Repairing disease causing genes and converting risk genes into protective genes
At PeterBio we want to make nature’s solutions to genetic diseases available through our RITDM™ technology. In nature, specific genes can exist in multiple variants, some of which can cause diseases, but others can also be protective or leading to milder symptoms. At this moment we are focusing the first applications of our technology on severe monogenic diseases. It is our vision that the technology at a later stage can also be applied to chronic diseases that have a strong genetic component, such as Alzheimer’s Disease.
Various monogenic metabolic disorders involve mutations in genes that code for enzymes that are expressed at high levels in liver hepatocytes. Editing the genes involved can restore enzyme functionality in the liver, which can result in curing or alleviating the severity of the disease. We are currently working on a number of targets in this category.
We expect that the potential to perform repeat dosing will provide the opportunity to intervene at a young age, to limit as much as possible pathology and then to perform follow-on treatments as required.
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy can be caused by a wide variety of mutations in the Dystrophin or DMD gene. The DMD gene is one of the largest human genes. In addition to mutations that cause the most severe form of dystrophy, also mutations are known that cause a milder form of dystrophy. In most of the later cases exons (pieces of coding DNA) towards the end of the gene are missing and a shorter, but still functional form of dystrophin is formed. Our RITDM™ technology can be used to replicate this form of “exon skipping”, thereby reducing the disease severity.
We anticipate that repeat dosing could be beneficial, as muscle cells make up a very large portion of the human body. Repeat dosing can help to achieve a that more muscle cells are being edited, compared to single treatments.
Bone marrow stem cells treatments
Sickle Cell Disease is caused by a point mutation in the beta globin gene. Studies have shown that protective mutations in other genes exist that can help to alleviate symptoms, for example by switching on another form of hemoglobin that can take over key functions. One such gene is called Bcl11A. Our RITDM™ technology can be used to replicate this compensation mechanism, reducing the impact of sickle cell disease for patients.