Tenaya CEO Faraz Ali(Tenaya Therapeutics)

Although heart disease remains the leading cause of death worldwide, its an area that hasnt seen as much interest or investment as other areas, and its treatments still focus on dealing with symptoms. With a multipronged approach and a fresh infusion of $92 million, Tenaya Therapeutics is trying to change that.

The heart is a complicated organ, and it can go wrong in different ways. Part of what weve learned the hard way is that prior approaches are not working, Tenaya CEO Faraz Ali told FierceBiotech.

Founded in 2016 by scientists from the Gladstone Institute in California and the University of Texas Southwestern Medical Center, Tenaya is going after the underlying causes of heart disease to head off heart failure. It raised $50 million in its series A round from The Column Group, which also pitched into its $92 million B round alongside the likes of Casdin Capital and GV.

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In an age where companies are forming themselves around one platform thats disease-agnostic and can be applied to many different areas, South San Francisco-based Tenaya is doing the opposite. It's working on three different platforms for heart disease:regenerative treatments, gene therapies and precision medicines.

The company was founded with a big, bold mission to follow the science and use the right tool for the job, Ali said. If the problem is loss of cardiomyocytes (the muscle cells that make the heart beat), such as after a heart attack, we can look for a way to generate new myocytes, create new tissue and improve the ability of the heart to contract that way.

Thats where regenerative treatments come in. Tenaya's approach delivers transcription factors that can nudge heart fibroblasts, cells that play a role in scar formation after a heart attack, to become heart muscle.

If the problem stems from geneticsif a faulty gene doesnt cause heart muscle to die, but leads to an arrhythmia or scarring, stopping it from working properlythe solution is not to create new muscle, but to get the muscle thats there to work, Ali said. And thats where gene therapy, adding a healthy copy of a defective gene, comes in.

Finally, Tenayas taking a leaf out of the book of cancer drug developers: Its become quite the norm to look for therapies that work in a particular genetic background, he said. The companys precision medicine platform uses stem cell-derived heart muscle cells as disease models to identify new targets for heart failure and screen new drugs. Its first focus is on small molecules for the treatment of dilated cardiomyopathiesa group of conditions in which an enlarged heart chamber makes it less efficient at pumpingin genetically defined patient groups.

What the funding allows us to do is advance multiple promising projects from each of the platforms out of the pure research stage and into the clinic, Ali said. Tenaya hopes to move to human studies over the next few years.

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Though the company isnt divulging just yet which targets its going after, Ali did say some of its programs are chasing more prevalent conditions such as heart attacks while others are looking at smaller, more defined populations.

Once we get a signal of efficacy and safety and advance into later-stage clinical development, we could potentially expand into larger, more prevalent indications, he said.

Tenaya has about 45 staffers who are mostly focused on research, early development and manufacturing. If all goes to plan, the company plans to double its size by the end of 2021, adding more employees in development and manufacturing.

Two-thirds of our platforms that were working on [gene therapy and regenerative treatments] are heavily dependent on viral vectors, adeno-associated viruses Everyone learned in the last decade or so that manufacturing is the Achilles heel of the gene therapy spaceit's difficult to do and highly technical, its nothing like small molecule manufacturing, Ali said. We made the decision to invest early, and well ahead of being in the clinic, to invest in manufacturing.

Tenaya isnt just doing it because other gene therapy players have proved it necessary. If it wants to go after larger heart disease populations rather than the smaller groups affected by rare disease, its going to need a lot of viral vector to deliver its treatments.

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Tenaya Therapeutics bags $92M to develop triple threat for heart disease - FierceBiotech