Assessing the clinical and commercial landscape for ATMPs – A Pharma/Biotech perspective

8.30      [Panel] Investor perspective: The opportunities of cell & gene therapy and why now?

  • Landmark deals and progress in advanced therapies
  • The commercial impact of a sustainable advanced therapy industry
  • What scientific or political issues (eg. Brexit, Trump Administration) will likely impact the ability to maintain robust pipelines in the cell & gene therapy space?
  • What analysis can we provide to help stakeholders better assess scientific, regulatory, and public policy issues that may have a dramatic impact on venture capital investments in the advanced therapeutic field and within specific disease areas? Insight into the financial hypothesis for advanced therapies treatment and tools
  • What is the current funding environment directed to novel drug R&D and how does this change at different phases of development?
  • Identifying robust science with a clear pathway to a differentiated approved product in a busy marketplace. How can you determine risk, reward and revenue?
  • What is the decision-making process when investing in compelling technologies that have commercial potential?
  • Lifecycle of advanced therapeutics: From discovery to patent expiration, and what that means for investors and drug makers.

Chair: Albert Ganyushin, former Head of International Listings, EURONEXT
Dr Genghis Lloyd-Harris
, Partner, Abingworth
Ferran Prat, VP Strategic Industry Ventures, MD Anderson Cancer Center
Dr Dmitry Kuzmin, Managing Partner, 4BIO Capital
Maina Bhaman
, Director of Healthcare Investment, Touchstone Innovations
Dr Sara Núñez-García
, Principal, Sofinnova Partners

      [Panel] Biotech Perspective: The opportunities of cell & gene therapy and why now?

  • What the major barriers are in bringing these new therapies into mainstream clinical practice and achieving market penetration into existing standards of care?
  • The next, pivotal, decade will see several therapies currently in Phase II/III launch into the market and begin to set the precedent for those that follow. What are the unprecedented hurdles, and risks, when taking these novel therapies from a clinical trial setting to real world practice.

Chair: Dr Ian Campbell, Director, Health and Life Sciences, Innovate UK
Dr Thomas Taapken,
CFO, Medigene
Dr John Beadle, CEO, PsiOxus Therapeutics
Dr Stephane Boissel, CEO, TxCell
Dr Joe Dupere
, CEO, Rexgenero

10.00      Fireside Chats: Evaluation of the clinical success for Ex-Vivo Gene Therapies

  • How encouraging progress has been made in the clinical use of gene-based therapy for a variety of diseases, most notable successes for severe combined immunodeficiency disease (SCID-X1), adenosine deaminase-severe combined immunodeficiency (ADA-SCID) and Leber’s congenital amaurosis (LCA).
  • Understanding the significant role of vector design, architecture of therapeutic molecule and delivery to the efficacy of these therapeutics.
  • The success of adoptive immunotherapy using genetically modified T cells and how factors influencing continued success – eg. co-stimulatory pathways, understanding the tumour microenvironment.
  • How CAR-T therapeutic success in hematologic cancers can provide a framework for investigational treatment of solid tumour malignancies.

Chair: Vibhor Gupta, Independent Researcher, Pangaea
Dr Sven Kili
, VP & Head of Gene Therapy Development, GSK
Dr Rick Morgan, VP, Immunotherapy, bluebird bio

10.25      How is the biopharma industry approaching business development and mergers/acquisitions in the advanced therapeutic space?

  • Profiling key recent deals and initiatives involving pharma, biotech and academia – what do they tell us about collaboration, innovation and overall industry trends and drivers that we can expect to see develop through 2017 and beyond?

Dr Angus Grant, Corporate VP, Business Development, Celgene

10.45      Refreshments & Networking

11.15      [Panel] Bringing the benefits of advanced therapeutics to patients

  • What scientific evidence will be necessary to support the authorization of new gene therapies?
  • What are the realities of bringing advanced therapeutics (stem cells, gene therapy, immune-oncology) to the patient bedside [Insight from clinicians on the frontline of care]
  • Will clinical and regulatory progress match patient expectations?

Chair: Dr Tony Jones, Director of Business Development, One Nucleus
Professor Adrian Thrasher
, Molecular Immunology Unit, UCL Institute of Child Health; and Head, Gene Therapy Programme, Great Ormond Street Hospital
Professor Marc Turner
, Medical Director, Scottish National Blood Transfusion Service
Dr Magda Papadaki, Head of Manufacturing Innovation, The Association of the British Pharmaceutical Industry
Professor Uta Griesenbach
, Professor of Molecular Medicine, National Heart and Lung Institute, Imperial College; and President of the British Society for Gene and Cell Therapy

      [Panel] Industrialization of the cell and gene therapy industry

  • Bridging the gap from production to patients
  • At what stage do cell therapy companies typically start to consider up-scaling manufacturing and what upstream/downstream issues can that create?
  • The development of flexible technology platforms to allow gene and cell therapies that are adapted to each individual.
  • How the high price of gene therapies reflects the cost of preclinical development, manufacturing, and distribution
  • Driving the transition of cell therapies from “point of care” therapies to “off-the-shelf” pharmaceuticals
  • Bringing down the costs-of-good: making cell therapies cost-effective and widely available to patients in the world
  • Leveraging the transformative potential of gene editing in the cell & gene therapy industry
  • Gene therapies have the potential to deliver a substantial, long-lasting benefit to patients on a single administration, which may offset the cost of the standard treatment of the condition and its complications. Nevertheless, the high costs associated with gene therapies, they will challenge the standard reimbursement policies of governments, healthcare systems and insurance companies. How do we expect pricing and payments to work for gene therapies?

Dr David Sourdive, EVP Technical Operations, Cellectis
Timothy Moore, EVP Technical Operations, Kite Pharma
Dr Ger Brophy
, GM, Cell Therapy, GE Healthcare, Life Sciences
Dr Madhusudan Peshwa
, Chief Scientific Officer, Executive Vice President, MaxCyte

12.25      [Panel]
Identify barriers and opportunities in infrastructure supply chains

  • Translational challenges of creating a supply chain for commercialization to a much broader market as well as passing typical drug regulatory body scrutiny.
  • What investment is required to provide reproducible and GMP-compliant products, at a large scale, which can net substantial profit?
  • Key considerations for centralized manufacturing of cell therapies – Cost and difficulties in establishing standardized quality assurance programs and reliable transport systems for the movement of patient cells from the clinic through the manufacturing facility and back.

Senior Representative, TrakCel

12.50      [Panel]
 Getting advanced therapies to frontline patient care. What are the challenges associated with market access, pricing & reimbursement?

  • What are the unique regulatory and reimbursement challenges for ATMPS? Understanding the complex HTA environment in Europe for advanced therapeutics
  • How the requirements of payers and HTA bodies are a must-know for developers seeking pricing and reimbursement

Chair: Dr Tim Farries, Director of Regulatory Affairs, ATMPs, ERA Consulting
Dr Nick Crabb
, Programme Director, Scientific Affairs, NICE
Tay Salimullah, Global Pricing & Market Access Director, Cell & Gene Therapies, Novartis Pharma AG

13.20      Lunch & Networking

      Company Presentations (8 x early stage | 8 x late stage)

  • Presentations from a range of publicly listed and private life science companies looking to raise finance and/or find partners.

Biotechs include:
Aris Constantinides
, General Partner, Kreos Capital
Dr Sara Núñez-García
, Principal, Sofinnova Partners
Dr Robert Jan Lamers, CEO, Arthrogen
Dr David Venables, CEO, Synpromics
Dr Gregg Sando
, CEO, Cell Medica
Dr Michael D Leek, 
Chief Executive, TC BioPharm
Dr Carlos de Sousa
, CEO, Immunicum AB
Dr Joe Dupere
, CEO, Rexgenero
Peter Rayson
, CEO, Telocyte

16.15      Refreshments & Networking

16.45      Chairpersons remarks

Dr Patrik Frei, CEO, Venture Valuation

16.50      Risk assessment in the development of advanced therapies – academic interest versus commercial reality
Prof Mark Lowdell, Director of Cellular Therapeutics & RFH/UCL Cancer Biobank, University College London

17.15      Closing Panel: Forecasts and Predictions
The gene therapy industry is predicted to achieve revenues of $204million in 2020 and the immuno-oncology market up to $34billion by 2024. 

  • How are earlier stage investors helping develop and build the next wave of advanced therapies companies?
  • What are the additional technologies/targets that are being approached in recent times for oncology; e.g. ADCs, RNA targeting moieties and DNA damage repair platforms
  • The evolving and dynamic role that pharma and corporate venture plays in early stage finance and industry collaborations
  • The challenges and opportunities in assessing profitable and revolutionary medicines and achieving predicted revenues

Dr Markus Goebel, Managing Director, Novartis Venture Fund
Robin Davison, Healthcare Analyst, New Science Global Healthcare Fund
Lucas De Breed
, Director, INKEF Capital

17.45      Networking Drinks Reception