Deep Pipeline of Rare Monogenic CNS Disease Therapies*

Indication
Gene
Discovery
IND Enabling
Clinical Development
Leading Programs
GM1
GLB1
1H 2020
FTD
PGRN
1H 2020
Krabbe Disease
GALC
2H 2020
Other Programs
Program 4
2021
Programs 5
2021

*Option to license 7 additional indications