01 / Cure genetic diseases

Why it matters

There are over 10,000 known genetic disorders caused by mutations in a single gene, in multiple genes or by chromosomal abnormality. Some are life-threatening, while others cause severe health problems that significantly reduce quality of life. Most are currently not curable. We seek out platform-based solutions that could intervene at the gene level, with the aim to prevent metabolic diseases, degenerative conditions like Alzheimer’s disease, and blindness.

What if a baby born with cystic fibrosis or sickle cell anemia could be treated with cell and gene therapy in the first months of life, preventing these diseases from ever taking hold? What if medicines could address the root of the problem, preventing conditions with strong genetic predispositions?