Our pipeline

A pipeline of life-changing gene therapy treatments

Our Gaucher disease and Parkinson’s disease programs reflect our ambition to provide innovative, one-time treatments that change the lives of people with debilitating chronic diseases.

Disease

Research

IND-Enabling

Phase 1/2

Phase 3

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Gaucher Disease Type 1 FLT201

Phase 1/2

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GBA1-linked Parkinson’s Disease

Research

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