Full Year 2023 Investor Presentation slide image

Full Year 2023 Investor Presentation

92 Investor presentation Full year 2023 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 Blood
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