Investor Presentation: First Nine Months of 2020
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Investor presentation
First nine months of 2020
Novo NordiskⓇ
Novo Nordisk and bluebird bio join forces in next-generation
genome editing for children and adult patients with haemophilia A
Potential curative treatment in
haemophilia A
.
mRNA-based megaTAL TM-driven
gene editing
Highly specific and efficient way
to silence, edit or insert genetic
components.
• Allows for gene editing in all age
groups
کلے
AAV
Gene
Nucleus
Targeting megaTAL mRNA
Lipid
nanoparticle
Gene
bluebird bio/Novo Nordisk's joint
approach
•
megaTAL™: Proprietary, patented
technology, broad IP
•
Correcting FVIII-clotting factor
deficiency
любовная
Protein
• Potential lifelong effect
.
Hepatocyte
Possibility to explore additional
therapeutic targets
AAV: Adenovirus vector; BDD: B-domain deleted; hFVIII: human factor VIII; LNP: Lipid nanoparticle; mRNA: messenger ribonucleic acid; TAL: transcription activator-like.View entire presentation