- **Tenaya Therapeutics** plans to file an IND for its lead BAG3 dilated cardiomyopathy program later this quarter (Q4 2025) and expects clinical results in early next year (2026), with a potential BLA submission as soon as **2027**.  **[0001858848_2194341_0]** **[0001858848_2194341_3]** 
- **Lexeo Therapeutics** is completing a Phase 1 study for arrhythmogenic cardiomyopathy (PKP2 mutation) this year (2025), with data readout early next year (2026), and is moving its Friedreich’s ataxia program into a pivotal study next year (2026).  **[0001858848_2194341_1]** 
- **AskBio** has two lead programs in Phase 2 for Parkinson's and heart failure, emphasizing **local delivery** to maintain low doses and avoid immunosuppression.  **[0001858848_2194341_0]** **[0001858848_2194341_6]** **[0001858848_2194341_8]** 
- Panelists highlighted significant advancements in manufacturing, such as AskBio's new process with **threefold better yield** and Tenaya's proprietary plasmid system yielding **10 times more product**, which is substantially reducing the cost of goods sold (COGS) for gene therapies.  **[0001858848_2194341_9]** 
- The regulatory environment is viewed as flexible, particularly for rare diseases, with optimism for successful commercial launches in the cardiac gene therapy field.  **[0001858848_2194341_1]** **[0001858848_2194341_8]** **[0001858848_2194341_12]**

Chardan Conference Panel Discusses Cardiac Gene Therapy Outlook

- **Tenaya Therapeutics** is advancing two AAV9-based gene therapies, **TN-201** for MYBPC3-positive hypertrophic cardiomyopathy (HCM) and **TN-401** for PKP2-positive arrhythmogenic cardiomyopathy (ARVC), both currently in clinical trials.  **[0001858848_2181083_0]** 
- **Interim data from the MyPeak-1 study for TN-201** demonstrated safety and tolerability in the first dose cohort, with evidence of high transduction, robust RNA and protein expression, and clinical improvements including significant reductions in cardiac troponin I and hypertrophy, and all three patients achieving **New York Heart Class I**.  **[0001858848_2181083_4]** 
- **Further data for TN-201** will be presented as a late breaker at the American Heart Association (AHA) on **November 8th**, providing additional safety and tolerability from both dose cohorts, biopsy data, and one-year durability results for the first cohort.  **[0001858848_2181083_5]** **[0001858848_2181083_6]** 
- For **TN-401**, the **first data release from the Phase 1b RIDGE-1 study is anticipated in Q4**, focusing on safety and early biopsy data (transduction, RNA, and protein expression).  **[0001858848_2181083_8]**

Tenaya Therapeutics Provides Clinical Program Updates for TN-201 and TN-401

- Tenaya Therapeutics is advancing two gene therapy programs, **TN-two zero one** for MyBPC3 associated hypertrophic cardiomyopathy (HCM) and **TN-four zero one** for PKP2 associated arrhythmogenic right ventricular cardiomyopathy (ARVC), with initial data readouts for both planned for **Q4 2025**.  **[1858848_tx_377649_1]** 
- Early results for TN-two zero one's Cohort one demonstrated the therapy was **well tolerated** and showed robust evidence of DNA transduction, increasing RNA expression, and improvements in protein levels, alongside clinical improvements in cardiac hypertrophy and heart failure symptoms for some patients.  **[1858848_tx_377649_4]** 
- The company employs **liquid chromatography mass spectrometry (LCMS)** for precise protein measurement in cardiac biopsies, a method considered vital for demonstrating gene therapy efficacy and supporting potential **accelerated regulatory approvals** based on protein as a surrogate marker.  **[1858848_tx_377649_6]** **[1858848_tx_377649_27]**

Tenaya Therapeutics (TNYA)