Tenaya Therapeutics

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Heart disease has been woefully neglected by biopharma: Tenaya wants to change that. (Tenaya Therapeutics)

Tenaya Therapeutics

Tenaya is going after the world’s biggest killer

CEO: Faraz Ali, MBA

Founded: 2016

Based: South San Francisco, California

Clinical focus: The Californian biotech is attacking heart disease from three different angles: gene therapy, cellular regeneration and precision medicine. It’s taking this multi-pronged approach to solve various problems that lead to heart disease and heart failure.

The scoop: Cardiovascular diseases (CVDs) are the leading cause of death globally, taking an estimated 17.9 million lives each year, according to the World Health Organization. While there are drugs that combat underlying conditions such as high cholesterol, there has been a surprisingly small amount of money invested in actually treating the disease itself. Tenaya aims to change that.

What makes Tenaya fierce: Cancer and rare diseases dominate the Fierce 15 as research targets not because we are actively seeking them out but because they typically offer better returns on investment. Cancer, in particular, also taps into a deep sense of dread, so much so that it is often shorthanded as "the big C."

What we tend to forget is that heart disease and its many forms are the biggest killers in the world. And the risk isn’t just linked to diet, smoking, the environment or age. It also has a genetic aspect with congenital heart defects (CHDs) as the most common types of birth defects. They can cause lifelong disability and even death.

But treatments for this prolific killer are few and far between. Tenaya Therapeutics, helmed by biotech veteran Faraz Ali of Regenxbio, Genzyme, and GE Healthcare, is hoping to turn that around.

It doesn’t have billions, but it’s not looking for money down the back of the couch either. It raised $106 million earlier this year in a Series C round and swiftly followed that with an upsized $180 million IPO. The capital will help fund its work on a gene therapy for hypertrophic cardiomyopathy (HCM), and pay for a site in the Bay Area to manufacture vectors to deliver its treatments.

HCM, the company’s lead indication, has multiple causes, both genetic and non-genetic. But Tenaya is starting with an AAV-based gene therapy targeting mutations in the MYBPC3 gene, the leading genetic cause of HCM.

“What we’re doing is using a different approach from what’s gone before,” Ali says. “Regenerative platforms have been used in the past, but we’re doing things from a new perspective. At the time when Tenaya was founded [in 2016], there was a novel scientific approach centered on in vivo regeneration. What we do when it comes to cardiac regeneration is not trying to push cells from the outside but stimulate the resident cells in the patient’s own heart to create new heart muscle.”

That's just one approach, Ali says. The company also is looking at a precision medicine and gene therapy approaches to tackle the problem. What Tenaya hopes to do is tackle genetic forms of heart disease, including the congenital forms that can affect newborns and young children.

“This is too often forgot about,” Ali says. “And by our estimates, there are more than 250 disorders where you can identify genetic conditions associated with the heart. We believe there are around 35,000 children born every year with congenital heart disease, which massively increases their risks of disability and mortality.”

Whilst vaccines have helped massively reduce child mortality, heart conditions can and do kill many under the age of five in the U.S. If a gene therapy approach can safely help these children, it would dramatically lower child mortality rates.

“So what makes us fierce? We’re tackling the leading cause of death in the world. We’re attacking the genetic and non-genetic forms of the condition. We’re tackling some of the most rare and also the prevalent forms of heart disease and using cellular regeneration, gene therapy and precision medicine," Ali said. "This really gives us multiple shots on goal.”

He adds that with COVID-19, we may begin to see a whole new subset of CVDs in the future caused by complications from the virus, even in the young. “We might likely find ourselves developing treatments for COVID heart, too,” he said.

Tenaya is named after a peak in Yosemite National Park. The biotech’s founder is a serious mountain climber and spends a lot of time in the park. “He wanted to name the company for something that was not your normal Xgen type thing,” Ali explained.

Investors: RTW Investments, LP; RA Capital Management; Fidelity Management & Research Company, The Column Group, Casdin Capital and GV.

Tenaya Therapeutics