Our broad and active pipeline

With current programs in ophthalmology, neurological and neuromuscular disorders, and rare diseases, Ascidian is developing a broad pipeline to impact significant diseases that cannot be addressed with existing technologies.

Therapeutic Area

Discovery

Lead
Optimization

IND-Enabling

Phase 1/2

ABCA4

Gene targets beyond reach
of current technologies

Clear unmet need and
differentiated from
competitive approaches

First-in-class potential

Ocular

Neuro &
Neuro-
Muscular

Other
Areas

Rewriting RNA to stop blindness

Ascidian’s lead program targets ABCA4 retinopathy, including Stargardt disease.

Stargardt disease is caused by mutations in the ABCA4 gene which lead to progressive retinal degeneration and vision loss, typically beginning in childhood and young adulthood. Affecting more than 30,000 individuals in the United States alone, Stargardt disease is the most common form of inherited macular degeneration.

There are no approved treatments.

Traditional gene therapies or gene editing approaches are not sufficient to meet patient needs. The ABCA4 gene is too large to be delivered with a single AAV construct. Too many patient mutations exist to feasibly address each mutation via mutation-specific base editing.

By rewriting RNA, Ascidian's approach has the potential to treat most patients with a single dose of a single exon-editing RNA therapeutic.