Our pipeline:
multiple modalities and novel targets
We are developing disease-modifying therapies for several genetic conditions with a high degree of unmet need. While our initial focus is in neurology, our stereopure chemistry platform may unlock the potential for transformative therapies in many therapeutic areas.
Our diverse discovery programs aim to explore the potential of our platform across a broad range of serious genetic diseases.
| Therapeutic Area/Target | Modality | Discovery | Preclinical | Clinical | Rights |
|---|---|---|---|---|---|
| ALS and FTD C9orf72 |
|
||||
| Huntington's disease mHTT SNP3 |
|
Takeda 50:50 option |
|||
| Spinocerebellar ataxia 3 ATXN3 |
|
||||
| CNS diseases Multiple |
|
||||
| Duchenne muscular dystrophy Exon 53 |
|
100% | |||
| global | |||||
| AATD - Lung and liver disease SERPINA1 |
|
||||
Therapeutic Modality
ADAR editing (AIMers)
ALS: Amyotrophic lateral sclerosis; FTD: Frontotemporal dementia; CNS: Central nervous system
CNS
ALS and FTD
C9orf72
| Modality | Discovery | Preclinical | Clinical |
|---|---|---|---|
|
|
|||
|
Partner Takeda 50:50 option |
|||
Huntington's disease
mHTT SNP3
| Modality | Discovery | Preclinical | Clinical |
|---|---|---|---|
|
|
|||
|
Partner Takeda 50:50 option |
|||
Spinocerebellar ataxia 3
ATXN3
| Modality | Discovery | Preclinical | Clinical |
|---|---|---|---|
|
|
|||
|
Partner Takeda 50:50 option |
|||
CNS diseases
Multiple
| Modality | Discovery | Preclinical | Clinical |
|---|---|---|---|
|
|
|||
|
Partner 100% global |
|||
Duchenne muscular dystrophy
Exon 53
| Modality | Discovery | Preclinical | Clinical |
|---|---|---|---|
|
|
|||
|
Partner 100% global |
|||
HEPATIC (GalNAc)
AATD - Lung and liver disease
SERPINA1
| Modality | Discovery | Preclinical | Clinical |
|---|---|---|---|
|
|
|||
|
Partner 100% global |
|||
Therapeutic Modality
ADAR editing (AIMers)
ALS: Amyotrophic lateral sclerosis; FTD: Frontotemporal dementia; CNS: Central nervous system
