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.

During a four-year term, Wave and Takeda may collaborate on up to six preclinical targets at any one time.
ALS: Amyotrophic lateral sclerosis; FTD: Frontotemporal dementia; CNS: Central nervous system; OLE: Open-label extension

CNS

Huntington's disease

Allele-selective silencing

ALS and FTD

Variant-selective silencing

Spinocerebellar ataxia 3

Silencing

CNS diseases

Duchenne muscular dystrophy

Exon skipping

ADAR editing

HEPATIC

Alpha-1 antitrypsin deficiency

ADAR editing

OPHTHALMOLOGY

Retinal diseases

During a four-year term, Wave and Takeda may collaborate on up to six preclinical targets at any one time.
ALS: Amyotrophic lateral sclerosis; FTD: Frontotemporal dementia; CNS: Central nervous system; OLE: Open-label extension

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