‘We’re seeing profound reversal of a number of conditions’

Repair Bio is harnessing gene therapy’s potential to clear excess cholesterol with a platform that could reshape cardiovascular treatment.

Repair Biotechnologies is developing a novel approach to cholesterol management with its Cholesterol Degrading Platform (CDP); this innovative approach moves beyond conventional cholesterol-lowering strategies, aiming instead to degrade cholesterol at the cellular level.

The Syracuse, New York-based biotech is using unique lipid nanoparticle (LNP)-messenger RNA (mRNA) therapies designed to address the root causes of conditions stemming from localized cholesterol accumulation, setting a new standard in therapeutic interventions.

Unlike traditional methods that merely reduce LDL-cholesterol levels in the bloodstream, Repair Bio’s strategy targets the intracellular free cholesterol that is toxic to cells and a contributing factor in a variety of diseases. This approach not only promises to mitigate atherosclerosis but also shows potential in treating metabolic dysfunction-associated steatohepatitis (MASH), as evidenced by promising results in preclinical mouse models.

My take on this: Atherosclerosis, marked by the buildup of arterial plaque, significantly impedes blood flow and is a major contributor to cardiovascular disease (CVD) – the leading cause of death worldwide. To address this pressing health burden, scientists at Repair Bio have developed a novel LNP-mRNA therapy, and in recent experiments, atherosclerotic mouse models treated with this therapy over a six-week period demonstrated promising results. It is an innovative approach that not only holds potential for mitigating atherosclerosis but also offers a pathway to reducing the global burden of CVD, ultimately saving lives and improving health outcomes.

The viability of this approach was further underscored when Repair Bio recently received favorable pre-IND feedback from the FDA, marking a significant milestone as the company prepares for clinical trials. We sat down with Reason, the Founder and CEO of Repair Bio, to find out more about the platform, upcoming clinical trials and tackling the Goldilocks problem in delivery systems.

Reason on the nature of cholesterol

Most people understand that as you go through life, you are supposed to have low blood cholesterol. Cholesterol is bad in the sense that if you exhibit raised LDL cholesterol in your blood, then you are not in a good position. But the cholesterol we’re interested in occurs at the point at which the transport of cholesterol in your body becomes disrupted either by aging or obesity, producing our target of localized excesses of cholesterol. The important thing to remember here is that even though cholesterol is vital, unmodified free cholesterol is toxic. Cholesterol is made safe by cells in a variety of ways, but cells have only a limited capacity to deal with any excess of it. If you have too much cholesterol in one location, that excess will generate free cholesterol, which becomes toxic when inside cells. Those cells begin to die if there’s enough of this free cholesterol, and well before that point, they will begin to become dysfunctional in many ways. An example of this is atherosclerosis, wherein the localized excess exists in a small region of your blood vessels, and everything nearby starts falling apart. Eventually, you suffer a stroke or a heart attack, and you die because the atherosclerotic plaque ruptured. The other important example is the liver, the center of your cholesterol metabolism. It’s where most cholesterol is created and then sent out into circulation – but in the case of fatty liver, in obesity and in aging, you simply have too much cholesterol.

The liver is influential on the body in many ways, and if excessive cholesterol is stuck in the liver, becoming free cholesterol to some degree, then your liver suffers pathology. This appears to drive a number of conditions, but that was hard to prove until we introduced our approach to specifically and deliberately break down and remove only the excess free cholesterol. Given that capability, suddenly we can demonstrate profound reversal of a number of conditions, age-related and otherwise – and that is the fundamental basis for our technology.

Find out more about the necessity of gene therapy, Repair Bio's future steps and more, straight from Reason right HERE.

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