According to a story from Angioedema News, the drug development company Regenxbio has announced that it is in the process of developing a new gene therapy treatment for hereditary angioedema, a rare condition that causes episodes of extreme swelling. Such a therapy would have the capability to revolutionize how this disease is treated. Unlike most gene therapies, which deliver a corrected copy of the mutated gene that causes a genetic disease, Regenxbio plans to create a version that delivers the genetic data for an antibody designed to target a protein called kallikrein.
About Hereditary Angioedema (HAE)
Hereditary angioedema is a genetic disorder which is characterized by chronic episodes of swelling that can affect multiple areas of the body. The condition is caused by mutations affecting the HAE gene. Swelling attacks generally occur every two weeks or so; they can usually last for several days. Swelling may affect the limbs, digestive tract, face, and airway, with blockage of the airway being the most dangerous complication. Vomiting and abdominal pain may accompany attack as well if the digestive tract is involved. Treatment involves reducing the likelihood for attacks to appear and preventing them from worsening when they do. Hereditary angioedema is typically only life-threatening if left untreated. Prevalence of the condition is estimated to be around one in 10,000 to one in 50,000, at least in the US and Canada. To learn more about hereditary angioedema, click here.
Designing a Gene Therapy
The reason for targeting the kallikrein protein is because excessive concentrations of it are closely associated with swelling attacks in hereditary angioedema. Like currently approved gene therapies, the new therapy will used adeno-associated viruses (AAV) as its method of delivery; however, Regenxbio plans to use an upgraded version of the virus that should be more effective at transporting the genetic material and allow for longer gene expression. With this in mind, the company hopes that the therapy will be a single-use treatment that can provide sustained benefit.
This company is busy attempting to develop gene therapies and antibodies that can be delivered in order to treat a variety of diseases. Regenxbio has goals to develop therapies for Alzheimer’s, homozygous familial hypercholesterolemia, wet age-related macular degeneration, Hunter syndrome, and Hurler syndrome.
