Speaker Information


 

Robert Baffi, Ph.D.
Special Advisor to the Chairman and Chief Executive Officer, BioMarin

Robert A. Baffi, Ph.D., joined BioMarin in May 2000 and currently serves as Senior Advisor to the Chairman and Chief Executive Officer. As part of his role, Dr. Baffi is assisting with the regulatory review of valoctocogene roxaparvovec and the preparation of the vosoritide marketing application. Prior to this position, Dr. Baffi served as President of Global Manufacturing and Technical Operations, responsible for overseeing our manufacturing, process development, quality, and analytical chemistry departments. From 2000 to December 2009, he was our Senior Vice President of Technical Operations. 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, he 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 currently 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.

Mark Battaglini, J.D.
Senior Vice President and Head of Global Government Affairs, Alliance Development & Public Policy, bluebird bio

Mark Battaglini has been with bluebird bio (NASDAQ:  BLUE) since November 2015. Mr. Battaglini was also tasked with starting bluebird bio Italy S.r.l and acted as the subsidiary’s Interim General Manager from March 2017 to July 2018. Prior to bluebird bio, Mr. Battaglini had oversight over Cubist Pharmaceutical’s policy and legislative efforts in global markets. After serving as a commissioned officer in the U.S. Navy Judge Advocate Generals Corps, Mr. Battaglini started his private sector career with the Schering-Plough Corporation in their commercial division in 1992.  

Mr. Battaglini is a former Brookings Institute Congressional Fellow and was assigned to U.S. Senator Michael Enzi’s Health, Education, Labor and Pensions Committee staff. He received a B.S. in Management from Boston College (Magna Cum Laude). He also holds a J.D. from Seton Hall University School of Law and is a member of the New Jersey and Federal Bar.

Mr. Battaglini participates in numerous non-profit organizations for veterans, including MVPvets.org and the World War II Foundation. He is also currently on the Board of Directors for bluebird bio (Italy) S.r.l. and the Public Affairs Council in Washington, D.C. Mr. Battaglini is also a frequent guest lecturer on the role of public affairs in the U.S. and in the EU. He resides in McLean, Virginia with his wife Aileen and has twin daughters.

Disclosures: Mr. Battaglini has a financial relationship with bluebird bio.

Diane Berry, Ph.D.
Senior Vice President, Global Health Policy, Government, and Patient Affairs, Sarepta Therapeutics

Diane joined Sarepta Therapeutics in December 2011 and serves as Senior Vice President, Global Policy, Government & Patient Affairs. She engages policymakers at the federal, state, and local levels, as well as patient advocacy organizations, to advance critical policies related to newborn screening, regulatory policy, and reimbursement and access with a goal of expediting development and patient access to genetic-based therapies for rare diseases. Previously, Dr. Berry served in leadership roles for the federal government, across the legislative and executive branches, overseeing and implementing science and technology and public health activities. She served as a Subcommittee Staff Director and Senior Professional Staff Member for the U.S. House of Representatives Committee on Homeland Security and as Chief Scientist and Senior Biodefense Advisor at the Department of Homeland Security in the Office of Health Affairs. Dr. Berry was also a Senior Science Advisor at McKenna, Long, and Aldridge and a Science and Technology Policy Advisor and Fellow within the Department of Defense through the American Association for the Advancement of Science. She earned her Ph.D. in chemical engineering from Northwestern University and received her B.S. and M.S. in chemical/biochemical engineering from Tufts University.

Disclosures: Dr. Berry has a financial relationship with Sarepta Therapeutics, Inc.

Andrew P. Byrnes, Ph.D.
Chief, Gene Transfer and Immunogenicity Branch, Division of Cellular and Gene Therapies, FDA

Dr. Byrnes received his undergraduate degree from Yale University, earned his Ph.D. from the University of Oxford, and then conducted postdoctoral research at Johns Hopkins University. Dr. Byrnes has 20 years of experience at FDA in review of manufacturing of gene therapies, cell therapies and other products for clinical use. Dr. Byrnes has a background in virology and gene therapy, and his laboratory research focuses on adenovirus gene therapy in rodent models. Research interests include vector biodistribution, clearance of vector by the liver, interactions of virions with natural antibodies and complement, and innate immune reactions to viral vectors.

Disclosures: Dr. Byrnes has a financial relationship with Leidos.

R. Alta Charo, J.D.
Warren P. Knowles Professor of Law and Bioethics, University of Wisconsin Law School

In government, Charo served as a legal analyst for the former congressional Office of Technology Assessment, focusing on new reproductive technologies, and as a policy analyst for the U.S. Agency for International Development, focusing on family planning in Latin America and Francophone Africa.  She has served as a senior policy advisor in the FDA’s Office of the Commissioner, working on drug safety, personalized medicine, and bioengineered foods. She was a member of  President Clinton’s National Bioethics Advisory Commission, helping to draft reports and recommendations on ethical approaches to research. She also served on the Obama transition team, focusing on NIH and FDA. A member of the National Academy of Medicine, Charo has co-chaired the National Academies’ committees that wrote guidelines for embryonic stem cell research and recommendations for U.S. policy and global principles regarding human genome editing, was a member of the organizing committee for the international summit on genome editing, and is a member of the standing committee on emerging science technology and law. At present, she is a member of the Nuclear Threat Initiative’s Biosecurity Innovation and Risk Reduction project, and of the World Health Organization’s committee on global governance of genome editing, 

Disclosures: Ms. Charo has a financial relationship with Johnson & Johnson.

Daniel Chung, D.O.
Medical Strategy Lead- Ophthalmology, Spark Therapeutics​

Dr. Chung is the Ophthalmology Therapeutic Area Leader for Spark Therapeutics and the company’s inherited retinal disease and ophthalmology expert and resource. He also served as Spark’s Medical Affair Ophthalmic Lead and the Clinical Development Ophthalmic Lead. Prior to joining Spark Therapeutics, he was a senior investigator at the FM Kirby Center for Molecular Ophthalmology at the Scheie Eye Institute at the Perelman School of Medicine of the University of Pennsylvania, working in retinal gene therapy and transfer. Concurrently, he served as the scientific advisor on the RPE65 gene therapy study team at the Children’s Hospital of Philadelphia (CHOP). He was the PI and first author for the RPE65 natural history study and the Multi-luminance Mobility Test (MLMT) validation study. Dr. Chung earned his medical degree from the New York Institute of Technology College of Osteopathic Medicine and completed his ophthalmology residency in Akron, Ohio. He then completed fellowships in pediatric ophthalmology and ocular genetics research at the Cole Eye Institute at the Cleveland Clinic, with additional training in retinal gene therapy fellow at the National Eye Institute/NIH in Bethesda, MD. As the Therapeutic Area Leader for ophthalmology, he works in the areas of medical affairs, clinical development and operations, pre-clinical studies, patient advocacy, commercial and marketing and business development.

Disclosures: Dr. Chung has a financial relationship with Spark Therapeutics.  

George Q. Daley, M.D., Ph.D.
Dean and Caroline Shields Walker Professor of Medicine, Harvard Medical School

Dr. Daley's 25-year career as a physician-scientist has focused on stem cell science and cancer biology, with an emphasis on development and diseases of the bone marrow, blood and immune system. He has co-authored international guidelines for the conduct and clinical translation of stem cell research and regenerative medicine, and for ethical oversight of emerging biotechnologies. His priorities as Dean of HMS include fostering biomedical, computational, and health care policy research, building the pipeline of novel therapeutics that are founded on breakthrough basic science, nurturing the next generation of physician-scientists, and advancing diversity in science, technology, engineering and medicine. Daley earned his AB and M.D. degrees from Harvard and a Ph.D. in biology from MIT, and has worked as a trainee, fellow and staff physician at the Massachusetts General Hospital, Brigham and Women’s Hospital, Dana Farber Cancer Institute and Boston Children’s Hospital. 

Disclosures: Dr. Daley has financial relationships with 28/7 Therapeutics and MPM Capital.

Joanne S. Eglovitch
Senior Editor, Pink Sheet

Joanne Serpick Eglovitch is a senior editor for Pink Sheet where she writes about manufacturing quality issues, and covers issues related to cell and gene therapy manufacturing. She was previously the editor of the International Pharmaceutical Regulatory Monitor and the International Medical Device Regulatory Monitor. Joanne also covered the mortgage finance industry and employment and training programs for various newsletters, and has worked as a general assignment reporter for a newspaper in southern Maryland and as an editorial assistant for a national newsmagazine. She received her BA in journalism and political science from the George Washington University.

Beverley Francis-Gibson
President & Chief Executive Officer, Sickle Cell Disease Association of America

Beverley Francis-Gibson is President/CEO of the Sickle Cell Disease Association of America, Inc. (SCDAA)

Francis-Gibson previously served as the Executive Director of the National Alliance on Mental Illness (NAMI HC) of Howard County in Columbia, Maryland and has more than 25 years of experience serving non-profits and community foundations. She is co-chair of the Maryland Statewide Steering Committee on Adults Living with Sickle Cell. Francis-Gibson is currently a Board Member of the Global Patient Advocacy Advisory Board on Sickle Cell Disease; a member of the Sickle Cell Disease Coalition and the Sickle Cell Steering Committee. She also serves on the National Minority Quality Forum’s Sickle Cell Disease Working Group, the SCD Patient Survey Steering Committee, oneSCD Voice Engagement Working Group, Emergency Department Sickle Cell Care Coalition, Steering Committee of the Black Women’s Health Imperative. She is Immediate Past Chair of the Board of Directors of Maryland Nonprofits. She is a 2009 Leadership Premiere Graduate and previous Board member of Leadership Howard County and is a proud member of Delta Sigma Theta Sorority, Inc.

Francis-Gibson is the creator and co-facilitator of “Leadership Conversations” a quarterly gathering of nonprofit executive directors who share best practices, network, and hear from engaging speakers on various topics. Currently, she mentors executive directors through her affiliation with the Association of Community Services.

A native of St. Thomas, US Virgin Islands, Francis-Gibson holds a B.A. in Public Relations and Communications and an M.A. in Industrial and Community Counseling from Eastern Kentucky University, Richmond, Kentucky.

Disclosures: Ms. Francis-Gibson has a financial relationship with SCDAA. 

Denise Gavin, Ph.D.
Chief, Gene Therapies Branch, FDA

Dr. Gavin is the Chief of the Gene Therapy Branch (GTB) at the United States Food and Drug Administration (FDA), Office of Tissues and Advanced Therapies (OTAT), Division of Cellular and Gene Therapies in the Center for Biologic Evaluation and Research (CBER). In this role, she oversees the chemistry, manufacturing and controls (CMC) evaluations for gene therapy products under Investigational New Drug (IND) and Biological License Applications (BLA). The GTB oversees a wide variety of products including viral and non-viral vectors, ex vivo genetically modified cells and products that include human genome editing. The GTB is also responsible for developing policy related to regulation of cell and gene therapy products; and performing outreach to gene therapy stakeholders. Dr. Gavin previously performed fellowships at the NIH, National Institute of Neurological Disease and Stroke, Laboratory of Molecular Biology (LMB), Bethesda, MD and at the University of North Carolina-Chapel Hill, Gene Therapy Center. Dr. Gavin has a PhD. in Microbiology/Immunology with a focus on molecular virology from Rush University School of Medicine in Chicago, Illinois.

Bradley J. Glasscock, Pharm.D.
Group Vice President, Head of Global Regulatory Affairs, BioMarin​

With over 20 years of experience in drug development, working in both industry and government positions, Brad Glasscock has successfully developed and implemented regulatory strategies across many different therapeutic areas and across all stages of the regulatory process, leading to several new product approvals.

Brad joined BioMarin in 2012. During his tenure, Brad has overseen multiple product approvals across the globe in the rare disease setting, including Vimizim for MPS IVA (2014), Brineura for CLN2 (2017), and Palynziq for PKU (2018). Prior to BioMarin, Brad was with Amgen for 7 years where he served as a global regulatory leader responsible for several late stage programs in oncology and metabolism, including the approvals of Prolia (2010) and XGEVA (2010). Prior to joining Amgen, Brad was with FDA for 6 years, serving in both the Office of Orphan Products Development (OOPD) and the Center for Biologics Evaluation and Research (CBER).

Brad received his Doctor of Pharmacy degree from the University of North Carolina at Chapel Hill.

Disclosures: Dr. Glasscock has a financial relationship with BioMarin.

Jeet Guram, M.D.
Senior Advisor in the Office of the Commissioner, Food and Drug Administration

Dr. Jeet Guram is a Senior Advisor in the Office of the Commissioner at the Food and Drug Administration. He joined FDA in February from the Centers for Medicare & Medicaid Services, where he served for nearly three years as a Senior Advisor in the Office of the Administrator. At CMS, Jeet led a series of initiatives including efforts to lower the cost of prescription drugs and to overhaul the program for Accountable Care Organizations or “ACOs.” Prior to joining CMS, Jeet was an Associate at McKinsey & Company. Jeet has an M.D. from Harvard Medical School and an M.B.A. from Harvard Business School.

Christine Harrison
Vice President, Corporate Affairs, Orchard Therapeutics

Christine brings nearly 20 years of strategic communications, public affairs, advocacy, and policy expertise to Orchard Therapeutics as Vice President, Corporate Affairs. She oversees multiple related organizational functions, including corporate and internal communications, patient advocacy, government and public affairs, and policy. Christine has extensive background in the life sciences and health insurance industries, and her areas of expertise include coalition-building, campaign development, issues and crisis management, media relations and corporate social responsibility. Prior to joining Orchard in 2018, Christine held senior leadership positions at various global health care communications and public affairs consulting firms, including Edelman and APCO Worldwide. Most recently, she served as managing director of Reservoir Communications, a firm specializing in health care advocacy and policy communications, where she was instrumental in building and growing the organization from the ground-up. Christine holds a BA from the University of Virginia, where she graduated with high distinction. 

Disclosures: Ms. Harrison has a financial relationship with Orchard Therapeutics.

Nicola Heffron
Head of Europe, bluebird bio​

Nicola is Head of Europe for Bluebird Bio, a leading biotech in gene therapy. Today Nicola is focused on partnering with European payers to achieve reimbursement, working with EU medical institutions to qualify them as gene therapy treatment centres and to launch several novel gene therapies in rare hematological diseases. Nicola has over 25 years of experience in pharma and biotech spanning R&D to commercial launch, and she has held a number of senior leadership roles at Celgene, Shire, GlaxoSmithKline and Eli Lilly. In the last five years Nicola has specialized in working with cross-functional teams to achieve regulatory approval, reimbursement, and launch of life-changing medicines and technologies in rare and ultra-rare diseases.

Nicola is currently based in Switzerland and is a registered pharmacist with the Royal Pharmaceutic Society in the UK and holds an MBA from the University of Warwick, England.

Ying Huang, Ph.D.
Pharmacologist, Pharmacology/Toxicology Branch, FDA

Dr. Huang joined FDA/CBER/OTAT/DCEPT in 2004 and is a Pharm/Tox master reviewer responsible for the review of regulatory submissions for cell and gene therapies, including genome editing and edited products on oncology and non-oncology diseases. She is the FDA Topic Leader in the ICH S12 Expert Working Group on Non-clinical Biodistribution Studies for Gene Therapy Products. Prior to the FDA, Dr. Huang received her Ph.D. in Pharmacology and Toxicology at the University of Toronto, and subsequently an NIH IRTA fellowship before became a senior scientist at former Genetic Therapy Inc., a Novartis Company.

Adam Hutchings
Managing Director, Dolon

Adam Hutchings is the Managing Director of Dolon, a strategic market access consultancy that specialises in rare diseases. A health economist by training, he has worked in biopharmaceutical pricing and market access for 20 years. Adam is a member of the multi-stakeholder ORPH-VAL Working Group that has developed and published recommendations on pricing and reimbursement processes for orphan medicines. He is also a member of the RARE-IMPACT Working Group, an initiative led by EURORDIS (a rare disease patient group) which is seeking to define pricing and reimbursement pathways for cell and gene therapies for rare diseases. He has published extensively on the economics of rare diseases and has a particular interest in the role of pricing within the orphan drug business model. 

Annie Kennedy 
Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases 

Focused on improving health outcomes for people living with rare diseases by advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy, Annie’s work includes building strong partnerships with policy makers, federal agencies, industry, and alliances. Current areas of emphasis include leading the national Burden of Rare Disease Study, 21st Century Cures Act and PDUFA VII engagement, national newborn screening program engagement and state RUSP alignment legislation, and innovating around therapy valuation and access issues.   

Annie has served within the community for nearly three decades through her roles with Parent Project Muscular Dystrophy (PPMD) and the Muscular Dystrophy Association (MDA). In that time she helped lead legislative efforts around passage and implementation of the MD-CARE Act (2001, 2008, 2014), the Patient Focused Impact Assessment Act (PFIA) which became the Patient Experience Data provision within the 21st Century Cures Act (sec 3001), engagement with the FDA and industry around regulatory policy and therapeutic pipelines, led access efforts as the first therapies were approved in Duchenne, and engaged with ICER around the development of the modified framework for the valuation of ultra-rare diseases.

Peter Marks, M.D., Ph.D.
Director, FDA Center for Biologics Evaluation and Research (CBER)

Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in industry on drug development. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in January 2016.

Michelle McMurry-Heath, M.D., Ph.D.
President and CEO, Biotechnology Innovation Organization (BIO)

The common thread in McMurry-Heath’s work across academia, government and industry has been her focus on broadening access to scientific progress so more patients from diverse backgrounds can benefit from cutting-edge innovation. Driven by her own past family experiences navigating clinical trials and funding uncertainties within the rare disease community, McMurry-Heath calls “the distribution of scientific progress the social justice issue of our age.” 

She comes to BIO from Johnson & Johnson where she served as Global Head of Evidence Generation for Medical Device Companies and then Vice President of Global External Innovation and Global Leader for Regulatory Sciences. She was also instrumental in bringing J&J’s incubator, JLabs, to Washington, DC. She led a global team of 900 with responsibilities in 150 countries around the globe. 

Prior to her time at J&J, Dr. McMurry-Heath was also a key science policy leader in government. The Obama-Biden transition team tapped her to conduct a comprehensive analysis of the National Science Foundation’s policies, programs and personnel. President Obama then named her associate science director of the FDA’s Center for Devices and Radiological Health under Commissioner Peggy Hamburg. In that role, she championed clinical trial evolution, the use of real-world evidence in product evaluation, and an embrace of the patient’s voice in health research so new medical products deliver outcomes that matter to them. 

McMurry-Heath received her M.D./Ph.D. from Duke’s Medical Scientist Training Program, becoming the first African-American to graduate from the prestigious program. She spent 12 years working at the research bench before taking policy and leadership roles in government and industry.

Katie Miller, Ph.D.
Principal, Dark Horse Consulting 

Katie is an accomplished biopharmaceutical executive with over 25 years of industry experience leading multiple CMC functions in the development and commercialization of advanced therapeutics including cell and gene therapies, inhalation and parenteral formulations, multi-active solid oral dosage forms, and device/combination products. Believing strongly in the criticality of robust product characterization for the success of the regenerative medicine industry, at Dark Horse Consulting she specializes in method development, validation and transfer, as well as quality control testing and specification strategies, comparability studies, and analytical capability build vs. buy analysis. 

Christopher K. Murphy
Vice President and General Manager of Viral Vector Services in Thermo Fisher Scientific’s Pharma Services Group

Christopher Murphy is Vice President and General Manager of Viral Vector Services in Thermo Fisher Scientific’s Pharma Services Group. Prior to joining Thermo Fisher, Chris served as chief operating officer for Brammer Bio, a leading viral vector/gene therapy CDMO. Chris has more than 30 years of experience in biopharmaceutical manufacturing, process development, plant operations, and quality system implementation. Prior to joining Brammer Bio, Chris served as general manager for the Sanofi-Genzyme Allston Landing site overseeing operations and leading an integrated remediation program associated with the consent decree. Chris has led gene therapy operations and recombinant protein manufacturing at Genzyme and Bioreliance and he began his career in viral vaccine development at American Cyanamid/Wyeth (now Pfizer) in Pearl River, NY. He has an M.Sc. in biochemistry at New York Medical College, and his B.Sc. in biology at Rutgers University.

Nancy Bradish Myers, J.D.
President/Founder, Catalyst Healthcare Consulting, Inc.

Nancy Myers is a Washington-based attorney with expertise in health care law and regulation, policy development, government relations, and political analysis for investors. She is the President of Catalyst Healthcare Consulting, Inc., a boutique consulting firm that provides clients with strategic regulatory insight and advice as they position biopharmaceutical and medical device companies, trade associations, and patient advocacy organizations on regulatory and health policy matters before the FDA and other regulatory agencies.

Ms. Myers has served in FDA’s Office of the Commissioner in various positions, including as Senior Strategic Advisor. She has also served as Special Counsel for Science Policy for PhRMA, Vice Presidential-level political healthcare analyst for a Wall Street financial services firm, Reimbursement Counsel and Director of Government Affairs for BIO, a lobbyist for the Blue Cross Blue Shield Association, and staff person to a Member of Congress on Capitol Hill.

Herb Runnels, Ph.D. 
BioTx Pharmaceutical Sciences, Analytical R&D, Pfizer

Trained as a molecular and cellular immunologist, I joined Pharmacia (legacy GD Searle/Monsanto; later Pfizer in 2003) Discovery Research to help design cell-based therapeutics and chemokine receptor antagonists (small and large molecule) as well as to shuttle therapeutic antibodies, against various soluble and cell-bound targets, into human clinical trials. In 2010, I transitioned my career with a move to Analytical R&D in Pfizer’s Biotherapeutics Pharmaceutical Science (BioTx PS) division. This move allowed me to focus on candidates in clinical development and licensure stages. In BioTx PS, I have worked on complex bioassays for many modality-types, led global analytical teams in the areas of Vaccines and Gene Therapy and have been pivotal on many business development alliances. A notable achievement was leading the design and implementation of viral vector analytical strategy and capabilities for Pfizer BioTx PS to support both early and late phase programs with accelerated cycle times.

Disclosures: Dr. Runnels has a financial relationship with Pfizer.

Becky Schweighardt, Ph.D.
Senior Director, Immunogenicity Assessment, BioMarin​

Dr. Schweighardt is an executive director at BioMarin Pharmaceutical in San Rafael, CA. The IA group that she leads focuses on developing and implementing strategic risk-based approaches for determining the impact of drug-related immunogenicity on the clinical safety and efficacy of novel biologic therapeutics, including gene therapies. The CP group characterizes nonclinical and clinical Pharmacokinetics (PK) and Pharmacodynamics (PD) and uses quantitative approaches to define therapeutic index, exposure-response relationships, and inform dose, route, and therapeutic schedule. BioMarin is a leader in rare disease drug development for small molecules, therapeutic proteins, and gene therapies. 

Disclosures: Ms. Schweighardt has a financial relationship with BioMarin.

Tim Scott
United States Senator, South Carolina

Growing up in a poor, single parent household in North Charleston, South Carolina, a young Tim Scott grew accustomed to moving every few years, as well as the long hours his mom worked to keep a roof over their heads. After failing four classes his freshman year of high school, Tim’s path forward was murky at best. 

But thankfully, he had a mom who stuck with him, and met a mentor that showed him the wisdom of conservative principles. Through their belief and his own determination, Tim got his grades back on track, graduated from Charleston Southern University, and eventually built his own successful small business. 

Through the lessons taught by his mentor, Tim developed his mission statement: to positively affect the lives of a billion people. That led him to public service, where he was elected to Charleston County Council, the South Carolina House of Representatives, the United States House of Representatives, and the United States Senate. 

Since joining the Senate in 2013, Senator Scott has been a national leader on efforts to bring opportunity to every American family. His signature legislation creating Opportunity Zones was passed as part of the 2017 tax reform package, and has the potential to bring billions of dollars of private investment into distressed communities across the country. 

As he continues to lead on the implementation of the Opportunity Zones initiative, Senator Scott also plays a critical role in issues regarding workforce development, education and diversity. Whether it’s tackling the skills gap, making sure children have access to the educational environment best suited for them, or working to bring everyone to the table to find solutions, Senator Scott will keep fighting for South Carolinians every single day he serves in the U.S. Senate. 

Andrea Spezzi, M.D.
Chief Medical Officer and Co-Founder, Orchard Therapeutics

Andrea's expertise lies in the development and execution of all aspects of clinical development and medical affairs, particularly in paediatric rare diseases. Prior to joining Orchard Therapeutics, Andrea served as VP and Medicine Development Leader at GSK Rare Diseases Unit Rare Diseases Unit, and before that she was Senior Medical Director at the Immune Inflammation Discovery Performance Unit.  

Before GSK, Andrea was Global Medical Director R&D at Takeda in the metabolic and cardiovascular area and in charge of the paediatric developments for Takeda Europe R&D. She previously worked as Research Physician at Hammersmith Medicines Research. Before moving to the pharmaceutical industry Andrea practiced as a paediatrician for 12 years in Argentina, Spain and UK. She was recognized as one of 2018’s “Fiercest Women in Life Sciences” by FiercePharma and as one of BioBeat’s “50 Movers & Shakers in BioBusiness” in 2017.  

Andrea obtained her M.D. degree from the University of Buenos Aires (UBA) and completed residency in paediatrics and post graduate training in paediatric immunology at the Dr Elizalde Children's Hospital in Buenos Aires, Argentina. She obtained a Specialist in Pharmaceutical Medicine degree from University of Basel, Switzerland and she is a Fellow of the Faculty of Pharmaceutical Medicine in London, UK (FFPM).

Dan and Jodi Symes

Dan was born and raised in Stoneham, Massachusetts and after attending college in Colorado and living in the Midwest for several years, he returned there to raise his family. Dan worked as a construction supervisor in a family-owned business for almost 40 years before retiring in 2018.

Jodi also grew up in Massachusetts where she too raised a family. She worked in human resources in the healthcare and biotech industries until her retirement in 2018.

Dan and Jodi married in 2011 and live on the Merrimack River in Amesbury, MA. Since their retirement they have spent most of their time boating on their power catamaran and recently returned from Dan’s “bucket list trip” to America’s Great Loop. They enjoy spending time with their granddaughter and extended family and friends and keep busy with home and boat projects.

Without CAR-T cell therapy, Dan would not have been able to live out this lifelong dream and they are both eternally grateful for this life changing therapy.

Jan Thirkettle, Ph.D.
Chief Development Officer, Freeline

Jan is Chief Development Officer at Freeline, a clinical-phase AAV gene therapy company focussed on the development of systemically delivered gene therapies for the treatment of chronic diseases. As such, Jan has responsibility for Programme Management and CMC/Manufacturing. Jan is a Director of the US, Irish and German Subsidiaries. 

Jan has extensive experience in the development of novel manufacturing platforms including natural product and enzyme-derived NCEs, biologics and gene therapies, and has enabled the launch of medicines in all of these areas. Prior to joining Freeline Jan led the establishment of GSK’s Cell & Gene Therapy platform and was responsible for CMC/supply for Strimvelis, the first ex-vivo gene therapy to receive an EU Marketing Authorisation Application. He has held pharma industry positions spanning from discovery to commercial manufacturing, but is most passionate about the cross-functional elements of late-state development  and new platform deployment. Jan holds an MA in Chemistry and a PhD in Biological Chemistry from Oxford University.   

Disclosures: Dr. Thirkettle has a financial relationship with Freeline.

Andrew Walker, Ph.D.
Director, Salus Alba

Dr. Walker is a UK-based health economist with 30 years postgraduate experience. After my MSc from York and PhD in cancer screening, my career started in a variety of academic and payer roles in England and Scotland. In 2002 I was involved in setting up the cost-effectiveness review system for Scottish Medicines Consortium, and then stayed on for 12 years (working from a university base) as a reviewer and committee member in later years. A huge advantage of SMC is the breadth of experience it gave in applying HTA to new medicines in very disease area.   

I became self-employed in 2015, and have worked with over 80 pharma companies on numerous projects. My areas of expertise are advising on the HTA requirements to consider when designing a clinical study programme and planning a submission to a UK HTA agency. 

In 2018 I was approached by an Evidence Review Group to work as a part-time reviewer on NICE STA and HST submissions. From 2020 I am also an HTA expert for the NICE Early Scientific Advice service, so when selected I am one of the team who read the pharma company’s briefing and answers their questions. 

 

2020

ASGCT Policy Summit

September 23-25, 2020