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 Modality

Silencing

Splicing

ADAR editing (AIMers)

ALS: Amyotrophic lateral sclerosis; FTD: Frontotemporal dementia; CNS: Central nervous system

CNS

ALS and FTD

C9orf72

Huntington's disease

mHTT SNP3

Spinocerebellar ataxia 3

ATXN3

CNS diseases

Multiple

Duchenne muscular dystrophy

Exon 53

HEPATIC (GalNAc)

AATD - Lung and liver disease

SERPINA1

Therapeutic Modality

Silencing

Splicing

ADAR editing (AIMers)

ALS: Amyotrophic lateral sclerosis; FTD: Frontotemporal dementia; CNS: Central nervous system

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