The lipodystrophy project

With the lipodystrophy project, which was inlicensed from Lipigon Pharmaceuticals AB in autumn 2019, CombiGene expands its scope to include metabolic diseases. The initial goal of the project is to develop a gene therapy treatment for partial lipodystrophy, a very rare disease that today completely lacks adequate treatment. The project is at an early stage of development.

Priority-based patent application

At the beginning of autumn 2020, CombiGene filed a priority patent application with the UK Patent Office for patent protection of the vectors developed within the lipodystrophy project CGT2. The filing of this patent application paves the way for global patent protection for CGT2, which is of great importance for protecting key functions of CGT2 during further development and commercialization.

Selection of drug candidate

CombiGene is currently evaluating various drug candidates with the goal of selecting the final candidate for further preclinical studies in 2021. A first selection of the drug candidates has been made in in vitro experiments with liver cells and CombiGene has started the first out of two in vivo studies to evaluate which protein expression can be achieved in experimental models.

The first in vivo study aims to measure the levels of protein expression from the different drug candidates and the organs in which it is expressed. The second in vivo study is performed to measure the effect of the different candidates on the storage of fat in different organs.

About lipodystrophy

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 only 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 at all.

Collaboration partners


Lipigon is developing new treatments for patients with lipid-related diseases. The company’s foundation is based on 50 years of lipid research at Umeå University. The company’s primary focus is on orphan drugs and niche indications.

Stockholm University

In the spring of 2020, Brazilian researcher Ruda Feitoza was linked to the CGT2 project. He has a PhD in how drugs and nutrient uptake effect the function of mitochondria. Ruda is active at the Wenner-Gren institute at Stockholm University. His current focus is, together with his supervisors professors Barbara Cannon and Jan Nedergaard, to understand in detail the mitochondrial functions and conditions of the liver, the organ at the heart of the CGT2 project.

CombiGene s project CGT2 is supported by the Eurostars Programme.Project ID: 114714

About CombiGene

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 has an exclusive collaboration and licensing agreement for the CG01 project with Spark Therapeutics.

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



CombiGene AB, Agavägen 52A, SE-181 55 Lidingö


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