TNYA — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-05-06

• Early on in our company history, we invested in differentiated capabilities to enable modality-agnostic target identification and validation, anchored in human genetics and the use of human disease models. To support our initial focus on gene therapy candidates, we also internalized expertise in cap…

• We are primarily focused on advancing our clinical-stage gene therapy candidates, TN-201 for MYBPC3-associated hypertrophic cardiomyopathy (HCM), and TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC). Each candidate is currently in Phase 1b/2 trials to establish the s…

• For programs arising out of our modality agnostic drug discovery platform that address relatively rare conditions our strategy is to develop, manufacture and commercialize at least some of these programs on our own, although we may selectively consider partnerships to access technology, accelerate o…

• TN-201 is our investigational gene therapy for individuals with HCM due to MYBPC3 gene mutations. These mutations result in a deficiency of myosin-binding protein C (MyBP-C), which in turn can cause the heart walls of affected individuals to become significantly thickened, leading to fibrosis, abnor…

• working MYBPC3 gene to specific cells of the heart in order to produce MyBP-C and thereby potentially slow or even reverse the course of MYBPC3-associated HCM following a single infusion.

Text removed vs the prior filing · source: 10-Q · 2025-11-10

• We are advancing a deep and diverse modality agnostic pipeline of targeted therapies for heart conditions discovered internally using our extensive target identification and validation and genetic medicine design and manufacturing capabilities. All of our programs are currently in the clinical or pr…

• We are primarily focused on development of our lead investigational product candidates, TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), and TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC). Each candidate is currently i…

• TN-201 is our investigational gene therapy for individuals with HCM due to MYBPC3gene mutations. These mutations result in a deficiency of myosin binding protein (MyBP-C), which in turn can cause the heart walls of affected individuals to become significantly thickened, leading to fibrosis, abnormal…

• MyPEAKTM-1 is our Phase 1b/2a multi-center, open-label clinical trial, designed to assess the safety, tolerability and efficacy of a one-time intravenous infusion of TN-201. Enrollment and dosing in both the 3E13 vg/kg dose (Cohort 1) and 6E13 vg/kg dose (Cohort 2) cohorts are complete. In summer 20…

• In November 2025, we presented interim data from MyPEAK-1 during the Late-Breaking Science: Main Event session at the American Heart Association’s (AHA) Scientific Sessions 2025. Interim data presented at AHA included safety, biopsy and efficacy results for the three patients enrolled in Cohort 1 wi…

Text added vs the prior filing · source: 10-Q · 2026-05-06

• There were no changes in our internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) during the quarter ended March 31, 2026 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

Text removed vs the prior filing · source: 10-Q · 2025-11-10

• interim Principal Financial Officer, to allow timely decisions regarding required disclosure. We believe that a control system, no matter how well designed and operated, cannot provide absolute assurance that the objectives of the control system are met, and no evaluation of controls can provide abs…

• There were no changes in our internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) during the quarter ended September 30, 2025 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

Text added vs the prior filing · source: 10-Q · 2026-05-06

• Drug development involves a lengthy and expensive process with an uncertain outcome. The preclinical studies, clinical trials and post-marketing studies of our product candidates may not demonstrate safety and efficacy to the satisfaction of the FDA, European Medicines Agency (EMA) or other comparab…

• experience delays in completing, or ultimately be unable to complete, the development and commercialization of our product candidates.

• Gene therapies are novel, complex and difficult to manufacture. We could experience production problems that result in delays in development or commercialization of our product candidates, limit the supply of our products, if approved, or otherwise seriously harm our business.

• may be more difficult for investors to accurately predict our likelihood of success and viability than it would be if we had a longer operating history.

• the achievement of development, regulatory and sales-based milestones under our collaboration agreement with Alnylam;

Text removed vs the prior filing · source: 10-Q · 2025-11-10

• Our loan agreement with Silicon Valley Bank (SVB), a division of First-Citizens Bank & Trust Company (the Loan Agreement), requires us to comply with specified operating covenants and places restrictions on our operating and financial flexibility.

• Drug development involves a lengthy and expensive process with an uncertain outcome. The preclinical studies, clinical trials and post-marketing studies of our product candidates may not demonstrate safety

• and efficacy to the satisfaction of the FDA, European Medicines Agency (EMA) or other comparable foreign regulatory authorities or otherwise produce positive results and the results of preclinical studies and early clinical trials may not be predictive of future results. We may incur additional cost…

• Due to our limited manufacturing experience, there can be no assurance that we will be able to successfully manufacture product candidates to support our clinical development and commercialization plans.

• We have not yet demonstrated our ability to successfully complete any late-stage clinical trials, obtain marketing approvals, manufacture a late stage clinical- or commercial-scale product or arrange for a third party to do so on our behalf, or conduct sales and marketing activities necessary for su…