European Gene Therapy Markets

1
European Gene Therapy Markets

• Analyst Briefing
• hosted by Christian Downton
• 10th December 2pm GMT (3pm CET)

2
Agenda

• Definition of Gene Therapy
• Gene Therapy Now
• Gene Therapy in 2010
• Administration
• Impact on Established Markets
• European Revenues
• Collaborations
• Round-up and Questions

3
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

4
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

5
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
CNS
Infectious Dis
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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

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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

8
PolI

• A major hurdle for Gene Therapy will be
  end-users perceiving it as unsafe.

9
European Revenues

• Gene Therapy for
• Cancers from 2005
• Vascular Diseases from 2005
• Monogenic Diseases from 2006/7

10
Collaborations, Now and Future
Now
Schering acquired Collateral Thx
Celltech
Targeted Genetics
Avigen
Bayer
Chiron
Ribozyme
Future
Aventis
Novartis
AstraZeneca
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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

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Contact Us

• For further information on
• European Gene Therapy
• or our other services
• email enquiries_at_frost.com
• Tel [phone]
• web www.frost.com