genetic correction

Genetic Correction of Inherited Hemoglobin Disease

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Genetic Correction of Inherited Hemoglobin Disease

300 million families with Hb gene diseases

CRISPR-CAS9

Treatment strategy

No Hb = disease

Patient mutations

Hemoglobin gene

Cure with functional fetal Hb

CRISPR THERAPY

Fetal hemoglobin gene

Fetal hemoglobin (HbG/ HbA)

100% fetal Hb increase after genetic modification

1 22

Untreated cells

Genetically modifiedcells

Treatment benefiting millions by 2035

2015 2035

• Scientific proof of principle

• 2 innovation disclosures• 2 research hospitals• World class mentors

• Investors• International biotech• Clinical trials for safety &

efficacy

• Ethics• Politics

2025

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