OPA1 Gene Mutation
2
Pipeline Programs
1
Companies
2
Clinical Trials
1 recruiting
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
1
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
PYC TherapeuticsAustralia - Nedlands
2 programs1
1
PYC-001Phase 1/21 trial
PYC-001Phase 11 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2024
2025
2026
2027
2028
PYC TherapeuticsPYC-001
PYC TherapeuticsPYC-001
Clinical Trials (2)
Total enrollment: 36 patients across 2 trials
Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)
Start: Sep 2025Est. completion: Jan 202818 patients
Phase 1/2Recruiting
SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)
Start: Oct 2024Est. completion: Aug 202618 patients
Phase 1Active Not Recruiting
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
1 actively recruiting trials targeting 36 patients
1 companies competing in this space
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.