Transthyretin-Mediated Amyloidosis
2
Pipeline Programs
2
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
2
0
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
RNA Therapeutic
1100%
+ 2 programs with unclassified modality
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Alnylam PharmaceuticalsCAMBRIDGE, MA
2 programs1
ALN-TTRSC04Phase 11 trial
ConTTRibute ATTR Amyloidosis RegistryN/A
Active Trials
AstraZenecaCAMBRIDGE, United Kingdom
1 program1
Eplontersen Solution for InjectionPhase 1RNA Therapeutic1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2023
2024
2025
2026
AstraZenecaEplontersen Solution for Injection
Alnylam PharmaceuticalsALN-TTRSC04
Clinical Trials (2)
Total enrollment: 108 patients across 2 trials
A Study to Assess the PK, PD, Safety and Tolerability of Eplontersen in Healthy Chinese Volunteers
Start: Aug 2024Est. completion: Nov 202412 patients
Phase 1Completed
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects
Start: Jan 2023Est. completion: Sep 202596 patients
Phase 1Completed
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
2 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.