Current Programs
We are creating novel small molecules and antisense oligonucleotides for a variety of serious genetic conditions. With a current focus in neurological disorders, our technology platform can also be leveraged to discover therapeutic candidates across many disease areas.
Disease
Partner
Discovery
Candidate
Clinical
Neurological
Spastic Paraplegia Type 49
TECPR2 Foundation
Genetic Epilepsy
UCB
Pain
Internal Program
ALS
QurAlis
GBA Parkinson's Disease
Silverstein Foundation for Parkinson's with GBA
Other
Undisclosed Genetic Disorder
Vertex