Recommended
Recommended
More Related Content
Similar to Gene editing to treat cystic fibrosis_Vertex
Similar to Gene editing to treat cystic fibrosis_Vertex (20)
More from EURORDIS Rare Diseases Europe
More from EURORDIS Rare Diseases Europe (20)
Recently uploaded
Recently uploaded (20)
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
- 7. ©2016 Vertex Pharmaceuticals Incorporated 7
- 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