We are Remute Bio

We develop a proprietary and highly innovative, broadly applicable platform-technology, to cure genetic diseases, with highest medical need. Our drugs silence mutated genes, a strategy that we call “remuting”, thereby rescuing health by restoring biological function at cellular level.

The Challenge:

Genetic diseases without cures

Inheriting a disease, condition, or trait depends on the type of chromosome affected and also on whether the trait is dominant or recessive. A single abnormal gene on one of the 22 autosomal chromosomes from either parent can cause an autosomal disorder. Dominant inheritance means an abnormal gene from one parent is already sufficient to cause disease. This happens even when the matching gene from the other parent is normal – the abnormal gene dominates. A parent with an autosomal dominant condition has a 50 % chance of having a child with the condition.

Challenges of siRNA-based drugs

But there are barriers a successful siRNA therapeutic must over-come:

  1. Drug targeting to peripheral tissues except liver and kidney
  2. Degradation and elimination of drug substance in the cytoplasm
  3. Toxicity owing to non-redundant functions in the normal physiology of the target gene
  4. Unwanted side effects, e.g. immunogenicity

The Solution:

Targeted expression and delivery (TED)

To overcome these barriers, siRNAs can be combined with targeted delivery moieties or modified to enhance their stability without affecting gene silencing activity. Remute Bio will revolutionize the targeted application of gene silencing and replacement using a Targeted Expression and Delivery (TED)-platform-technology. This technology will overcome the present barriers using two different technologies:

  1. Production of shRNA/siRNA in high numbers directly in the targeted cells to    reduce side effects, dosis and costs compared to systemic siRNA delivery
  2. Targeted delivery tools to protect the DNA/RNA on the way to the target cell, where it will be released and active.

Small interfering RNAs (siRNAs) to treat genetic diseases

To date, there are no generalizable cures available for autosomal dominant diseases. These genetic conditions can be treated using targeted gene silencing of defective allels by RNA interference (“remuting”).  siRNA are 15 to 25 nucleotide sequences, highly specific and efficient in silencing gene expression.

The Challenge:

Genetic diseases without cures

Inheriting a disease, condition, or trait depends on the type of chromosome affected and also on whether the trait is dominant or recessive. A single abnormal gene on one of the 22 autosomal chromosomes from either parent can cause an autosomal disorder. Dominant inheritance means an abnormal gene from one parent is already sufficient to cause disease. This happens even when the matching gene from the other parent is normal – the abnormal gene dominates. A parent with an autosomal dominant condition has a 50 % chance of having a child with the condition.

Challenges of siRNA-based drugs

But there are barriers a successful siRNA therapeutic must over-come:

  1. Drug targeting to peripheral tissues except liver and kidney
  2. Degradation and elimination of drug substance in the cytoplasm
  3. Toxicity owing to non-redundant functions in the normal physiology of the target gene
  4. Unwanted side effects, e.g. immunogenicity

The Solution:

Targeted expression and delivery (TED)

Inheriting a disease, condition, or trait depends on the type of chromosome affected and also on whether the trait is dominant or recessive. A single abnormal gene on one of the 22 autosomal chromosomes from either parent can cause an autosomal disorder. Dominant inheritance means an abnormal gene from one parent is already sufficient to cause disease. This happens even when the matching gene from the other parent is normal – the abnormal gene dominates. A parent with an autosomal dominant condition has a 50% chance of having a child with the condition.

Challenges of siRNA-based drugs

But there are barriers a successful siRNA therapeutic must over-come:

  1. Drug targeting to peripheral tissues except liver and kidney
  2. Degradation and elimination of drug substance in the cytoplasm
  3. Toxicity owing to non-redundant functions in the normal physiology of the target gene
  4. Unwanted side effects, e.g. immunogenicity

Disease Areas

Familial hypercholesterolemia (FH)

inherited in an autosomal dominant manner, causes high levels of LDL (low density lipoprotein) cholesterol levels beginning at birth, and heart attacks at an early age. This condition affects about 1 in 500 people in most countries. FH is commonly caused by a loss-of-function mutation in the LDL-receptor gene (LDLR) or by a mutation in the gene encoding apolipoprotein B (APOB). The overall goal of treatment at the moment is to lower the risk for atherosclerotic heart disease by lowering the LDL cholesterol levels in the blood stream.

Huntington’s disease (HD)

is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. It deteriorates a person’s physical and mental abilities. Symptoms usually appear between the ages of 30 to 50, and worsen over a 10 to 25 year period and has no cure. Experts estimate that one in every 10,000 persons. Mutations in the HTT gene cause Huntington disease. The HTT gene provides instructions for making a protein called huntingtin. Although the function of this protein is unknown, it appears to play an important role in nerve cells (neurons) in the brain. At the moment no treatments can alter the course of Huntington’s disease. But medications can lessen some symptoms of movement and psychiatric disorders.

 

Disease Areas

Familial hypercholesterolemia (FH)

inherited in an autosomal dominant manner, causes high levels of LDL (low density lipoprotein) cholesterol levels beginning at birth, and heart attacks at an early age. This condition affects about 1 in 500 people in most countries. FH is commonly caused by a loss-of-function mutation in the LDL-receptor gene (LDLR) or by a mutation in the gene encoding apolipoprotein B (APOB). The overall goal of treatment at the moment is to lower the risk for atherosclerotic heart disease by lowering the LDL cholesterol levels in the blood stream.

Huntington’s disease (HD)

is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. It deteriorates a person’s physical and mental abilities. Symptoms usually appear between the ages of 30 to 50, and worsen over a 10 to 25 year period and has no cure. Experts estimate that one in every 10,000 persons. Mutations in the HTT gene cause Huntington disease. The HTT gene provides instructions for making a protein called huntingtin. Although the function of this protein is unknown, it appears to play an important role in nerve cells (neurons) in the brain. At the moment no treatments can alter the course of Huntington’s disease. But medications can lessen some symptoms of movement and psychiatric disorders.

Get in touch: get_in_touch_icon Do you want to learn more about Remute Bio´s Targeted Expression Platform and when it is ready? Get in touch to receive more information about our technology and milestones.
 
Get in touch: get_in_touch_icon
Do you want to learn more about Remute Bio´s Targeted Expression Platform and when it is ready? Get in touch to receive more information about our technology and milestones.