Gene-based medicines

Transforming the lives of patients
with serious diseases

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Programs

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Hemoglobinopathies

Aiming to treat β-thalassemia and sickle cell disease with gene-edited hematopoietic stem cells

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Immuno-Oncology

Creating the next generation of cell therapies for cancer enabled by gene editing

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In Vivo

Editing cells inside the body to treat genetically-defined diseases

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Regenerative Medicine

Broadening the applications of stem cells through gene editing

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CRISPR/Cas9

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Rapidly translating a revolutionary technology into transformative therapies

Rapidly translating a revolutionary technology into transformative therapies

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