Pipeline

We are advancing a pipeline of genetic medicines to deliver functional cures for patients with severe lifelong diseases in both large and rare populations.

Discovery

Preclinical

IND-Enabling

Clinical

Internal Programs

Rare Obesity Indication (Undisclosed)

Fabry Disease

Research collaborations

In vivo CAR-T

Pharma (undisclosed)

Obesity

Pharma (undisclosed)

HIV

Gates Foundation