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Join industry experts for a compelling series of panel discussions exploring the significant opportunities and challenges in cell and gene therapies (CGT). During this three-session series, experts in the area will discuss the important clinical trial considerations for these revolutionary therapies, including implementing novel development strategies, adopting strategies early in development to keep long-term production and commercialization goals on track, and addressing key clinical trial concerns when considering CGT for broader indications.


Panel Discussion

Implementing Novel Development Strategies For Cell & Gene Therapy Clinical Trials

Available On Demand

Cell & gene therapy (CGT) holds significant promise to advance patient treatments. As shown by recent trends, such as the growing interest in CAR T-cell therapy and mRNA gene editing, CGT is the future. Despite its potential, CGT often does not progress from the lab to commercial markets. This is in part due to the unique requirements of working with these technologies, and the new challenges they present to clinical trials.

This roundtable reviewed the necessary considerations and logistics of conducting CGT trials, and how sponsors and service providers can optimize their clinical research to maximize the prospect of success.

Other discussion points included:

  • The need for and management of study stopping rules to mitigate risks - protocol amendments - for patients and sites
  • The importance of transportation, including the complexity of handling living materials
  • How sequencing on site can reduce both the costs and risk associated with CGT clinical trials ?

Speakers

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Moderated By:

Ly Nguyen-Jatkoe, PhD

Executive Director, Custom Intelligence & Analytics, Informa Pharma Intelligence


As head of the Custom Intelligence team for the Americas and lead for cell and gene therapy thought leadership at Informa, Ly partners with clients to develop personalized solutions using Informa’s suite of pharma data and insights. She has 20+ years of experience in the healthcare and pharmaceutical industry and extensive experience in market research and analysis.

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Tamie Joeckel

Cell and Gene Therapy Global Business Lead, ICON


Tamie has over 25 years of experience in commercializing specialty biologics and has worked in cell and gene therapies for the last 8 years in global cryologistics and operations. As a former senior executive at one of the largest drug distributors, she specialised in commercialisation, patient hub services and reimbursement support for plasma derived drugs and therapies for oncology and rare diseases. At ICON, she is a member of the Cell and Gene Therapy Centre of Excellence and supports ongoing strategy and innovation across therapeutic areas. She is very active in industry groups focused on developing standards for the regenerative medicine sector.

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Jakob Dupont, M.D

Executive Vice President, Head of Global Research & Development, Atara Biotherapeutics


Dr. Jakob Dupont is a renowned expert in the fields of cell therapy and oncology, with long-standing and deep experience in developing therapies and programs dedicated to addressing high unmet medical needs. Dr. Dupont serves as Global Head of Research & Development including Medical and Regulatory Affairs. Prior to joining Atara, he served as the Chief Medical Officer at Gossamer Bio, overseeing global development, regulatory, and quality activities for the company, and advancing therapeutics in the disease areas of immunology, inflammation, and oncology. Dr. Dupont is committed to bringing transformative therapies to patients. Dr. Dupont has been involved in tumor immunology research and clinical investigations for more than 25 years, ranging from cellular therapy to tumor vaccine therapy and immune checkpoints. Dr. Dupont has received numerous grants and awards, and has co-authored 47 peer-reviewed publications, has 30 patents, and has served as a faculty member and laboratory researcher at Memorial Sloan Kettering Cancer Center (MSK) and adjunct clinical faculty in medical oncology at Stanford University.

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C. David Pauza, PhD

Chief Science Office, American Gene Technologies International


Dr. Pauza received his PhD in Molecular Biology from the University of California, Berkeley and was a postdoctoral fellow in the laboratory of Sydney Brenner, MD, Director, Laboratory of Molecular Biology in Cambridge, England, Noble Laureate (2002). Dr. Pauza held faculty positions at the Salk Institute for Biological Studies, La Jolla, CA, where he founded Salk’s HIV research program, University of Wisconsin-Madison, University of Maryland Biotechnology Institute, and University of Maryland School of Medicine where he served as Associate Director for the Institute of Human Virology and Co-Leader for the Viral Oncology Program in the Greenebaum Cancer Center. Since 2016 Dr. Pauza has been Chief Science Officer for American Gene Technologies International in Rockville, Maryland. He is an inventor on 14 issued US patents and numerous foreign filings representing efforts to treat and cure HIV, develop gene therapies for phenylketonuria (PKU) and apply genetic medicines to cancer. He led a successful effort to obtain an IND and initiate a Phase 1 clinical trial using an innovative gene therapy strategy for HIV disease. He is a frequent speaker at academic and industry conferences.

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Gallia G. Levy, M.D., Ph.D.

Chief Medical Officer, Spark Therapeutics


Dr. Gallia Levy is the Chief Medical Officer at Spark Therapeutics and leads strategic and operational teams throughout the product development lifecycle with responsibility for setting the global development strategy for current and pipeline programs. She serves as the principal physician and resident medical expert at Spark.

Prior to joining Spark, Gallia was Vice President and Global Head of the Rare Blood Disorders Franchise in Product Development at Genentech, another member of the Roche Group, where she was responsible for the clinical development of treatments for hemophilia A and other rare blood disorders such as paroxysmal nocturnal hemoglobinuria (PNH), and atypical hemolytic uremic syndrome (aHUS). Gallia first joined Genentech in 2009, where she worked in both early and late-stage clinical development. She later moved to Portola Pharmaceuticals, where she led the clinical development program for hematology and oncology indications spanning Phases 1 to registrational trials. She eventually returned to Genentech, taking responsibility for the global clinical development program for HEMLIBRA® during which time she led the transition from Phase 1 through to global registration for adults and children with hemophilia A.

Gallia is board-certified in hematology and earned an M.D. and Ph.D. in Molecular and Cellular Biology from the University of Michigan, during which time discovered ADAMTS13, responsible for Thrombotic Thrombocytopenic Purpura (TTP). She completed her residency in internal medicine at Stanford University and a fellowship in hematology at the University of California, San Francisco. She also spent time at the Pasteur Institute during which she received an M.S. in Molecular and Cellular Biology from the University of Paris, VI and she holds a B.A. from the University of California, Berkeley. Gallia was also appointed as a College Fellow to the College of Physicians of Philadelphia in 2021.

Panel Discussion

The Importance Of Early Planning In Cell & Gene Therapy Commercialization

Available On Demand

Successful clinical trials have shown industry stakeholders the value cell and gene therapy (CGT) offers patients now and as the path of future medicine. Despite the clinical benefits, CGT has not yet achieved significant market penetration. In addition to struggling with production roadblocks, companies also face pricing issues and a global healthcare system unprepared for CGT roll out.

This roundtable identified successful strategies that underpin currently-marketed therapies and examined how establishing a framework early in development helps keep long-term production and commercialization goals on track.

Discussion points included:

  • Areas with growth potential for future CGT pipelines
  • How to avoid roadblocks and reduce the cost of CGT development and delivery
  • What actions do you take and what price point can this be sold at??
  • The benefit of advances in technology to production cost, and impact on clinical trial approaches

Speakers

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Moderated By:

Amanda Micklus

Principal Analyst, Datamonitor Healthcare, Informa Pharma Intelligence


As a Senior Pharma Consultant for Informa Pharma Custom Intelligence, Amanda Micklus works with pharma and biotech clients on disease landscaping and market opportunity assessments involving both primary and secondary market research. Amanda has experience in searching and mining Informa’s portfolio of pharma intelligence solutions for valuable insights and regularly contributes content across multiple products including In Vivo, Scrip, and The Pink Sheet. With a focus on research and analysis of corporate strategy and deal-making, Amanda provides analysis of market-moving events that influence business development at leading companies.

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Jeffrey Hung

Chief Commercial Officer, Vigene Biosciences


Jeffrey has over 20 years of experience in the biotechnology industry. Jeffrey is instrumental in Vigene’s $350 million acquisition by Charles River Labs in 2021. He joined Vigene in 2016 and orchestrated the acquisition of Omnia Biologics. He has overseen Vigene’s expansion into GMP manufacturing and new product areas such as biosensors. An experienced entrepreneur, Jeffrey was instrumental in successfully growing GenScript and SABiosciences, two previous companies, to IPO and acquisition stage, respectively. He also previously held the position of Chief Marketing Officer at ATCC. Jeffrey is the author of multiple patents, publications, and book chapters. He holds a Ph.D. in genetics from Cornell University, an MBA from UC Berkeley, and a B.S. in biology from Peking University.

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Shesh Sharma

Sr. Director, Clinical Stage CAR-T, Innolynx


Shesh is a managing principal of Innolynx Inc., a boutique life sciences management consulting firm based in Irvine, CA. Shesh is an experienced Biotech operations executive and management consultant with over fifteen years in life sciences, including five years in cell and gene therapy operations. Shesh has a proven track record of driving strategic, operational, and tactical initiatives covering Pre-clinical through Phase III/launch. Shesh is recognized for his deep expertise in operations strategy, clinical and commercial supply chains, supply planning, CDMO relationship management, GxP sourcing, cold chain logistics, COI/COC business process integration, and related commercial launch operations. Shesh worked with Kite Pharma from Phase II through post-launch and acquisition and helped developed Kite’s capabilities in clinical/commercial supply chain, raw material risk management & governance, end-end business process integration, and revenue cycle management in support of the launch of Kite’s first immunotherapy drug “Yescarta”, both in the US and European markets. Shesh recently worked with Poseida Therapeutics in developing their global supply chain, logistics, and direct raw material sourcing capabilities. Shesh is currently advising a CDMO on their warehouse and 3PL strategies and a cell and gene therapy manufacturer to scale their operations for commercial manufacturing. Shesh has previously worked with top-tier management consulting firms A. T. Kearney and PRTM. Shesh graduated from the Ross School of Business with an MBA in 2001. He also has a MS in Aerospace Engineering from the University of Florida, and a BS in Mechanical Engineering from the Bangalore University, India.

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Ben Cieply, Ph.D

Sr. Consultant Global Pricing and Market Access, ICON


Ben is an experienced consultant with ICON’s Global Pricing, Market Access, and Reimbursement (PMAR) team. He has been largely focused on drugs in development, aiming to help clients understand their asset’s value and price potential and the evidence needed to support this.

Ben’s experience and knowledge of the payer landscape and pricing and access projects spans the US, Europe, and rest of world and emerging markets and has focused multiple engagements on cell and gene therapies.

In Europe, he has worked with HTAs, including early scientific advice with GBA, NICE, HAS, ZIN, and EUnetHTA, and primary research with payers focused on product value, HTA evidence expectations and price implications.

Ben also has a deep understanding of the fundamental mechanisms of cell and gene therapy from his experience as an NIH-NHRA post-doctoral fellow at the University of Pennsylvania where he studied the molecular mechanisms of induced pluripotent stem cells and CRISPR mediated genome editing.

Ben is an author on 25 peer reviewed publications in scientific and medical journals and he earned his Doctorate of Philosophy (Ph.D.) in Cancer Cell Biology from the University of West Virginia School of Medicine.

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Matthew Durdy

Chief Executive Officer, Cell and Gene Therapy Catapult


Matthew Durdy is Chief Executive of the Cell and Gene Therapy Catapult, having been part of the founding team in 2012. Prior to 2020, he was Chief Business Officer and was responsible for strategy, communications, marketing, and business development. He is credited with leading the design and implementation of the commercial model for the highly successful Cell and Gene Therapy Catapult manufacturing center and being a global champion for the early integration of healthcare economics and reimbursement expertise into decision-making and clinical product design.

He began his career in international investment banking and venture capital, and has successfully invested in and managed a number of biotechnology SMEs and regional operations of multinational organizations. He has an MA from the University of Oxford in Pure and Applied Biology, an MBA from the University of Chicago, and is a Fellow of the Chartered Institute for Securities and Investment. He is also Vice Chair of the Board of Trustees of the charity Parkinson’s UK.

Panel Discussion

Navigating The Future Of Cell & Gene Therapy: The Potential For Broader Indication

16 September 2021 | 10:00AM ET

With cell & gene therapy (CGT) already providing life-changing options to patients worldwide, the science used for treatments that have already been approved offer a foundation for developing therapeutics to target many other conditions. Just as off-label drug use is credited with a number of successful healthcare initiatives, similar positives are becoming increasingly likely in the CGT space.

This roundtable will explore the significant opportunities and challenges posed by CGT clinical trials, and how a CRO partner may be uniquely placed to help a pharma innovator navigate them. When there is potential for trials in larger indications where scaling up to meet demand may be required, what are the key considerations and what experience do CROs offer?



Proposed discussion points include:
  • Faster regulatory decision making and streamlining of data
  • Consideration of applying sciences across therapeutic areas
  • The lessons learnt from mRNA COVID-19 vaccine trials

Speakers

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Moderated By:

Kevin Grogan

Managing Editor Europe - Scrip, Informa Pharma Intelligence


Kevin Grogan has been writing about pharmaceuticals for over twenty years in roles that have included online editor for PharmaTimes. After four years freelancing, which involved writing for all the principal titles in the sector, as well as consultancy work with major pharmaceutical companies, he joined Scrip as Managing Editor, Europe, Commercial in the summer of 2017.

Covering all aspects of the pharma industry, Kevin has interviewed pretty much all the leading figures in the sector, both in the UK and globally. A regular attendee at financial and medical conferences worldwide (and moderating at some), he has also appeared on BBC television and radio, ITV and Channel 4 to discuss events in the pharmaceutical industry.

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Dr. Benjamin Yerxa

CEO, Foundation Fighting Blindness


Benjamin Yerxa serves as the CEO for the Foundation Fighting Blindness, the world’s leading private funder of research on retinal degenerations. He also serves as CEO of the Retinal Degeneration Fund, not-for-profit venture philanthropy investment arm. Dr. Yerxa has more than 25 years’ experience in biotechnology, drug development, and in translating promising research discoveries into clinical milestones and treatments. Prior to joining the Foundation, Dr. Yerxa was president and co-founder of Envisia Therapeutics, a company focused on developing novel ocular sustained delivery therapies for the front and back of the eye. Dr. Yerxa holds 60 U.S. patents and is an inventor of DIQUAS™, an innovative treatment for dry eye approved in Japan. Dr. Yerxa earned his PhD in organic chemistry from University of California, Irvine, and BA in chemistry from the University of California, San Diego.

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Martin Lachs

Vice President Global Project Management, ICON


Martin has headed up ICON’s Oncology and Cell Therapeutics Project Management Group since 2012, lending operational and indication expertise across a group of over 240 international project management staff globally, dedicated to oncology and cell therapy drug development. Martin has worked in developing key oncology site networks in the US and the UK and in 2020 was a member of a clinical trial review panel for University of Sydney affiliated hospitals. Martin was at the heart of establishing ICON’s Cell Therapy service capabilities from 2016.

Martin has a BSc in Biotechnology and after completing a PhD thesis (cell biology, breast cancer) at the University of Manchester in the UK, Martin’s career has spanned laboratory research in large bio-pharma, clinical development in CROs and Imaging Core Labs. Martin has held Project Management Professional Certification (PMP), from the Project Management Institute (PMI) for 19 years.

Martin has produced a number of position papers in the Pharmaceutical Press as well as hosting/presenting at numerous international events at scientific and pharma industry meetings. Recently the key focus of his publications has been thought leadership related to Cell Therapies in oncology.

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William T. Andrews, M.D., F.A.C.P.

Chief Medical Officer, Flexion Therapeutics


Dr. Andrews joins Flexion Therapeutics with more than 20 years of biopharmaceutical experience in clinical development, medical affairs, and medical/commercial strategy, including seven product launches. Dr. Andrews most recently served as CMO at Akcea Therapeutics, a global biopharmaceutical company focused on the development and commercialization of RNA-targeting antisense oligonucleotide therapies for rare diseases. Prior to Akcea, Dr. Andrews served as CMO for Acer Therapeutics, where he managed and led clinical development programs for rare diseases in multiple therapeutic areas. In addition, Dr. Andrews held senior leadership positions and roles of increasing responsibility at Aegerion Pharmaceuticals, Santhera Pharmaceuticals, Sepracor, and ClinQuest.

Dr. Andrews practiced Internal Medicine full-time for seven years before joining industry and continued practicing after joining industry through 2012. During this time, he served as Clinical Faculty in Internal Medicine at Harvard Medical School and Attending Physician in Internal Medicine at Brigham and Women’s Hospital in Boston. He earned his B.A. in Biology from Harvard University and his M.D. from Yale University School of Medicine.

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Richard Francis

CEO – Purespring Therapeutics


Richard Francis is the CEO of Purespring Therapeutics a gene therapy company focused on the kidney, he is also an operating partner for Syncona. Prior to his current role, Richard was the CEO of Sandoz a global leader in generics and biosimilars. He was also a member of the Executive Committee of Novartis. Sandoz. Richard joined Novartis from Biogen Idec, where he was most recently Senior Vice President of US Commercial. Prior to this role and during his 13 year career at the company he was the Head of Global Strategy and Marketing, Managing Director of Canada, Managing Director of the UK & Ireland. From 1998 to 2001, Richard was at Sanofi in the United Kingdom, and held various marketing roles across the company’s urology, analgesics and cardiovascular products. he also held sales and marketing positions at Lorex Synthelabo and Wyeth. Richard received a B.A. in economics from Manchester Metropolitan University in the UK.

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