation and John continued to play a lead role in the development of a drug for Pompe disease as Senior Vice President, Genzyme Therapeutics.
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Jeff Sherman
Exec VP, R&D and Chief Medical Officer
Horizon
Jeffrey W. Sherman, MD, FACP, is Chief Medical Officer and Executive Vice President, Research and Development, at Horizon Pharma. He has over 25 years’ of experience in the biopharmaceutical research and development. Jeff received his Bachelor’s degree in Biology from Lake Forest College and Medical degree from the Rosalind Franklin University of Medicine and Science/The Chicago Medical School. He completed an internship, residency, and chief medical residency in internal medicine at Northwestern University. He completed fellowship training in infectious diseases at the UCSF and was a research associate at the Howard Hughes Medical Institute at UCSF. Jeff is an Adjunct Assistant Professor of Medicine at the Northwestern University Feinberg School of Medicine. Jeff is a Past President of the DIA and a former member of the Board of Directors. He also was chairperson of the DIA Annual Meeting, is an inaugural fellow, and serves as the liaison to the FDA CTTI Steering Committee.
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Jeremy Springhorn
Advisor
Road's End
Dr. Springhorn mostly recently served as Partner at Flagship Pioneering until June 2017 where he worked in various strategic and corporate development capacities creating the next generation of startups. Prior to joining Flagship, Dr. Springhorn was one of the original scientists at Alexion. In 2006, Dr. Springhorn moved to business development, leveraging much of his drug development experience looking for rare disease opportunities. Dr. Springhorn led many of the deals for Alexion totaling more than $5.5 billion in deal value. Along with building business development for Alexion, Dr. Springhorn also served as Head of Corporate Strategy as Alexion transitioned from a development firm to a global commercial stage company. Dr Springhorn currently serves on the Board of Overseers for Colby College and is a Director nominee for UniQure. Dr Springhorn holds a PhD in Molecular Biology from LSU Medical Center and did his postdoctoral training at Harvard Medical School.
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Karen Erickson
Associate Executive Director
Alpha-1 Project and Alpha-1 Foundation
Karen Erickson is an Associate Executive Director at the Alpha-1 Foundation. She currently sits on the Scientific Business Advisory Council of The Alpha-1 Project, the Clinical Trials Transformation Initiative steering committee, the American Thoracic Society Public Advisory Round Table, represents the Alpha-1 Foundation in the Rare Lung Disease consortium, and serves as co-chair in the Patient Advocacy Group coalition under NCATS. She has extensive experience in performance management, clinical operations and product/process development from her over fifteen years of employment in the biotechnology arena. She received a bilateral lung transplant in 2013 due to alpha-1 and started her position at the Alpha-1 Foundation shortly after. She couples her patient experience with her past volunteer and biotechnology expertise to collaboratively move innovative clinical trial design and research forward and to support, educate, engage and advocate for the Alpha-1 and rare disease communities.
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Kush Parmar
M.D., Ph.D.
Managing Partner
5AM Ventures
Kush M Parmar, M.D., Ph.D. is a Managing Partner at 5AM Ventures and joined in 2010 from Harvard Medical School, where he was an NIH-sponsored M.D./Ph.D. Physician Scientist Fellow in the Harvard-MIT Health Sciences and Technology Program. Dr. Parmar serves as a Director on the Boards of Arvinas, Audentes (NASDAQ: BOLD), Homology and scPharmaceuticals. He previously served on the boards of Novira (acquired by J&J), Envoy (acquired by Takeda), Achaogen (NASDAQ: AKAO) and Pulmatrix (NASDAQ: PULM). Dr. Parmar serves on the Scientific Advisory Council of Princeton University’s Department of Molecular Biology and for the Grace Wilsey Foundation. He is a Fellow of the Society of Kauffman Fellows. He holds an A.B. in Molecular Biology and Medieval Studies from Princeton University, a PhD in Experimental Pathology from Harvard University, and an M.D. from Harvard Medical School.
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Matthew Howes
Executive Vice President, Strategy & Growth
PALIO, an inVentiv Health Company
Matthew Howes has been instrumental reframing how companies reach and engage patients and physicians and has a passion for working with organizations on the forefront of healthcare. His vision is informed by over 20 years of experience developing commercialization strategies for businesses that serve more than 100 million people every month—and generates more than a billion dollars in revenue every year. He is an active thought leader in the healthcare advertising industry, and has been recognized by PM360 as a 2016 ELITE Award Winner for Transformational Leadership. Matthew has served as a juror for the CLIO Healthcare Awards and is a regular speaker at industry events.
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Melanie Ivarsson
VP, Head of Clinical Programs
Shire
Melanie Ivarsson is currently Vice President, Head of Clinical Programs at Shire where she has accountability for the delivery of clinical research programs in phase I-IV. Prior to joining Shire, Melanie was at Pfizer for 9 years, most recently as Head of Clinical Operations for the Clinical Research organization. Melanie has a background in early clinical development, with previous roles as Head of Clinical Operations for early development at Pfizer UK, and 8 years at Eil Lilly in the Clinical Pharmacology group. She has a PhD in Neuroscience and a BSc in Physiology from the University of Bristol, UK and completed post-doctoral research at the University of New York, USA and the University of Lund, Sweden.
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Michael Broxon
VP and Head of R&D Business Development
Takeda Pharmaceuticals
Mike Broxson is currently VP and Head of Research and Development Business Development for Takeda Pharmaceuticals and is based in Cambridge, MA. Mr. Broxson has served in diverse leadership roles across finance, strategy, and business development at Takeda for 16 years. He has lead or played significant roles in over 70 transactions including the acquisitions of Millennium Pharmaceuticals, Inc., Nycomed, and Envoy Therapeutics, and more recently established Takeda’ externalization practice to spin out Takeda assets through new ventures and innovative alliances. Mike holds degrees in Economics (BS) and Public Health (MSPH) from Tulane, has an MBA from University of Chicago Booth School of Business, and is a CFA Charter holder.
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Michael Pistone
Director of Business Development & Marketing
Cincinnati Children's Hospital Medical Center
Mike Pistone is passionate about innovation and collaboration. In his current role, he leads the strategic marketing and business development for Cincinnati Children's innovation and commercialization efforts, focused on advancing discoveries to the market where they can positively impact patient lives and the healthcare system. This includes managing a robust portfolio of therapeutics, diagnostics, software/HIT, and medical devices and partnering with pharmaceutical, biotech, medtech, and venture funds.
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Molly Harper
VP Commercial Development
Akcea Therapeutics
Molly Harper is an experienced commercial leader with nearly 20 years of experience in the life sciences industry, including expertise in pre-launch market development and commercial operations for biopharmaceuticals. Ms. Harper joined Akcea as Vice President, Commercial Development, in April 2015 from the Rare Disease division of Genzyme, where she was most recently Senior Director and Head of US Endocrinology, responsible for all commercial functions of an orphan-disease biological injectable and the management of a molecular diagnostic co-promotion with Veracyte, Inc. Her earlier roles at Genzyme included Global and US Marketing leads for the Endocrine and Cardiovascular businesses. Prior to Genzyme, Ms. Harper was with Merck & Co., where she held positions in the Atherosclerosis and Acute Care franchises across marketing management, market research, and sales.
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Morrie Ruffin
Managing Director
Alliance for Regenerative Medicine
Morrie has more than 20 years of experience in the Biotech and Healthcare industries. He is a founder and managing director of the Alliance for Regenerative Medicine (ARM), the global organization representing the interests of the regenerative medicine community. Morrie is also the managing partner of Adjuvant Partners, a boutique regenerative medicine and advanced therapies business consulting firm. Prior to joining Adjuvant Partners, he was the chief executive officer of LifeTech Innovations, LLC a business development consulting firm based in Bethesda, MD. Prior to his position at LTI, Mr. Ruffin was executive vice president of Capital Formation and Business Development at the Biotechnology Industry Organization (BIO), the largest trade organization representing the biotech and drug development industries.
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Neil Kumar
CEO
BridgeBio
Dr. Neil Kumar, Ph.D., is the CEO and Founder of BridgeBio Pharma, LLC, a company focused on the development of novel therapies for rare genetic disorders. Prior to that, he was a principal at Third Rock Ventures where he supported and managed various portfolio companies in addition to focusing on new company formation and due diligence. He also held the role of Vice President, Business Development and Operations for MyoKardia. Prior to joining Third Rock, Dr. Kumar was an associate principal at McKinsey & Company, where he developed strategies for pharmaceutical and medical device companies and helped lead McKinsey’s personalized medicine efforts. Before joining McKinsey, Dr. Kumar was involved in the formation of a gene chip startup and was a technical consultant for AstraZeneca’s pathway signaling group. Dr. Kumar is the author of several peer-reviewed papers in the fields of oncology and systems biology.
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Tanya Zaremba Fischer
MD, PhD
Global Project Head of Early Development for Parkinson’s Disease/Movement Disorders
Sanofi Genzyme
Tanya Zaremba Fischer is the Global Project Head of Early Development for Parkinson’s Disease/Movement Disorders at Sanofi, Inc., based in Cambridge, MA. She oversees the clinical development, at different stages in development, of Parkinson’s disease and other rare CNS Neurology assets. Dr. Fischer is an M.D. with neurology residency training at Yale University and a Ph.D. in Neuroscience and Cell Biology from Rutgers University. Since completion of her neurology residency training in 2006, she pursued both clinical and research work as a faculty member of the Yale University, School of Medicine’s Department of Neurology, where she continues to maintain an academic appointment as Adjunct Professor. Dr. Fischer has been successfully able to integrate her dual training in neuroscience and clinical neurology into translational research of chronic neuropathic pain disorders. She has authored many scientific papers and has been the recipient of several awards and honors.
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Toby Freyman
Director, Genetic Diseases and Hematology Business Development
Shire
Toby Freyman has a broad base of experience from working across the biopharmaceutical, pharmaceutical, and medical device industries over the past fifteen years. In his current role as Director, Business Development at Shire, he focuses on identifying and evaluating business opportunities in the genetic disease space and leading negotiations on transactions. Prior to joining Shire, Dr. Freyman had roles as a Vice President at Arsenal Medical, Inc. and as an Associate Principal at Charles River Associates, where he led the development of product launch, pricing, and reimbursement strategies for a number of biopharmaceutical products. He received a Bachelor of Science degree from Syracuse University and a Ph.D. from the Massachusetts Institute of Technology, where he was awarded a National Institutes of Health Graduate Fellowship through a joint program with MIT and Harvard University.
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Vern Norviel
Partner
Wilson Sonsini Goodrich and Rosati
Vern Norviel is a partner at Wilson Sonsini Goodrich & Rosati, where he leads the patents and innovation counseling practice. He represents a wide variety of companies, as well as venture capital firms, in areas such as therapeutics, diagnostics, nanotechnology, genomics, proteomics, and personalized medicine. In fact, Vern's interest in the field of personalized medicine prompted him to become the first attorney to have had his or her entire genome sequenced and made available in a public database. Before joining the firm in 2003, Vern was the general counsel and corporate secretary of Perlegen Sciences, Inc. Previously, as senior vice president and general counsel, he was an early employee of Affymetrix. Vern is a member of the Board of Wilson Sonsini and an adjunct professor at UC Berkeley.
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