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Returning in April 2018
Boston, USA

 

2017 Speakers

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Sven Kili
VP & Head of Cell & Gene Therapy Development
GSK

Day One

Tuesday, April 25, 2017

09.00 | Keynote Panel Discussion: The Future of Gene Therapies for Rare Disease

Rune Kjeken
Scientific Director, Advanced Therapies
CAT, EMA

Day One

Tuesday, April 25, 2017

16.00 | Panel Discussion: Cross-Industry Perspectives on Defining the Regulatory Course for Gene Therapies in Rare Disease

15.30 | Gene Therapy for Rare Diseases: EMA Perspective

Adrien Lemoine
VP, Business Development
Orchard Therapeutics

Day Two

Wednesday, April 26, 2017

16.00 | Panel Discussion: Analyzing the Evolution of Deal Structures Between Companies Pioneering the Gene Therapy Space

Al Hawkins
CEO
Milo Biotechnology

Al Hawkins has worked as an early stage investor and entrepreneur, translating academic innovations into start-up companies in multiple geographies and settings.   In Madison, Wisconsin from 2000-2005, he was Founder and Partner of Agave Group, which worked with over 25 university spin-outs and conducted diligence for multiple angel investment groups in the Upper Midwest.  In Boston, Al was Director of New Ventures at Boston University, managing the BU Venture Fund and the process of forming spin-out companies from BU’s IP base.  In 2011, Al moved to Cleveland and became CEO-in-Residence at BioEnterprise founded two start-ups from Nationwide Children’s Hospital.  Then became VP of Business Development and an initial employee at BioMotiv, a $140M therapeutics accelerator partnered with Takeda, Biogen and University Hospitals Cleveland. In 2013, Al co-founded gene therapy company Abeona Therapeutics and served as its Chairman until 2015 when Abeona was merged into a NASDAQ listed company.  He is currently CEO of Milo Biotechnology, which he co-founded in 2011; Milo is a mid-clinical stage gene therapy company that has established clinical proof of concept in two muscular dystrophy indications.   Al received an SM in Health Sciences and Technology from the Harvard-MIT Institute of Health Sciences, a Masters in Business from UW-Madison and a BA from Emory University.

Day Two

Wednesday, April 26, 2017

16.00 | Panel Discussion: Analyzing the Evolution of Deal Structures Between Companies Pioneering the Gene Therapy Space

11.30 | Panel Discussion: What Does it Take to Succeed in the Gene Therapy Space?

Andrea Spezzi
CMO
Orchard Therapeutics

Day One

Tuesday, April 25, 2017

14.30 | The Patient Pathway: Examining the Complex Patient Logistics Required to Progress Ex Vivo Gene Therapies

Aniz Girach
CMO
NightstaRx

Aniz Girach, qualified as an Ophthalmologist (specialty in Retina), from Cambridge (UK). After having spent 11 years in academia, he joined the pharmaceutical industry with Eli Lilly, focusing on retinal diseases. He has in total 20 years industry experience in roles with Merck (as their Global Head of Ophthalmology) and Alcon, where he was Vice President of Clinical Development, and ThromboGenics, where he was the Global Head of Ophthalmology/Chief Medical Officer overseeing the development and approval of Ocriplasmin (Jetrea)—a first in class biologic therapy for retinal disease. He is the Chief Medical Officer at NightstaRx, overseeing the development of gene therapies for inherited retinal diseases. He is a member of 3 Scientific Advisory Boards for international ophthalmic organizations currently, and reviewer for 5 peer-reviewed journals, including Eye and IOVS. He has edited 4 books and published over 60 abstracts/manuscripts in peer-reviewed journals in Ophthalmology, with numerous invited lectures at national/international ophthalmology meetings. His special interests are Medical Retina, Vitreomacular Interface Abnormalities and Gene Therapy.

Day Two

Wednesday, April 26, 2017

09.30 | The Selection of Endpoints for Ophthalmology Gene Therapy Trials in the Rare Disease Space

Beth White
AVP, Rare Disease Commercial Development
Pfizerx

Day Two

Wednesday, April 26, 2017

15.30 | Funding & Investment to Further the Development of Gene Therapies

Casey Maguire
Assistant Professor
Harvard Medical School

Pre Conference Morning Workshop C

09.00 | Workshop C: Discussing Novel Technologies With the Potential to Impact the Gene Therapy Field

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

Tuesday, April 25, 2017

16.00 | Panel Discussion: Cross-Industry Perspectives on Defining the Regulatory Course for Gene Therapies in Rare Disease

Day Two

Wednesday, April 26, 2017

11.30 | Panel Discussion: What Does it Take to Succeed in the Gene Therapy Space?

Daniel Leonard
Director, Global Patient Advocacy
UniQure

Day Two

Wednesday, April 26, 2017

14.00 | Patient Advocacy in the Gene Therapy Space: A Case Study in Hemophilia

Edmund Pezalla
Consultant
EJ Pezalla Independent Consultant

Pre Conference Afternoon Workshop B

Monday April 24, 2017

13.00 | Workshop B: How will Gene Therapies be Reimbursed?

Eileen Sawyer
Director, Global Medical Affairs
UniQure

Day Two

Wednesday, April 26, 2017

11.00 | Bridging Gaps Through Early Integration of the Internal Medical Affairs Function

Emily Culme-Seymour
External Strategy Manager, Gene Therapy
Rare Diseases Unit, GSK

  Emily is External Strategy Manager, Gene Therapy in GSK’s Rare Diseases Unit. She is a scientist looking at developing external relationships and partnerships in the field, as well as providing guidance and input to the internal gene therapy strategy and pipeline. She has a background in Chemistry and Biochemical Engineering, looking at engineering growth substrates for pluripotent stem cell bioprocessing during her PhD at University College London (2006-2010). Emily is also a Director of the London Regenerative Medicine Network, sits on the International Society for Cell Therapy’s Executive Committee as Global Treasurer, is a member of the UK BioIndustry Association’s Cell and Gene Therapy Advisory Committee, sits on the American Society for Gene and Cell Therapy’s International Committee, and is a member of the European and British Societies for Gene and Cell Therapy.

Day Two

Wednesday, April 26, 2017

16.00 | Panel Discussion: Analyzing the Evolution of Deal Structures Between Companies Pioneering the Gene Therapy Space

Gopalan Narayanan
VP, Disruptive Biologics
Voisin Consulting Life Sciences

Dr Narayanan provides leadership in the area of complex and disruptive biologics such as Cell and Gene therapies, including guidance on product development and regulatory strategy and in the global effort of rationalising the development process through which such disruptive biologics can be brought to patients faster and more efficiently. Narayanan was a senior regulator at the UK MHRA for nearly 12 years as an expert medical assessor, and 4 years as Manager of the Biologicals Unit. He was a member of the Committee for Advanced Therapies (EMA/CAT 2009 – 2013), Scientific Advice Working Party (CHMP/SAWP 2009 - 2013) representing  EMA/CAT, Biosimilar Working Party (EMA/BMWP) and Gene Therapy Working Party (CAT/GTWP). Narayanan qualified as a physician, and has dual specialist postgraduate qualification in general internal medicine and pharmaceutical medicine. He is currently on the Scientific Editorial Board of Human Gene Therapy Clinical Development journal and is a Review Editor of Regulatory Science section of Frontiers in Medicine, and is based in the UK.

Pre Conference Morning Workshop A

Monday April 24, 2017

09.00 | Workshop A: Understanding the Regulatory Landscape and Expectations of the Gene Therapy Field

Day One

Tuesday, April 25, 2017

16.00 | Panel Discussion: Cross-Industry Perspectives on Defining the Regulatory Course for Gene Therapies in Rare Disease

Lance Weed
VP, US Operations
UniQure

Day One

Tuesday, April 25, 2017

14.00 | Optimizing the Scalability of Gene Therapy Manufacturing

Lei Xu
Chief, General Medicine Branch 2, Division of Clinical Evaluation
and Pharmacology/Toxicology, OTAT, CBER, FDA

Dr. Lei Xu is the Chief of General Medicine Brach 2 in the Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), Office of Tissue and Advanced Therapies (OTAT), Center for Biologics Evaluation and Research (CBER) at the FDA. Dr. Xu joined DCEPT at FDA in 2009 as a Medical Officer working on clinical trials of gene and cell therapy products primarily for neurological, ophthalmological disorders and other disorders. Oversight of several additional clinical areas, including cardiology, is added upon her appointment as Branch Chief in 2017. In this position, she is responsible for monitoring trials of investigational agents for safety and efficacy. She is also responsible to manage, organize, and direct all of the regulatory review operations, program segments, functions and activities of the Branch.  In addition to her regulatory experience, she is actively involved in FDA guidance development, including the Guidance for Industry: Expedited Programs for Serious Conditions. Dr. Xu received her M.D. from Central South University Xiangya School of Medicine, China. She earned her Ph.D. in neuroscience from Yale University.  She completed residency training in Neurology at Loyola University Chicago. She is board-certified in Neurology by the American Board of Psychiatry and Neurology. She published more than 10 research papers in peer-review journal prior to joining FDA.

Day Two

Wednesday, April 26, 2017

08:30 | Clinical Development of Gene Therapies for Rare Disease: FDA Perspective

Luk Vandenberghe
Assistant Professor
Harvard Medical School

Day Two

Wednesday, April 26, 2017

16.00 | Panel Discussion: Analyzing the Evolution of Deal Structures Between Companies Pioneering the Gene Therapy Space

Michael Linden
VP, Gene Therapy
Pfizer

Day One

Tuesday, April 25, 2017

08.30 | Keynote: From Academic to Commercial Scale – Managing the Rapid Progression of Gene Therapies

Rajendra Kumar-Singh
Co-Founder & VP
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.

Day Two

Wednesday, April 26, 2017

11.30 | Panel Discussion: What Does it Take to Succeed in the Gene Therapy Space?

Victoria Sluzky
SVP, Quality & Process Development
BioMarin Pharmaceuticals

Day One

Tuesday, April 25, 2017

12.00 | Investigating CMC Considerations for the Production of a Gene Therapy Product

Sam Wadsworth
CSO
Dimension Therapeutics

Day One

Tuesday, April 25, 2017

11.30 | Overcoming the Capacity Bottleneck: Managing In-House & Outsourced Value Chains Effectively

09.00 | Keynote Panel Discussion: The Future of Gene Therapies for Rare Disease

Scott Burger
Principal
Advanced Cell & Gene Therapy

Pre Conference Afternoon Workshop D

13.00 | Workshop D: Enhancing the Outsourcing Process: How to Utilize Contract Manufacturing Services and the Broader Contract Infrastructure Effectively

Seng Cheng
Global Head of Research, Rare Diseases
Sanofi

Seng Cheng is the Global Head of Research of Rare Diseases at Sanofi. He received his BSc and PhD degrees in Biochemistry from the University of London, UK and trained as a postdoctoral fellow at the National Institute for Medical Research in the field of tumour biology. He worked as a Staff Scientist at Integrated Genetics Inc., and later joined Genzyme Corporation to work on several discovery projects including the structure and function of the cystic fibrosis transmembrane conductance regulator. At Genzyme, he managed the development of novel gene transfer systems as well as translational research in genetic diseases, a number of which transitioned to clinical development. He has co-authored 263 research articles and reviews, and is a named co-inventor on 96 issued patents in the area of biotechnology. In his current position, he is responsible for directing the translational research and early clinical development activities in rare genetic diseases.    

Day One

Tuesday, April 25, 2017

09.00 | Keynote Panel Discussion: The Future of Gene Therapies for Rare Disease

Tara O’Meara
VP, Clinical Development Operations
Bluebird Bio

Tara O’Meara is the Vice President of Clinical Development Operations at bluebird bio.  Tara oversees a team responsible for the execution and analysis of bluebird bio’s lentiviral vector based gene therapy development programs.  Currently bluebird bio has 4 active programs using ex vivo gene therapy for: cerebral adrenoleukodystrophy, transfusion dependent thalassemia, sickle cell disease and multiple myeloma.  bluebird’s objective is to develop and bring to market the most advanced products based on the transformative potential of gene therapy as a one-time treatment option for patients with severe genetic and rare diseases and cancer. Prior to joining bluebird, Tara ran multiple clinical trials in 2 other rare diseases, LAL Deficiency and Pompe disease.   Tara served as the clinical project manager for the clinical trials that were used as the basis for the regulatory submissions that led to the approval of Myozyme™ in the United States, Europe, and more than 40 additional countries worldwide. Tara has also been responsible for everything from small Phase II trials to very large, global Phase III trials in multiple therapeutic areas, including rare inherited diseases. Tara holds a BS in biology and a minor in women’s studies from Tufts University.

Day Two

Wednesday, April 26, 2017

09.00 | Overcoming Patient Recruitment & Trial Design Challenges to Accelerate Clinical Progression

Tim Miller
President & CEO
Abeona Therapeutics

Tim Miller, Ph.D. is President and Chief Executive Officer and Director of Abeona Therapeutics. He has 18 years of scientific research, product development and clinical operations expertise, with a focus on transitioning novel biotherapeutics through pre-clinical phases and into Phase 1 and 2 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). He has raised over $70M for therapies in cystic fibrosis, cardiovascular disease, wound healing, scar prevention, and rare diseases to advance these therapies into clinical trials. 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 Food and Drug Administration (FDA) and NIH advisory agencies on multiple Investigational New Drug (IND) 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, and recently returned from a successful summit climb of Mt. Rainier on a rare disease awareness and fundraising adventure.

Day Two

Wednesday, April 26, 2017

14.30 | Analyzing how a Single Gene Therapy Intervention May Be Priced Effectively for Different Rare Diseases

Vivian Choi
Associate Director, Head of Gene Therapy Research
Shire Pharmaceuticals

Day One

Tuesday, April 25, 2017

10.00 | Improve Decision Making Early in the Gene Therapy Drug Development Process to Enable Successful Progression Through the Clinic