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The prevalence of heart disease has increased substantially over the last few decades, with approximately 32% of global fatalities presently attributed to it. In the United States, the National Library of Medicine predicts that the occurrence of people with cardiovascular risk factors and cardiovascular diseases will rise considerably between 2025 and 2060. The complexity of the heart and the myriad of underlying causes, like genetics, aging, and environmental factors, have made it difficult to develop novel therapies for different forms of heart disease, leaving it a significant unmet medical need.
Although development and FDA approvals for heart disease-related drugs have declined recently, increasing insights into the genetic causes of heart disease and a push for more genetic testing are potential catalysts for scientific breakthroughs. In fact, upwards of 50% of everyone’s likelihood of developing the most common form of heart disease, coronary heart disease, is linked to their genetics.
Tenaya’s therapeutic programs are heavily rooted in leveraging the growing understanding of the links between genetic factors and heart disease to focus on treating the underlying causes instead of just the symptoms. The company operates its own manufacturing facility to support these programs in advancing medicines for prevalent and rare forms of heart disease. Its Genetic Medicines Manufacturing Center (GMMC) is based in Union City, California.
Hypertrophic cardiomyopathy (HCM) is a disease that affects one in every 500 people in the United States. A mutation in the MYBPC3 gene is the most common cause of familial HCM. It causes the heart muscle to become thickened, leading to fibrosis, cardiac dysfunction, abnormal heart rhythms, heart failure, and increased risk of sudden cardiac death. The MYBPC3 gene is responsible for encoding the MyBP-C protein, which plays a central role in the contraction and relaxation of the cardiac muscle.
TN-201 aims to be the first treatment to address the underlying genetic cause of MYBPC3-associated HCM. TN-201 delivers a fully functional MYBPC3 gene to restore MyBP-C protein levels to normal, halt disease progression, and stimulate its reversal in a single treatment.
Heart failure with preserved ejection fraction (HFpEF) is a type of heart failure where the left ventricle stiffens and does not relax. This leads to the build-up of blood inside the left atrium, which eventually swells into the lungs, veins, and other body tissues. HFpEF represents approximately half of heart failure patients, including 3 million affected in the United States and 32 million worldwide. Currently, there are no approved disease-modifying therapies for HFpEF.
TN-301 is a highly specific small molecule inhibitor of HDAC6. HDAC6 interacts with multiple proteins in the cell cytoplasm to coordinate cellular processes. In pre-clinical animal modules, HDAC6 inhibitors were observed to reverse preexisting cardiac hypertrophy, improving lunge congestion and exercise capacity, all typical indications of HFpEF.
Arrhythmogenic right ventricular cardiomyopathy (ARVC) is primarily an inherited condition where muscle cells in the heart’s right ventricle are progressively lost and replaced by fatty deposits and a composite of fibrotic tissue. This structural change can lead to an abnormally high heart rate, palpitations, fainting, and potential cardiac arrest. Most patients with ARVC are diagnosed before the age of 40, and the median age of cardiac arrest for ARVC patients is just 25 years old.
Approximately 1 in 2,500 to 1 in 5,000 of the general population are affected by ARVC, with a mutation in the PKP2 gene being the most common genetic cause. The PKP2 protein plays an integral role in maintaining heart structure. Mutations of PKP2 are observed to cause cell death, which leads to inflammation, scar formation, and fat deposition in the heart tissue.
TN-401 is an AAV-based gene therapy that delivers a fully functional copy of the human PKP2 gene to the heart to replace the missing PKP2 protein and restore proper structure and function within the heart.
Tenaya Therapeutics has yet to complete any clinical trials and receive FDA approval for the commercial sale of its products. The company has not generated any revenue from products or other sources and has incurred losses and negative cash flows since commencing operations in 2016.
Tenaya has primarily funded its operations through the sale of equity securities. To meet its objectives of moving its lead product candidates through clinical trials while continuing to test and assess new preclinical programs, Tenaya will require substantial additional funding. The company may need to consider further equity offerings, use debt financing, or collaborate strategically with third parties to obtain this funding.
The biotechnology and pharmaceutical industry is characterized by rapid change and extreme competition. However, Tenaya Therapeutics bears some underlying variables that make it a compelling bet to help usher in the future of medicine. The company comprises a team of leading cardiovascular scientists whose sole focus is improving available heart disease solutions. Instead of spreading resources and expertise along a broader spectrum of medical need, the company directs its resources to a singular focus. Furthermore, the company’s three product platforms leverage the latest technologies available to the medical world, like Pluripotent Stem Cells, gene therapy, and machine learning, to develop innovative and comprehensive therapeutic solutions for heart disease.
The real test is only just beginning with the commencing of its first clinical trials, but it wouldn’t be surprising to see humanity finally tackle heart disease through Tanaya’s therapies that address the issue at the source.