hTR Inhibitors
1 drugsAbout hTR
Human telomerase RNA component (hTR) is the RNA subunit of the telomerase ribonucleoprotein complex, essential for maintaining telomere length. Telomerase prevents telomere shortening during cell division, a process crucial for genome stability. Reactivation of telomerase in cancer cells allows them to bypass normal cell division limits.
hTR is a compelling oncology target due to its role in cancer cell immortality. There is currently no genetic evidence directly linking hTR mutations to specific diseases, suggesting therapeutic strategies focus on modulating overall telomerase activity.
hTR is targeted by one FDA-approved drug, RYTELO (small molecule), developed by GERON for oncology indications. RYTELO received its first approval in 2024, marking the beginning of hTR-targeted therapies.
Top hTR Drugs
GERON is currently the only company with an approved hTR-targeting drug.
The market is highly concentrated, suggesting high barriers to entry or a first-mover advantage for GERON.
hTR Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets hTR, using small molecule modality.
The absence of other modalities represents a whitespace opportunity for novel therapeutic approaches like oligonucleotides or gene therapies.
hTR Clinical Trials 18 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on hTR. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 8 | 2 | 2 | 4 | 50% |
| Phase 2 | 9 | 5 | 2 | 2 | 71% |
| Phase 3 | 1 | 0 | 0 | 1 | - |
Top Sponsors
By Modality
Top Conditions
Top Drugs
hTR Drug Approval Timeline (2024 - 2024)
RYTELO was first approved in 2024, marking the beginning of hTR-targeted therapies.
The recent approval suggests a growing interest in hTR as a target, but it is too early to assess market saturation.
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Competitive Landscape
- • 1 companies competing
- • Market share by company
Full Drug Portfolio
- • All 1 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 1-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 9 (2 companies)
- • Completion rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Count Trials
- Trials: phase 1-4 interventional trials on ClinicalTrials.gov that started in 2008 or later and test an FDA-approved drug acting on hTR. A trial counts if an approved drug acting on hTR is in any arm, including as a comparator, and a trial that tests two such drugs counts once. Trials that test only unapproved drugs are not counted. A trial listed as phase 1/2 counts as phase 1, and phase 2/3 as phase 2.
- Active: recruiting, not yet recruiting, enrolling by invitation, or active and no longer recruiting.
- Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn. Completed means the trial ended as planned; it does not mean the drug worked. Suspended trials are not counted as ended.
- Stopped: terminated or withdrawn. Trials stop for many reasons, including slow enrolment and business decisions, not only safety or efficacy.
- Sponsors: the lead sponsor of each trial. Companies are the sponsors ClinicalTrials.gov classes as industry; the rest are universities, hospitals, cooperative groups, government bodies and individuals.