Pipeline & Programs
Pipeline
<u>Pipeline</u>

Advancing towards tailored treatments for complex neurological disorders

The Alcyone pipeline features two candidates in or approaching clinical development and a number of additional candidates under preclinical development. Our streamlined approach facilitates application of technical and disease-area expertise for rapid progression of candidates to the clinic.

PLATFORM Candidate Indication Concept Preclinical IND-Enabling CLINICAL PHASES
Phase 1/2 Pivotal
Patient Prevalance
Undisclosed
> 10,000
Other x-linked dominant disorders
> 10,000
Vectorized Exon Skipping Vectorized Exon Skipping
Undisclosed
> 15,000
Undisclosed
> 15,000
Promoter Modulation Promoter Modulation
Undisclosed
> 15,000
Undisclosed
> 30,000