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European Gene Therapy Markets Analyst Briefing hosted by Christian Downton 10th December 2pm GMT (3pm CET) Agenda • • • • • • • • Definition of Gene Therapy Gene Therapy Now Gene Therapy in 2010 Administration Impact on Established Markets European Revenues Collaborations Round-up and Questions EU Gene Therapy 10th Dec 2002 Defining ‘Gene Therapy’ A therapeutic whose main aim is to genetically manipulate somatic cells for the benefit of disease progression, but not primarily to stimulate the immune system • • • • • Viral vector delivery Non-viral vector delivery Ribozymes Not antisense Not vaccines EU Gene Therapy 10th Dec 2002 Gene Therapy in 2002 15 products in late-stage trials Cancer 40% • • • • Vascular 40% Monogenic 20% Proof-of-principle desperately sought Companies cutting early-stage trials Some good results so far Poor reputation EU Gene Therapy 10th Dec 2002 Gene Therapy in 2010 • Cancer, Vascular Diseases indications beginning to be expanded • First products expected to arrive in 2005 • Gene Therapy will have filled niche where other therapies fail • Targeted positioning and controlled expression gene therapies emerging • Trials underway for: Inflammatory EU Gene Therapy CNS Infectious Dis 10th Dec 2002 Administration • Likely to be a limiting factor in uptake • Commonly • • • • Intra-tumoural injection (cancer) Via catheter (IHD) Via injection (PVD) Inhaled (cystic fibrosis) • Possibility for tissue-targeting in future • Most popular vectors are viral • AAV • Adenoviral EU Gene Therapy 10th Dec 2002 Impact on Established Markets • Products unlikely before 2005 • Impact on cancer markets • Initial focus on high unmet need • Expanding indications past 2009 • Impact on vascular disease markets • Initial focus on patients unresponsive to bypass • Once established, likely to be used earlier • Impact on monogenic disease markets • Very real threat EU Gene Therapy 10th Dec 2002 PolI ‘A major hurdle for Gene Therapy will be end-users perceiving it as unsafe.’ European Revenues European Gene Therapy Markets ($ millions), 2002-2010 1200 Gene Therapy for • Cancers from 2005 • Vascular Diseases from 2005 • Monogenic Diseases from 2006/7 1000 800 600 400 200 0 2002 EU Gene Therapy 2003 2004 2005 2006 2007 2008 2009 2010 10th Dec 2002 Collaborations, Now and Future Now Schering acquired Collateral Thx Avigen Bayer Targeted Genetics Celltech Ribozyme Chiron Future Aventis EU Gene Therapy Novartis AstraZeneca 10th Dec 2002 Round-up and Questions • Gene Therapy for Cancer and Vascular Diseases from 2005, Monogenic to follow • Moderate revenues in Europe, growing to $1.1bn in 2010 • Market noise and potential huge after first products reach market • Impact on established markets • Partnerships already built but many more expected EU Gene Therapy 10th Dec 2002 Contact Us For further information on European Gene Therapy or our other services email [email protected] Tel +44 (0) 20 7343 8383 web www.frost.com EU Gene Therapy 10th Dec 2002
