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March 26-28, 2019 | Boston, MA

Speakers

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Derek Adams
Chief Technology & Manufacturing Officer
Bluebird bio

Derek Adams, Ph.D., joined bluebird bio in March 2017 as chief technology and manufacturing officer. Prior to joining bluebird, Derek was the senior vice president of CMC at Evelo Biosciences. At Evelo, Derek established the initial process development function and supply chain for clinical studies and drove strategy for product development. Prior to his time at Evelo, he served as vice president of technical and Strategic Product Development at Alexion Pharmaceuticals, where his responsibilities included developing and supporting all manufacturing processes globally as well as global clinical supply chain. His Alexion career also included roles as Plant Manager for Alexion’s Rhode Island Manufacturing facility and head of upstream process development. Derek began his career in process technical support for live virus vaccine production at Merck & Co, Inc. Derek holds a Ph.D. in Chemical Engineering from the University of Minnesota and a B.S. in Chemical Engineering with High Distinction from Worcester Polytechnic Institute (WPI).

Sean Ainsworth
CEO
Immusoft

Sean Ainsworth has twenty years of experience in pharmaceuticals and biotechnology. Prior to founding RetroSense Therapeutics (acquired by Allergan in 2016), Sean had deep involvement in the launch of two earlier startup companies, Compendia Bioscience, Inc. and GeneVivo, LLC where he assisted in developing business models, licensing technologies, building management teams, securing capital and first customers. Compendia BioScience was acquired by Life Technologies in 2012, providing a successful exit for both founders and investors. Sean's experience also includes research at Medical Biology Institute (now Avanir Pharmaceuticals developers of Abreva, the leading cold sore medication) in San Diego, intellectual property at Koyama and Associates in Tokyo, and international corporate development consulting at The Mattson Jack Group in St. Louis, MO. Sean also founded Ainsworth BioConsulting to provide licensing, strategic planning, and business planning services to the life science and entrepreneurial community. His clients included large pharma, small biotechs, universities, CROs, and venture investment funds. He has worked with clients at all stages of development. Sean holds a BS in Microbiology from University of California, San Diego and an MBA in strategy and finance from Washington University in St. Louis.

Meisam Bakhshayeshi
Head of Purification Process Development
Biogen

Robert Baffi
EVP
BioMarin

Robert A. Baffi, Ph.D., joined BioMarin in May 2000 and currently serves as Executive Vice President of Technical Operations, responsible for overseeing manufacturing, process development, quality, and analytical chemistry departments. From 1986 to 2000, Dr. Baffi served in a number of increasingly responsible positions at Genentech, Inc., primarily in the functional area of quality control. Prior to Genentech, Dr. Baffi worked for Cooper BioMedical as a Research Scientist and at Becton Dickinson Research Center as a Post-Doctoral Fellow. Dr. Baffi has contributed to more than 20 regulatory submissions for product approval in the United States and Europe and to more than 50 regulatory submissions for investigational new drug testing. He serves on the board for the National Institute for Bioprocessing Research & Training. Dr. Baffi received a Ph.D., M. Phil and a B.S. in biochemistry from the City University of New York and an M.B.A. from Regis University.

Day Two

Thursday March 28, 2019

09.30 | Challenges and Considerations for Large Scale Manufacture of an AAV-Based Gene Therapy Product

Brian Bronk
Head of External Innovation & Rare Disease
Sanofi

Wei-Chiang Chen
Senior Scientist
Solid Biosciences

Reed Clark
SVP
Ultragenyx

Reed Clark, Ph.D. is a veteran molecular virologist and gene therapist with extensive experience in preclinical and translational adeno-associated virus (AAV) gene therapy product development. Prior to joining Ultragenyx, Dr. Clark served as head of Pharmaceutical Development at Dimension Therapeutics moving forward 4 AAV based programs to IND.  Prior to an Industry role, he was Director of the cGMP Clinical Manufacturing Facility and Preclinical Vector Core, and the Associate Center Director at the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital with a focus on utilizing AAV biologics for rare diseases targeting metabolic and neuromuscular indications from pre-clinical proof of concept through early phase clinical trials for SMA, Duchenne and Limb Girdle muscular dystrophies.  Dr. Clark also served on the faculty of the Ohio State University as a professor in the Department of Pediatrics, College of Medicine. His group made seminal discoveries in scalable engineered mammalian cell lines for AAV production, FPLC purification of AAV vectors and improved AAV titration approaches using qPCR and wt-AAV free reporter cell lines. He has served as a member on the both the AAV2 and AAV8 reference standard committees and multiple NIH and private peer review study sections. Dr. Clark received his B.S. in genetics and Ph.D. in molecular genetics from the Ohio State University, and was a post-doctoral fellow in gene therapy at Nationwide Children’s Hospital.

Jason Comander
Assistant Professor
Harvard Medical School

Dr. Jason Comander is a member of the Mass. Eye and Ear Inherited Retinal Disorders Service and Retina Service, where he specializes in diagnosing and treating inherited retinal diseases. He is also a member of the Ocular Genomics Institute, the Berman-Gund Laboratory for Retinal Degenerations, and the Howe Laboratory.   Dr. Comander is a graduate of Harvard Medical School (HMS), where he earned his medical degree and his PhD in Experimental Pathology. He completed a clinical fellowship at Brigham and Women's Hospital, followed by an ophthalmology residency in Harvard Ophthalmology.   As a clinician-scientist, Dr. Comander's research primarily focuses on the genetics and treatment of inherited retinal diseases. He has studied the genetic basis of distinct forms of retinitis pigmentosa, investigated methods to assay the functional significance of human mutations, and tested techniques for retinal gene therapy in primates.  He also is an investigator and surgeon for a number of ongoing clinical trials for patients with inherited retinal disorders.   A driven and industrious clinician with a demonstrated aptitude for research, Dr. Comander was selected to attend the Heed Foundation Retreat for Academic Ophthalmology in 2009. In 2012, he was recognized as an up-and-coming clinician scientist when he received the highly competitive K12 Mentored Clinical Scientist Development Program Award from the National Institutes of Health, to support his research on inherited retinal disorders. He also holds a Career Development Award from Research to Prevent Blindness and an Enhanced Career Development Award from the Foundation Fighting Blindness.

Day One

Wednesday March 27, 2019

17.00 | Keynote: Making Medical History – First-Hand Insights into the First FDA-Approved Gene Therapy Procedure for Inherited Disease

Omar Dabbous
VP, Global HEOR & RWE
AveXis

Accountable for global health economics and outcomes research and real-world evidence. Interact with Payers and Commercial teams to design and implement HEOR & RWE strategies to support AveXis products. Be the company expert in building and translating clinical and observational data into economic and societal value. Co-chair the value and access AveXis process to ensure optimal product value and reimbursement. Responsible for generating robust and relevant evidence to support payer and healthcare professional decision-making. Lead gene therapy forum to advance the science of HEOR & RWE to ensure early market access to gene replacement therapy.

Troy Dale
Global Head, Commercial Strategic Partnerships
Novartis

Olivier Danos
CSO
REGENXBIO

Olivier Danos is Senior Vice President and Chief Scientific Officer at REGENXBIO. He is a pioneer in the field of gene therapy, and has dedicated his career to advancing the use of this technology to develop life-saving therapies for patients.  Olivier joined REGENXBIO in 2017 from Biogen where he was a Senior Vice President in charge of Cell and Gene Therapy. Over the past twenty years, he has played leadership roles in cell and gene therapy as Director of the Gene Therapy Consortium of the University College of London, at the Necker Hospital – Enfants Malades in Paris, as Chief Scientific Officer of Genethon and as senior director of research at Somatix Therapy Corporation.  He has held senior research positions in France at the Centre National de la Recherche Scientifique and at the  Institut Pasteur.  Olivier is the former President and a founding member of the European Society of Gene and Cell Therapy. Olivier received a Master’s in Genetics and Molecular Biology at University of Paris Orsay, and his Ph.D. at the Pasteur Institute and University of Paris Diderot.

Day Two

Thursday March 28, 2019

15.30 | Using the NAV Platform of AAV Vectors for Gene Therapy in the Eye: The Case of Wet AMD

Eric David
CEO-in-Residence
BridgeBio

Eric Michael David is CEO of gene therapy at BridgeBio. He was a co-founder of Organovo, where he most recently served as Chief Strategy Officer and Executive Vice President of Preclinical Development. Prior to Organovo, Dr. David was an Associate Partner at McKinsey & Co, where he served stakeholders across the healthcare spectrum including biopharmaceutical companies, private equity firms, large public and private healthcare systems, academic medical centers, and foundations on topics ranging from operations to M&A and R&D strategy.  He was also Assistant Professor at The Rogosin Institute, adjunct faculty at The Rockefeller University, and a lecturer in Medicine at Weil Cornell Medical College. Dr. David received his clinical training in internal medicine at New York Presbyterian Hospital / Weill Cornell, his  MD from Columbia University College of Physicians and Surgeons, his JD from Columbia Law School and his BA in physics and fine arts from Amherst College. He is licensed to practice medicine in CA and NY, and admitted to the Bar in NY.

Jill Dolgin
Head of Patient Advocacy
AGTC

Jill Dolgin, PharmD is a healthcare professional with over 20 years of global biopharmaceutical experience in Medical Affairs, Patient and Professional Advocacy, and Public Policy. She has specific expertise in leading change, tackling complex and challenging problems and working with a diverse array of partners specializing in rare diseases in inherited retinal dystrophies, hematology/oncology, pulmonary/infectious diseases, and cardiovascular/metabolic diseases. Following years in academia, Jill began her pharmaceutical career at GlaxoSmithKline in roles of increasing responsibility in Medical Affairs, Corporate Communications, and Government Affairs & Public Policy for the Oncology, Cardiovascular, and Metabolic Franchises. She subsequently joined Daiichi Sankyo to spearhead the development of a new advocacy & professional relations function.  She also was responsible for developing and operationalizing internal policies for Corporate Philanthropy and employee engagement; followed by similar US and global roles at Onyx and Insmed.   Jill currently serves as the Head of Patient Advocacy at AGTC, Applied Genetic Technologies Corp, a biotech focused on early development of gene therapies for several rare ophthalmologic inherited conditions.

Scott Dorfman
CEO
Odylia Therapeutics

Scott Dorfman is president and CEO of Odylia Therapeutics, a non-profit biotech company whose mission is to facilitate the translation of rare disease with proof-of-concept from the lab into clinical trials. Prior to Odylia, Mr. Dorfman founded and was Chairman, & CEO of Innotrac Corporation (NASDAQ: INOC), which was a full-service provider of eCommerce technology, fulfillment, and call center services. Founded in 1984, Innotrac employed over 2,500 employees with $250 million in revenues. The company was taken private by Mr. Dorfman along with Sterling Capital and Longview Capital in 2014 and combined with eBay Enterprises (division of eBay) to form Radial Corporation. Radial Corporation is the one of the world’s leading eCommerce service companies with revenues of over $1 Billion and with 30 locations in North America, Europe and Asia. Mr. Dorfman served on the Board of Visitors for Emory University and is a past member of the Board of Directors of the Partnership against Domestic Violence (PADV). In addition to serving on the Radial board, he has also sat on the Board of Directors for Chatham Capital, Return.com and Market Velocity, Inc. Mr. Dorfman currently sits on the Board of Dropoff Inc., Complemar Corporation, Fulcrum Ventures, Odylia Therapeutics and Usher 2020 Foundation. Mr. Dorfman received a BBA from The University of Georgia in 1979.

Miguel Esteves
Associate Professor
UMass Medical School

Miguel Sena-Esteves, PhD, studied Biochemistry at the Universidade do Porto (Portugal) and earned his PhD from the same University. His graduate studies focused on gene therapy for GM1 gangliosidosis and vector design. He conducted his post-doctoral training at Children’s Hospital of Philadelphia where he continued his work on lysosomal storage diseases (LSDs) and in vivo gene delivery. In 2004, he became an Assistant Professor at MGH/HMS in Boston where he started using AAV vectors for CNS gene therapy in LSDs and brain tumors. In 2009, he became Associate Professor at UMass Medical School. His laboratory focuses on AAV engineering for CNS gene transfer and therapeutic applications in neurodegenerative diseases with emphasis on LSDs (e.g., Tay-Sachs and  Sandhoff diseases, GM1 gangliosidosis), HD, ALS and brain tumors. He is part of a team of researchers and physicians developing AAV gene therapies for TSD, SD and GM1, with clinical trials expected in 2019.

Day Two

Thursday March 28, 2019

16.00 | Mechanistic Insights & Enhancements Toward Effective CNS Transduction After Vascular Delivery

Alison Finger
CCO
bluebird bio

Alison Finger has served as bluebird bio’s chief commercial officer since February 2017. She joined bluebird in August of 2015 as our senior vice president, marketing and product launch. Prior to joining bluebird, Alison spent 21 years at Bristol-Myers Squibb (BMS) where her career began in consumer medicines and progressed to global leadership roles in hepatitis, HIV/AIDs, hematology and neurodegenerative diseases. She has launched global, regional and national brands in hepatitis, HIV/AIDs, diabetes, rheumatoid arthritis, oncology and migraine/pain, and has led teams in consumer medicines, U.S. operations and global commercial. Alison was also responsible for building and embedding marketing capabilities throughout the company as BMS’ Executive Sponsor for Marketing Excellence. Alison is currently a member of the board of directors of the Alliance for Regenerative Medicine Foundation which fosters education and research to support the development of transformational treatments and cures for patients worldwide. She holds a B.A. in English writing from Saint Lawrence University and an MBA from the Fuqua School of Business at Duke University.

Day One

Wednesday March 27, 2019

08.30 | Industry Leaders Panel: Pioneering the Next Generation of Gene Therapies

Jon Garen
CBO
uniQure

Mr. Jonathan Garen joined uniQure as Chief Business Officer in July 2016.

Most recently, Mr. Garen served as Chief Business Officer at Syros Pharmaceuticals, where he was responsible for business transactions including partnering Syros’ technology platform and drug assets, and bringing in products to enhance and accelerate its pipeline. Prior to joining Syros, Mr. Garen was the Assistant Vice President of Business Development at Forest Laboratories from 2003 to 2014, and subsequently, Actavis, plc until 2015 following its acquisition of Forest Laboratories. At Forest Laboratories and Actavis, Mr. Garen was responsible for numerous acquisitions and license agreements to build the companies' pipeline in all its focus therapeutic areas, and led a team of business development professionals. Earlier in his career, Mr. Garen was Director of Global Licensing with Pharmacia Corporation and a Founder and Vice President of Technology Exchange, Inc., in New York, NY.

Mr. Garen holds a Master of Environmental Science degree from Yale University and a Bachelor of Science degree in Physics from the Massachusetts Institute of Technology.

Marianne Hamilton Lopez
Research Director
Duke-Margolis Center for Health Policy

Kathleen Hehir
Head, Gene Therapy Clinical Manufacturing
Sanofi Genzyme

Kathy High
President and Head of R&D
Spark Therapeutics

Dr. Katherine High is Co-founder, President, and Head of R&D at Spark Therapeutics. Clinically trained in internal medicine and hematology, and previously the Founding Director of the Center for Cellular and Molecular Therapeutics at the Children's Hospital of Philadelphia, Dr. High joined Spark in 2014 and has led the company's efforts to discover, develop, and deliver gene therapies for genetic disease. The Spark team has developed human proof-of-concept for AAV-mediated gene delivery in both the retina and the liver, and in May 2017 filed the first marketing application for a gene therapy for genetic disease in the US. Spark has received breakthrough therapy designation for two programs, one for voretigene neparvovec to treat autosomal recessive mutations in RPE65, and the other for SPK-9001 for hemophilia B. 

Day One

Wednesday March 27, 2019

08.30 | Industry Leaders Panel: Pioneering the Next Generation of Gene Therapies

08.00 | Keynote: Turning Genes into Cures

Brian Kaspar
CSO, SVP & Scientific Founder
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 and principal investigator at Nationwide Children's Hospital. 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

Wednesday March 27, 2019

08.30 | Industry Leaders Panel: Pioneering the Next Generation of Gene Therapies

Rune Kjeken
Scientific Director, Advanced Therapies, CAT
EMA

Dr. Kjeken is a Scientific Director at the Norwegian Medicine Agency specialising in preclinical and clinical assessment advanced therapies and biological medicines. He is a member of the European Medicines Agency’s Committee for Advanced Therapy (CAT) and the Scientific Advice Working Party (SWAP), and was previously a member of EMA’s Gene Therapy Working Party (GTWP). Dr. Kjeken was previously Executive Director for R&D at Inovio Biomedical Company, San Diego, were he was heading development of therapeutic DNA vaccines for cancer and Infectious diseases and developing methods and medical devices for non-viral gene delivery. Dr. Kjeken is a graduate from the University of Oslo and received his Post-doctoral training at the European Molecular Biology Laboratory (EMBL) in Heidelberg.

Melissa Kotterman
Senior Director, Head of Discovery & Engineering,
4D Molecular Therapeutics

David Knop
Executive Director, Process Development
AGTC

Dr. David R. Knop is the Executive Director, Process Development at Applied Genetic Technologies Corporation (AGTC).  He has overseen all CMC-related activities for the company’s proprietary herpes-assisted vector expansion (HAVE) for AAV production over the last 17 years.  He has executed technology transfer to contract manufacturing organizations, academic and corporate partners resulting in seven IND filings.  He has authored numerous manuscripts, presentations, and posters encompassing the production, processing, and purification of rHSV and rAAV vectors for use in gene therapy applications.  Previously, Dr. Knop developed biocatalytic routes to high-value pharmaceutical intermediates employed in anti-viral compound syntheses, which resulted in multiple issued patents being licensed to large pharma.

Nerissa Kreher
CMO
AVROBIO

Rajendra Kumar-Singh
Founder
Hemera Biosciences

Rajendra Kumar-Singh is a tenured Professor in the Department of Developmental, Molecular and Chemical Biology at Tufts University School of Medicine.  He is also the Director of the Program in Genetics at Tufts University and a Co Founder and Vice President of Hemera Biosciences Inc.  – a start up company focused on the development of a gene therapy for the dry form of age related macular degeneration- the most common cause of blindness in the elderly.  Rajendra obtained his PhD at the University of Dublin, Ireland in the field of ocular genetics, after which he completed postdoctoral training at the University of Michigan in the field of gene therapy.  Rajendra then held faculty positions at the University of California, Los Angeles and the University of Utah prior to joining Tufts University.  His research is funded by the Department of Defense, National Institutes of Health and several private foundations.   Rajendra’s research has led to the development of an inhibitor of the membrane attack complex (MAC) that is thought to play a role in the pathogenesis of dry AMD and other complement associated disorders.

Ralph Laufer
CSO
Lysogene

Daniel Leonard
Director, Global Patient Advocacy
uniQure

Daniel Leonard is the Director of Global Patient Advocacy at uniQure, a pioneering company in gene therapy. In this role Dan works primarily with hemophilia and Huntington’s disease organizations in the US and abroad. In this role Dan serves as an ambassador to the patient community, but also as an internal advocate for patients’ interests. Prior to joining uniQure, Dan spent 16 years at Genzyme, a company known as a leader in patient centricity. Prior to transitioning to Patient Advocacy in 2011, Dan spent 11 years working in Regulatory Affairs, managing a team responsible for registering enzyme replacement therapies and small molecules throughout Asia and the Middle East. With a Master’s Degree in Japanese Language and Literature, Dan’s primary focus was registering products in Japan. Dan is active in the non-profit world, served on the Board of Directors of an educational non-profit in Cambridge, MA and developed and launched an award-winning community engagement program bringing students into corporate labs to teach them the science of biotechnology.

Day One

Wednesday March 27, 2019

17.30 | Panel Discussion: Patient Advocacy in the Gene Therapy Space: A Case Study in Hemophilia

Janet Lynch Lambert
CEO
Alliance for Regenerative Medicine

Janet Lynch Lambert joined ARM in July of 2017 as the organization’s first CEO. With more than 25 years in public and private sector management, Janet is an accomplished government relations and business professional. She previously served as the Acting Head of Engagement for the All of Us Research Program at the National Institutes of Health and as head of the Outreach Office in the Office of the NIH Director. Prior to joining NIH, she was Vice President of Government Relations and head of the Washington office of Life Technologies.  Her experience also includes roles at GE and InforMax and legislative and staff leadership positions in the U.S. Senate and House of Representatives.  Janet received her MBA in International Business from Georgetown University and her B.A. in Political Science from Stanford University.

Day Two

Thursday March 28, 2019

08.30 | Setting Out the Gene Therapy Landscape: Where Are We Now?

Peter Marks
Director, CBER
FDA

Peter Marks, M.D., Ph.D.  is the director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration. The center is responsible for assuring the safety and effectiveness of biological products, including vaccines, allergenic products, blood and blood products, and cellular, tissue, and gene therapies. Dr. Marks and center staff are committed to facilitating the development of biological products and providing oversight throughout the product life cycle.  Examples of these activities include: ·         reviewing and providing advice during product development ·         evaluating applications and making approval decisions based on safety and effectiveness data ·         monitoring the safety of biological products ·         conducting research that supports product development and characterization   "The center regulates and does research on complex biologic products that touch people's lives on a daily basis," says Dr. Marks. "Many of the products that we regulate are vital for promoting and protecting the public health, including vaccines, blood products, and tissues for transplantation. I'm very proud to lead a team of highly committed individuals whose efforts help to ensure the timely development of safe and effective products to meet important medical needs." Dr. Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University. Following this, he completed an Internal Medicine residency and Hematology/Medical Oncology fellowship at Brigham and Women's Hospital in Boston, where he subsequently joined the attending staff as a clinician-scientist and eventually served as Clinical Director of Hematology.
He then moved on to work for several years in the pharmaceutical industry on the clinical development of hematology and oncology products prior to returning to academic medicine at Yale University where he led the Adult Leukemia Service and served as Chief Clinical Officer of Smilow Cancer Hospital. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in 2016.  Dr. Marks is board certified in internal medicine, hematology and medical oncology, and is a Fellow of the American College of Physicians.

Day Two

Thursday March 28, 2019

09.00 | FDA’s Efforts to the Development and Availability of Gene Therapy Products

Chris Mason
CSO
AVROBIO

Chris is a clinician-scientist with over 20 years of cell and gene therapy experience including Full Professor of Regenerative Medicine at University College London. He has a multidisciplinary track record spanning R&D, clinical medicine, bioprocessing, regulation, healthcare economics, reimbursement and commercialization. Chris is co-founder of the London Regenerative Medicine Network, and a founding member of the UK-Israel Science Council. Current SAB appointments include; public and private companies, the UK Cell Therapy Catapult and the Canadian Centre for the Commercialization of Regenerative Medicine. Chris is Senior Editor of the journals ‘Cell and Gene Therapy Insights’ and ‘Regenerative Medicine’.

Day One

Wednesday March 27, 2019

18.00 | Chairs’ Closing Remarks & Scientific Poster Session Resumes

07.50 | Chair’s Opening Remarks

Day Two

Thursday March 28, 2019

16.30 | Chairs’ Closing Remarks

08.20 | Chair’s Opening Remarks

Bill Martin
CCO & VP
Accredo – An Express Scripts Company

Bill Martin, Vice President, Pharma Strategy and Account Management, leads a team that supports the long-term strategic needs, as well as the day-to-day needs of Express Scripts pharmaceutical and biotech clients. His team manages contractual relationships,  data reporting and implementation of new programs. Bill has more than 20 years of experience in the healthcare industry and has held various positions of leadership in general management, marketing, medical education, sales and product development.

Prior to his current role, Bill was vice president, Business Development & Strategy, at Accredo. His key areas of interest include optimizing complex distribution channels, rare disease strategies and the emerging biosimilars pathway.

Kevin Mayo
Global Head, Market Access & New Products
PTC Therapeutics

Dr. Kevin Walker Mayo is a Head of Global Market Access New Products for PTC Therapeutics.  Prior to joining PTC , Kevin founded Value Genome LLC. Kevin was previously Global Hemophilia Franchise Market Access and Patient Advocacy Head for Baxalta (now Shire). At Baxalta, Kevin lead a team of international market access and health economics experts responsible for negotiating with government and commercial payers to obtain pricing and formulary access for launching products. His responsibilities included conducting payer research and advisory boards to develop commercial strategies, developing and training his team upon value communication messages and negotiation tools and providing coordination for all health economics and outcomes research, pricing and reimbursement, tender management, advocacy and government affairs activities for a $6 billion portfolio.  Earlier in his career Kevin gained industry and consulting expertise in senior leadership roles with The Access Partnership, Endo Health Solutions, Bridgehead International, Daiichi Sankyo, Novartis Pharma AG and Pharmacia Corporation. Kevin completed a Master’s degree in Neuropsychology from the University of Memphis and a Doctoral degree in Pharmaceutical Marketing and Administration from the University of Louisiana at Monroe.  More recently Kevin augmented his business education by completing an Executive Juris Doctorate in Health Law and Policy from Concord Law School.  He has served on the adjunct faculties for the Mayes College of Healthcare Business and Policy (University of the Sciences) and the College of Population Health (Thomas Jefferson University) and is a frequent presenter at international market access conferences.  Kevin received a Distinguished Service Award from the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) for his contribution to the society.Kevin completed a Master’s degree in Neuropsychology and a Doctoral degree in Pharmaceutical Marketing and Administration.  More recently Kevin augmented his business education by completing an Executive Juris Doctorate in Health Law and Policy.  He has served on the adjunct faculties for the Mayes College of Healthcare Business and Policy (University of the Sciences) and the College of Population Health (Thomas Jefferson University) and is a frequent presenter at international market access conferences.  Kevin received a Distinguish Service Award from the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) for his contribution to the society.

Ilan McNamara
Senior Director, Global Regulatory Affairs
Prevail Therapeutics

Tim Miller
President & CEO
Abeona Therapeutics

Tim Miller, Ph.D. is Co-Founder, President and Chief Scientific Officer of Abeona Therapeutics. He has over 20 years of business development, scientific research product development and clinical operations expertise, with a focus on transitioning novel biotherapeutics through pre-clinical phases and into late Phase human clinical trials. Dr. Miller was President & CEO of Red5 Pharmaceuticals from 2013 until 2015 and was CEO-in-Residence at BioEnterprise Inc in 2015. He was Senior Director of Product Development at SironRX Therapeutics from 2010 to 2013. Between 1996 and 2010 Dr. Miller held various positions at several biotech companies focusing on gene therapy and regenerative medicine. Dr. Miller earned his Ph.D. in Pharmacology with a focus on Gene therapy/Cystic Fibrosis from Case Western University. He also holds a B.S. in Biology and M.S. in Molecular Biology from John Carroll University (Cleveland, OH).   As a serial entrepreneur, he has managed all aspects of research and development, manufacturing of biologics, and clinical program start-up in both public and private companies, with direct experience engaging FDA, EMA, and NIH advisory agencies on multiple clinical trial submissions. During his career, he has contributed to multiple patent applications, managed intellectual property, and published research in several internationally recognized journals. He has a passion for supporting patient advocacy and developing potential therapeutics for patients with rare disease.
 

Carl Morris
CSO
Solid Biosciences

Christian Mueller
Associate Professor
UMass Medical School

Knut Niss
CTO
Mustang Bio

Dr. Niss has served as Chief Technology Officer since March 2018. Dr. Niss joined Mustang in March 2017 as Vice President of Operations, where he initiated and oversees the establishment of Mustang’s cell therapy manufacturing facility. Prior to Mustang, Dr. Niss was Cell Therapy Asset Leader at Biogen, where he oversaw CMC-related activities for gene-edited hematopoietic stem cell and lentiviral gene therapy programs for sickle cell disease and hemophilia, respectively. Earlier in his career, Dr. Niss was Senior Technical Project Leader at Novartis’ cell therapy manufacturing facility in Morris Plains, New Jersey, where he directed the transfer and implementation of the CTL019 process from Penn to Novartis. He also served as Senior R&D Program Manager at EMD Millipore, where he established processes for the large-scale expansion of adult and pluripotent stem cells. Dr. Niss began his career in senior research positions in Pfizer’s Regenerative Medicine and Immunology groups. He holds a Ph.D. in molecular biology from Humboldt University of Berlin, and an M.S. in microbiology from the University of Göttingen in Germany. Dr. Niss completed his postdoctoral research at Boston Children’s Hospital and the Dana-Farber Cancer Institute.

Palani Palaniappan
Head, Technical Operations & Andover Site
Sarepta Therapeutics

Mark Pykett
Chief Innovation Officer
PTC Therapeutics

Dr. Pykett joined PTC in August 2018 as Chief Innovation Officer.  Previously, he was the CEO of Agilis Biotherapeutics. Prior to Agilis, he served as CEO of Navidea Biopharmaceuticals, President and COO of Alseres Pharmaceuticals, CEO of Cytomatrix and President of Cygenics. Dr. Pykett has also served as a Director of several public and private companies and the not-for-profit organization HealthBuilders, developing health infrastructure in central Africa.  Dr. Pykett received a B.A. degree from Amherst College, a V.M.D. and Ph.D. from the University of Pennsylvania, and an M.B.A. from Northeastern University, and completed post-doctoral fellowships at the University of Pennsylvania and Harvard University.

Satinder Rawat
Licensing Officer
UMass Medical School

Satinder manages a diverse portfolio of early and clinical stage intellectual properties. This portfolio has helped launch several gene therapy companies including Voyager Therapeutics, Generation Bio (non-viral), Apic Bio and ASPA therapeutics. Agalimmune, (now BiolineRx, oncology,), Acworth pharmaceuticals (oncology), Fulcrum Therapeutics (Rare disease) and Bacainn Therapeutics (Inflammation) were also founded around this portfolio technologies. His portfolio technologies led to development SpinrazaTM, the first approved therapeutic for Spinal Muscular Atrophy (SMA); AGI-134, which entered Phase 1 trial for Solid tumors in 2018 and several clinical stage gene therapies (two dosed in rare disease patients via expanded access). He is a member of Executive Board of M2D2 (Mass Medical Device Development), a startup incubator located at UMASS. Satinder received his bachelor’s from Delhi University, his MS and PhD from Jawaharlal Nehru University (JNU), and his MBA from the Isenberg School of Management, UMASS, Amherst.

Salvador Rico
VP, Clinical Development
Audentes Therapeutics

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).

Peter Saltonstall
CEO
NORD

Peter L. Saltonstall has been the President and CEO of the National Organization for Rare Disorders (NORD) since 2008.  Under his leadership, NORD has maintained the integrity of the Orphan Drug Act while forging new relations between the patient community and the U.S. Congress, and such federal agencies as HHS, FDA, NIH, CMS and the Social Security Administration.  In Peter’s view, advances for the rare disease patient are best achieved through joint efforts, thus he has built collaborations with drug and device companies as well as the medical, academic and investment communities.  He is regarded by Industry, the FDA, the U.S. Congress and the federal government as a leading voice on rare disease.  Peter is also committed to the globalization of the rare disease patient community as diseases do not recognize geographical boundaries and research is expedited when patients from many countries are involved.

Day One

Wednesday March 27, 2019

17.30 | Panel Discussion: Patient Advocacy in the Gene Therapy Space: A Case Study in Hemophilia

Jude Samulski
Professor
UNC School of Medicine

Dr. Samulski was the scientific founder of Bamboo Therapeutics, Inc. and served as the Chief Scientific Officer and Executive Chairman of the company until its acquisition by Pfizer. Dr. Samulski has worked with adeno-associated virus (AAV) for 40 years, and for the past 25 years, has been Director of the University of North Carolina Gene Therapy Center. Dr Samulski has been a serial entrepreneur, founding companies such as Asklepios Biopharmaceutical (AskBio), Chatham Therapeutics, Nanocor Therapeutics, and Merlin, all of which are focused on AAV-based gene therapy. Dr Samulski is a former member of the Recombinant DNA Advisory Committee (RAC), a committee tasked with assisting the FDA with approving or disapproving gene therapy clinical trials in the United States. He also frequently serves as a gene therapy consultant to the FDA. Dr. Samulski received his PhD in Medical Microbiology and Immunology from the University of Florida. Dr. Samulski’s graduate work (1978-82) invoked the cloning of AAV that demonstrated the first use of AAV as a viral vector and culminated in the first US patent involving non-AAV genes inserted into AAV.

Rosemarie Sellati
Director, Patient Advocacy-Hematology
BioMarin

Rosemarie Sellati is Director of Patient Advocacy for the Hemophilia A program at BioMarin, a gene therapy currently in Phase III Clinical Trials. She takes pride in being responsible for integrating the patient voice into clinical development, serving as the primary point-of-contact and establishing the strategic roadmap for patient advocacy activities.  A critical element to the success of drug development is listening and responding to the challenges and barriers the patient community is and will face.  Gene therapy has provided a new and unique set of obstacles for all stakeholders involved. An immunologist by training with over 20 years of bench/clinical research and patient advocacy experience, Rose began her scientific career at Boehringer Ingelheim Pharmaceuticals in 1995 as a Pharmacologist in Inflammatory Diseases in early drug discovery advancing clinical projects in the immunology, oncology and respiratory therapeutic areas. After identifying the need to ensure patient perspective early in the drug development process, she transitioned into Advocacy supporting R&D and later the Cardiovascular franchise at BI. Rose’s ability to apply an in-depth understanding of drug discovery to her efforts in patient advocacy gives her a unique perspective on how advocacy/patient insights play an integral role in research, development, and clinical operations.

Day One

Wednesday March 27, 2019

17.30 | Panel Discussion: Patient Advocacy in the Gene Therapy Space: A Case Study in Hemophilia

Michael Sherman
CMO & SVP
Harvard Pilgrim Health Care Institute

John Spaltro
Director, Global Regulatory Affairs
Amicus Therapeutics

Sarah Sullivan
Scientist I
Biogen

Holly Tabor
Associate Professor
Stanford University Medical Center

Lawrence Thompson
Principal Scientist & Group Leader
Pfizer

Lawrence (Larry) C. Thompson, PhD is a Principal Scientist in Analytical Research and Development within BioTherapeutic Pharmaceutical Sciences at Pfizer.  He has been with Pfizer for 5 years and is currently analytical lead on viral & plasmid based immunotherapeutics and gene therapies.  Previously, he spent 3 years in small biotech at two different companies as the analytical lead in the development of serum-based cancer diagnostics plus 4 years as a post-doc at the University of Tennessee investigating the binding interactions of serum proteins.  He has a BS Chemistry (University of Tennessee, Chattanooga) and PhD Biochemistry (Vanderbilt University). His education and research path covers a wide breadth including small molecule synthesis; protein cloning, expression and purification; enzyme structure/function determination; serum antigen identification and mAb production/selection; virus/plasmid analytics from method development to characterization to critical quality attribute determination to overall control strategy.  His work has generated a number of peer reviewed publications and presentations at scientific conferences as well as internally within Pfizer.

Mark Trusheim
Strategic Director, NEWDIGS
MIT

Mark Trusheim is a Visiting Scientist at the MIT Sloan School of Management and Strategic Director, NEWDIGS at the MIT Center for Biomedical Innovation.  He is the Founder and President of Co-Bio Consulting, LLC and has been a Special Government Employee for the FDA’s Office of the Commissioner. Mark’s research focuses on the economics of biomedical innovation, especially precision medicine, adaptive pathways, platform trials and their enablement by digital health advances. He also studies biotechnology regional clusters.  His work emphasizes using quantitative modeling in multi-stakeholder processes to inform public policy, corporate strategy, and product development and commercialization. Prior to MIT, his career spanned big data and simulations at Kenan Systems, marketing at Searle Pharmaceuticals, eHealth as Vice President of Monsanto Health Solutions, genomics as President of Monsanto’s Cereon Genomics, molecular diagnostics as CEO of start-up Cantata Laboratories and policy as the President of the Massachusetts Biotechnology Council.  Mark was a member of the Monsanto Pipeline Management Team and led the Monsanto Bioinformatics/IT activities. Mark is a frequent invited speaker on precision medicine, companion diagnostics, adaptive pathways and regional life sciences development at international conferences.  He holds degrees in Chemistry from Stanford University and Management (Finance and IT) from MIT.

Luk Vandenberghe
Associate Professor
Harvard Medical School

Dr. Vandenberghe is Assistant Professor at Harvard Medical School and directs the Grousbeck Gene Therapy Center at the Schepens Eye Research Institute and Mass Eye and Ear in Boston, Massachusetts, USA.  Dr. Vandenberghe trained as a Biochemical Engineer at the University of Leuven, Belgium after which he did his doctoral work on the structural basis for AAV as a gene therapeutic.  His work has focused on HIV, Adenovirus, and Adeno-associated Virus in the laboratories of Drs. Debyser (University of Leuven, Belgium), June, Wilson, and Bennett (University of Pennsylvania, USA). In 2012, Dr. Vandenberghe set up his independent research group aimed at deconstructing viral evolution to improve viral vector design and use in gene therapy and more profoundly understand capsid structure-function relationship. A primary focus is on the ssDNA mammalian adeno-associated virus (AAV) as a model.

Day Two

Thursday March 28, 2019

15.00 | Discovering, De-coding and Programming AAV

Sam Wadsworth
CSO
Ultragenyx

Day One

Wednesday March 27, 2019

16.30 | Rare Disease Gene Therapeutic Products – The Future is Here!

08.30 | Industry Leaders Panel: Pioneering the Next Generation of Gene Therapies

Yu Wang
Scientist II
Biogen

Ales Strancar
Managing Director
BIA Separations

Xiaohui Lu
Director, Analytical Development
CRISPR Therapeutics

Xin Swanson
Commerical Development Lead, Viral Vector Gene Therapy
Lonza

Day One

Wednesday March 27, 2019

09.15 | Flexible, Modular Manufacturing to Address Viral Vector Capacity Bottlenecks