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.
Disorder
Partner
Discovery
Preclinical
Clinical
Internal
Pain (antisense oligo)
Q-State - Owned
Pain (antisense oligo)
Q-State - Owned
Pain (antisense oligo dual target)
Q-State - Owned
Pain (small molecule)
Q-State - Owned
Epilepsy (antisense oligo)
Q-State - Owned
Undisclosed target 1
Q-State - Owned
Undisclosed target 2
Q-State - Owned
Partnered
Spastic Paraplegia Type 49
TECPR2 Foundation
ALS Target
QurAlis
Select Genetic Epilepsy
UCB
Parkinson's Disease Target
Silverstein Foundation
Undisclosed Genetic Disorder
Vertex