TTR mRNA Inhibitors
2 drugsAbout TTR mRNA
Transthyretin mRNA (TTR mRNA) encodes for transthyretin protein, which is primarily synthesized in the liver. The TTR protein transports thyroxine and retinol (vitamin A) in the blood.
TTR mRNA is a druggable target for modulating transthyretin protein production, offering a therapeutic avenue when aberrant protein levels or misfolded forms contribute to disease. There is currently no genetic evidence data available for this target.
Two FDA-approved drugs, ONPATTRO and AMVUTTRA, target TTR mRNA using siRNA modalities. These drugs, developed by Alnylam Pharmaceuticals, address therapeutic areas outside the common categories of diabetes, cancer, or autoimmune diseases.
Strategic Insights
ℹ️ How we calculate- White space opportunity in Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy with only 1 trials.
Top TTR mRNA Drugs
Alnylam Pharmaceuticals is the only company with approved drugs targeting TTR mRNA.
High market concentration indicates potential entry barriers; strategic partnerships or novel approaches may be necessary to compete.
TTR mRNA Drug Modality Landscape
Modalities
Routes of Administration
TTR mRNA requires biologic approaches (sirna), likely due to its structure or location.
Consider exploring alternative modalities to differentiate from existing therapies and potentially improve delivery or efficacy.
TTR mRNA Clinical Trials 29 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 13 | 12 | 0 | 1 | 100% |
| Phase 2 | 4 | 4 | 0 | 0 | 100% |
| Phase 3 | 9 | 6 | 0 | 2 | 100% |
| Phase 4 | 3 | 2 | 1 | 0 | 67% |
Top Sponsors
By Modality
TTR mRNA Drug Approval Timeline (2018 - 2022)
The first drug targeting TTR mRNA was approved in 2018 (ONPATTRO), with the most recent approval in 2022 (AMVUTTRA).
Relatively recent entry suggests market growth potential, but also a need to establish long-term efficacy and safety.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 1 companies competing
- • Market share by company
Full Drug Portfolio
- • All 2 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 2-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Competition: Moderate (9 sponsors)
- • White space: 10 underexplored indications
- • Success rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Calculate These Metrics
Target Attractiveness Score
A 0-100 score based on trial activity, sponsor diversity, and completion rates. Calculated from 19 clinical trials targeting TTR mRNA.
Completion rate: Percentage of trials that reached their planned endpoint. Trials terminated early, withdrawn, or suspended are not counted—these often indicate safety issues, lack of efficacy, or strategic pivots.
- Highly Attractive (80+): High trial activity, many sponsors, strong completion rates
- Attractive (60-79): Good trial activity and validation
- Moderate (40-59): Moderate interest from sponsors
- Low (under 40): Limited trial activity or validation concerns
Strategic Insights
Auto-generated insights based on trial analytics including competition intensity, white space opportunities, modality shifts, and failure patterns. We analyze trial sponsors, phases, indications, and outcomes.
Risk Signals
- High Competition: Many sponsors competing for this target (may reduce market opportunity)
- High Failure Risk: Low trial completion rates suggest development challenges
- Low Validation: Limited trial activity or poor outcomes indicate uncertain viability
- White Space Available: Underexplored indications present opportunities