Investor Presentation First Nine Months of 2022
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Investor presentation First nine months of 2022
Novo Nordisk and 2seventy bio extend partnership in next-
generation genome editing for people with haemophilia A
Lifelong correction via a unique modality
Mode of action
☐ Potentially lifelong correction of FVIII deficiency
FVIII gene engineered and packed in an AAV vehicle
Nuclear
translocation
megaTAL
protein
megaTAL
AAV-F8
Nucleus
Normal
Chromosome
Double strand
break
FVIII gene
introduced
Transfer and
unpacking
Translation
megaTAL
Utilising the skills of both 2seventy bio and Novo Nordisk
Utilisation of megaTAL™ technology, in-
2seventybio vivo mRNA manufacturing/purification
platform, and gene editing know-how
Ⓡ
novo nordisk
Haemophilia A understanding and protein
and molecular engineering capabilities
LNP-megaTAL
AAV vector
with N8 gene
(PoC design)
(Рос
PoC: Proof-of-Concept; AAV: Adeno-associated virus; Rag2: recombination-activating gene; F8: Factor 8
MRNA
Endocytosis
and endosome
escape
2 component
drug format
AAV-N8
F8 Rag2 mouse
Hepatocyte
Liver-
Novo NordiskⓇ
specific
FVIII
expression
LNP-formulated surrogate
megaTAL targeting site
specific locus
BloodView entire presentation