Pipeline Overview

Our initial focus is on developing a broad pipeline of transformative gene therapy product candidates designed to treat patients suffering from lysosomal storage diseases, lung diseases, muscular dystrophies and ophthalmic diseases. We are developing a diverse pipeline of wholly owned and partnered programs for both rare and large market diseases.

4DMT THERAPEUTIC PIPELINE

* Expected timing of milestone. FIH refers to first-in-human, Phase 1 or Phase 1/2, clinical studies.
** We are responsible for development of this product candidate and Roche has an exclusive option to assume development and commercialization at its sole cost and expense. Such option may be exercised prior to pivotal trial initiation.
§ We are independently optimizing lead assets in this field. If we elect to declare product candidate(s) in this field, Roche could exercise the option to license, further develop and commercialize this product candidate at any time prior to pivotal trial initiation.
Proof of concept studies for 4D-710 were conducted in a pig model of CF and utilized a species-specific (ie, pig-specific) vector rather than the primate-specific 4D-A101 vector which is the vector for CF product candidate 4D-710.