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1 ©2012 Vertex Pharmaceuticals Incorporated
Gene editing to treat cystic fibrosis and other
rare diseases
Andrew J. Fadden Ph.D., Vertex Pharmaceuticals
EURORDIS Workshop on Genome Editing
4 November 2016
©2016 Vertex Pharmaceuticals Incorporated
Agenda
• Reflect on today’s presentations
• Talk about Vertex’s commitment to people living with cystic
fibrosis
• Talk about how gene editing will play a role in Vertex’s mission
• Talk about how Vertex’s commitment to treatment of rare
diseases is moving beyond cystic fibrosis
©2016 Vertex Pharmaceuticals Incorporated
2
©2016 Vertex Pharmaceuticals Incorporated
3
Vertex employees are united around a shared vision: that together
we can make a positive impact on the lives of all people with cystic
fibrosis. We partner with the cystic fibrosis community around the
world, inspired by the patients and families who are directly
affected by a diagnosis of cystic fibrosis.
Vertex is developing a range of innovative treatments for
people living with cystic fibrosis
©2016 Vertex Pharmaceuticals Incorporated
4
Ivacaftor
Lumacaftor + ivacaftor
mRNA therapies
VX-###
Gene editing
therapies
Future combinations
5 ©2016 Vertex Pharmaceuticals Incorporated
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represents 1000 people
with cystic fibrosis
Future
Combinations
How Vertex aspires to help all people living with cystic
fibrosis
6 ©2016 Vertex Pharmaceuticals Incorporated
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represents 1000 people with CF
How Vertex plans to help all people living with cystic
fibrosis
©2016 Vertex Pharmaceuticals Incorporated
7
Beyond cystic fibrosis – haemoglobinopathies
©2016 Vertex Pharmaceuticals Incorporated
8
Vertex is collaborating with CRISPR
Therapeutics to develop gene editing-
based treatments for sickle cell
disease and beta-thalassaemia
Blood cells are
removed from the
patient
The stem cells are
treated with the
gene editing
technology
The corrected cell
are returned to the
patient
Treatment schematic adapted from CRISPR Therapeutics
Blood cells are
sorted to obtain
blood stem cells
We are all in the same boat…….
©2016 Vertex Pharmaceuticals Incorporated
9
Thanks for listening
Any questions?
©2016 Vertex Pharmaceuticals Incorporated
10

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Gene editing to treat cystic fibrosis_Vertex

  • 1. 1 ©2012 Vertex Pharmaceuticals Incorporated Gene editing to treat cystic fibrosis and other rare diseases Andrew J. Fadden Ph.D., Vertex Pharmaceuticals EURORDIS Workshop on Genome Editing 4 November 2016 ©2016 Vertex Pharmaceuticals Incorporated
  • 2. Agenda • Reflect on today’s presentations • Talk about Vertex’s commitment to people living with cystic fibrosis • Talk about how gene editing will play a role in Vertex’s mission • Talk about how Vertex’s commitment to treatment of rare diseases is moving beyond cystic fibrosis ©2016 Vertex Pharmaceuticals Incorporated 2
  • 3. ©2016 Vertex Pharmaceuticals Incorporated 3 Vertex employees are united around a shared vision: that together we can make a positive impact on the lives of all people with cystic fibrosis. We partner with the cystic fibrosis community around the world, inspired by the patients and families who are directly affected by a diagnosis of cystic fibrosis.
  • 4. Vertex is developing a range of innovative treatments for people living with cystic fibrosis ©2016 Vertex Pharmaceuticals Incorporated 4 Ivacaftor Lumacaftor + ivacaftor mRNA therapies VX-### Gene editing therapies Future combinations
  • 5. 5 ©2016 Vertex Pharmaceuticals Incorporated                                                                             represents 1000 people with cystic fibrosis Future Combinations How Vertex aspires to help all people living with cystic fibrosis
  • 6. 6 ©2016 Vertex Pharmaceuticals Incorporated                                                                             represents 1000 people with CF How Vertex plans to help all people living with cystic fibrosis
  • 8. Beyond cystic fibrosis – haemoglobinopathies ©2016 Vertex Pharmaceuticals Incorporated 8 Vertex is collaborating with CRISPR Therapeutics to develop gene editing- based treatments for sickle cell disease and beta-thalassaemia Blood cells are removed from the patient The stem cells are treated with the gene editing technology The corrected cell are returned to the patient Treatment schematic adapted from CRISPR Therapeutics Blood cells are sorted to obtain blood stem cells
  • 9. We are all in the same boat……. ©2016 Vertex Pharmaceuticals Incorporated 9
  • 10. Thanks for listening Any questions? ©2016 Vertex Pharmaceuticals Incorporated 10