Approval Probability
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TN-401 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | Dose for Cohort 1 will be 3E13 vg/kg |
| Cohort 2 | EXPERIMENTAL | Dose for Cohort 2 will be 6E13 vg/kg |
| Name | Type | Description |
|---|---|---|
| TN-401 | GENETIC | TN-401 is a recombinant adeno-associated virus serotype 9 (AAV9) gene therapy containing Plakophilin-2 (PKP2) transgene. It is a single (one-time) intravenous dose. |
Inclusion Criteria: * PKP2 mutation (pathogenic or likely pathogenic) * Arrhythmogenic Right Ventricular Cardiomyopathy as defined by the 2010 revised Task Force Criteria * Left Ventricular Ejection Fraction ≥50% * Functioning Implantable Cardiac Defibrillator with remote integration capabilities a...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Lexeo Therapeutics, Inc. | LXEO | 3 | PHASE1 | LX2020 |
TN-401 is an investigational gene therapy being developed for arrhythmogenic right ventricular cardiomyopathy, or ARVC, in adults who carry mutations in the PKP2 gene. It is not approved for any use and remains in clinical development.
TN-401 targets PKP2, the gene that encodes plakophilin-2. In patients with PKP2 mutation-associated ARVC, loss of functional plakophilin-2 disrupts the proteins that hold heart muscle cells together. TN-401 is designed as a gene therapy to deliver a working copy of PKP2 to heart cells.
TN-401 is being developed by Tenaya Therapeutics, Inc., which trades on the Nasdaq under the ticker TNYA. Tenaya is a cardiovascular-focused biotechnology company advancing TN-401 as its gene therapy candidate for PKP2 mutation-associated ARVC.
TN-401 is in Phase 1 clinical development. It is an investigational therapy and has not been approved by the FDA. The Phase 1 study is an open-label, dose escalation trial evaluating safety and preliminary efficacy in adults with PKP2 mutation-associated ARVC.
TN-401 is being studied in one active clinical trial, NCT06228924, titled an open-label, dose escalation study of safety and preliminary efficacy of TN-401 in adults with PKP2 mutation-associated ARVC. The trial is recruiting in the United States and plans to enroll 15 participants aged 18 years and older.
TN-401 has received three FDA designations: Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designation. These designations relate to its development for PKP2 mutation-associated arrhythmogenic right ventricular cardiomyopathy, a serious cardiovascular condition with limited treatment options.