Telocyte’s gene therapy funded through to clinical trials

Michael Fossel on securing new investment, taking telomerase gene therapy to the clinic, and its potential in cardiovascular disease.

Longevity.Technology has learned that telomerase gene therapy developer Telocyte has secured new investment, ensuring that the company’s work is now funded through to clinical trials and potentially beyond. Telocyte, which recently published a new paper describing a unified model of age-related cardiovascular disease, is primarily focused on developing a gene therapy for Alzheimer’s.

Longevity.Technology: Despite Telocyte’s current focus on Alzheimer’s, it’s interesting to see that the company clearly has ambitions beyond neurodegenerative disease. This new paper is confirmation that a gene therapy for cardiovascular disease is also in its future roadmap. Telocyte has long been seeking fresh investment to move its Alzheimer’s therapy towards the clinic, so we caught up with founder and president Dr Michael Fossel to find out more.

Fossel gladly shares the news that Telocyte has been successful in finding an investor to back the company’s operations for the foreseeable future, and not just for its work in Alzheimer’s disease.

“Ultimately, all age-related diseases are our targets, and luckily, we now have an investor who sees exactly that sort of potential,” he says. “They are basically willing to underwrite us to an unlimited degree to see if we can take this all the way to the clinic – not only for age-related neurodegenerative diseases and cardiovascular disease, but beyond that as well.”

Clinical trials by 2025

While Fossel says that the new investor prefers to remain behind the scenes, he does share some information about their approach to investing in longevity.

Telocyte's gene therapy funded through to clinical trials
Credit: Dr Michael Fossel

“They’ve stressed that, from their perspective, most companies like ours fail because of lack of funds, and they do not want to let that happen,” he says. “They have stressed that they’re not in this just for the money – it’s not just a matter of making a profit, it’s a matter of making it work.”

“Our chief investor feels the same way we do, which is we’d like this to be financially available for everyone. He says he’d like to make it as cheap as Tylenol. Now that probably won’t happen, but I’d rather treat a billion people for one dollar than one person for a billion dollars.”

Regarding the company’s roadmap, Fossel says that the next steps will be large animal studies, looking at toxicity, dosing and delivery systems – ideally later this year.

“Of course, that depends on a lot of things that are outside our control, like the supply of dogs and the production of what we need. If all goes well, and it will likely not go this well, we would start the human trials late the following year. But more likely we’re looking at 2025.”

Gene therapy and CVD

Telocyte’s new cardiovascular disease paper explains why interventions aimed at upstream risk factors or downstream clinical outcomes have limited success, and why telomerase gene therapy is a promising intervention due to its potential to prevent and reverse cell senescence. In many ways it parallels Fossel’s 2020 paper on neurodegeneration.

“When we look at neurodegenerative diseases, we tend to separate them but, the truth is, clinically, that they tend to overlap,” he says. “And the same is true regarding age-related cardiovascular disease. The reason is we end up with one specific disease or another is because we start in a different place, genetically and epigenetically.”

Rather than looking at cardiovascular disease outcomes or how they start, Fossel believes the key lies in the central process – cellular aging.

“Cell aging essentially takes all of the inputs, the upstream genetic induction factors, and explains why it is that they result in the downstream outcomes,” he says. “We should be addressing the central issue, which is cell aging, because it’s more likely to be able to give us an optimal point of intervention clinically, one that can not only slow the outcome, but stop it and reverse it.”

The key question, says Fossel, is not what causes cardiovascular disease, or even what causes aging, but what is the optimal point of intervention?

“You can do a heart transplant, but that’s expensive, not only financially, but in terms of risk and other problems, so it’s not an optimal point of intervention,” he says. “And in many ways, the same is true of all the other interventions. Stents, statins and so on – they are useful, but are they optimal? And I think we can do a lot better if we can deal with some of the intervening issues regarding cell aging.”

Why telomerase therapy?

There are two key reasons why Fossel believes a telomerase gene therapy holds potential in age-related disease – one philosophical, the other practical.

“The philosophical issue is, can you construct a model that makes sense and explains all the current data?” he says. “For example, we know that there’s mitochondrial dysfunction in aged cells. Is that a primary cause of telomere shortening or is it secondary to the telomere shortening. We have a model that suggests it’s secondary. And our model is consistent – it explains all the known data, and has been predictably valid as well, which is nice. For example, it explained ahead of time, what the outcome would be in the lecanemab trial, and why it should happen that way.”

But the main practical reason he believes in the potential of Telocyte’s gene therapy, says Fossel, is that it actually works. So far, at least.

“We showed some 25 years ago that we could reverse aging in human cells in the laboratory, which increased mitochondrial function, improved DNA repair and so on,” he says. “This has been repeated in the last year or two, so we know we can do this in tissues and organisms too.”

“But can we do it in human patients? That’s really where we are today. A good, logical, consistent theory is excellent – but until you take it human trials, that’s all it is, because human data trumps theory every time. So, we’ll see.”

Image by kjpargeter on Freepik