Progress Towards Degrading Cholesterol by Repair Biotechnologies

Our pipeline

Our path to the clinic starts with the regression of atherosclerotic plaque, but the Cholesterol Degrading Platform offers the ability to treat a broad range of both rare and widespread medical conditions.

Description

Atherosclerosis is the buildup of cholesterol-based plaque inside arteries. It affects everyone, and is the world’s deadliest disease, being responsible for more than 5 million deaths per year. Current drugs generate about $22B in yearly revenues.

Shrinking of existing atherosclerotic plaque burden is strongly correlated with reduced incidence of cardiovascular events. And that is the goal that Repair Biotechnologies aims to achieve with our innovative CDP therapy.

In mice, CDP therapy rapidly, safely, and robustly reduces plaque burden. Read more about our science here.

Our initial pathway to the clinic will be through homozygous familial hypercholesterolemia (HoFH), an orphan indication.

HoFH is a genetic disorder characterized by two defective copies of the LDLR gene. These patients have an extremely accelerated form of atherosclerosis, which starts at childhood and where LDL-C levels can reach 1,000 mg/dL. Untreated, patients have a life expectancy of 33 years. HoFH is extremely rare, with a prevalence of about 1 in a million people.

Description

Metabolic-dysfunction associated steatohepatitis (MASH) is an epidemic of liver disease in the Western world, and is projected to be a $21B market by 2025.

Animal studies have produced a profound and rapid reversal of liver fibrosis following CDP treatment. Liver fibrosis is the feature of MASH that remains the most challenging to address, and presently approved therapies can achieve little on this front.

Description

Repair Biotechnologies’ CDP platform can be used to produce gene therapies targeting any indication in which a localized excess of cholesterol is a major driver of pathology. This includes many conditions associated with aging, obesity, and inherited mutations disruptive to cholesterol metabolism.

 

Description

Atherosclerosis is the buildup of cholesterol-based plaque inside arteries. It affects everyone, and is the world’s deadliest disease, being responsible for more than 5 million deaths per year. Current drugs generate about $22B in yearly revenues.

Shrinking of existing atherosclerotic plaque burden is strongly correlated with reduced incidence of cardiovascular events. And that is the goal that Repair Biotechnologies aims to achieve with our innovative CDP therapy.

In mice, a single treatment of CDP therapy reduced atherosclerotic plaque by 48% in mice. Read more about our science here.

Our initial pathway to the clinic will be through homozygous familial hypercholesterolemia (HoFH), an orphan indication.

HoFH is a genetic disorder characterized by two defective copies of the LDLR gene. These patients have an extremely accelerated form of atherosclerosis, which starts at childhood and where LDL-C levels can reach 1,000 mg/dL. Untreated, patients have a life expectancy of 33 years. HoFH is extremely rare, with a prevalence of about 1 in a million people.

Description

Repair Biotechnologies’ CDP therapy is modular, and can be used for several indications where cholesterol is a major driver of pathology.

This undisclosed liver indication is an epidemic in the Western world, and is projected to be a $21B market by 2025.

Cell efficacy studies for this indication were successful, and animal studies are ongoing.

Description

Repair Biotechnologies’ second liver indication is an orphan disease which kills hundreds of thousands of people every year.

It is an extremely deadly disease, with a majority of patients dying over the course of a few years.

Cell efficacy studies for this indication were successful, and animal studies are ongoing.

Atherosclerosis/HoFH (orphan)
Phase
Animal Efficacy
Description

Atherosclerosis is the buildup of cholesterol-based plaque inside arteries. It affects everyone, and is the world’s deadliest disease, being responsible for more than 5 million deaths per year. Current drugs generate about $22B in yearly revenues.

Shrinking of existing atherosclerotic plaque burden is strongly correlated with reduced incidence of cardiovascular events. And that is the goal that Repair Biotechnologies aims to achieve with our innovative CDP therapy.

In mice, a single treatment of CDP therapy reduced atherosclerotic plaque by 48% in mice. Read more about our science here.

Our initial pathway to the clinic will be through homozygous familial hypercholesterolemia (HoFH), an orphan indication.

HoFH is a genetic disorder characterized by two defective copies of the LDLR gene. These patients have an extremely accelerated form of atherosclerosis, which starts at childhood and where LDL-C levels can reach 1,000 mg/dL. Untreated, patients have a life expectancy of 33 years. HoFH is extremely rare, with a prevalence of about 1 in a million people.

Undisclosed Liver Indication
Phase
Cell Efficacy
Description

Repair Biotechnologies’ CDP therapy is modular, and can be used for several indications where cholesterol is a major driver of pathology.

This undisclosed liver indication is an epidemic in the Western world, and is projected to be a $21B market by 2025.

Cell efficacy studies for this indication were successful, and animal studies are ongoing.

Undisclosed Liver Indication (orphan)
Phase
Cell Efficacy
Description

Repair Biotechnologies’ second liver indication is an orphan disease which kills hundreds of thousands of people every year.

It is an extremely deadly disease, with a majority of patients dying over the course of a few years.

Cell efficacy studies for this indication were successful, and animal studies are ongoing.