The lipodystrophy project
With the lipodystrophy project, which was inlicensed from Lipigon Pharmaceuticals AB in autumn 2019, CombiGene has expanded its operations to include metabolic disorders. The initial aim of the project is to develop a gene-therapeutic treatment for partial lipodystrophy, a very rare condition for which there is currently no adequate treatment. The project is in an early stage of development.
Lipodystrophy is a rare disorder that is characterized by abnormal distribution of fat in the body. Patients suffer from lipoatrophy, which means that body fat is lost. In the absence of normal body fat different organs begin to accumulate fat, which subsequently leads to serious metabolic complications, among them, extreme insulin resistance, hypertriglyceridemia (elevated levels of the blood fat triglyceride) and hepatic steatosis (fatty liver disease).
There are currently a few treatments that can alleviate the symptoms of lipodystrophy, but no form of therapy that is targeted directly at the fundamental cause of the disorder. For patients suffering from partial lipodystrophy there are currently no treatments whatsoever.
Lipigon Pharmaceuticals is a spin-off company from Umeå University, Sweden. Based on five decades of research, the company develops drugs targeting lipid related diseases. Lipigon aims at building a sustainable research-based company by bringing their programs to major inflection points. In addition to the agreement with CombiGene, Lipigon’s pipeline comprises one project for the treatment of high blood lipids through the use of antisense technology and a project that aims to activate an enzyme that breaks down fat. The latter project is based on a small molecule.
CombiGene’s vision is to provide patients affected by severe life-altering diseases with the prospect of a better life through novel gene therapies. CombiGene’s business concept is to develop effective gene therapies for severe life-altering diseases where adequate treatment is currently lacking. Development assets are sourced from an external research network and developed to achieve clinical proof of concept. Drug candidates for common diseases will be co-developed and commercialized through strategic partnerships, while the company may manage this process on its own for drugs targeting niched patient populations. The company is public and listed on the Swedish marketplace Nasdaq First North Growth Market and the company’s Certified Advisor is FNCA Sweden AB, +46 (0)852 80 03 99 email@example.com.