The lipodystrophy project reaches a significant milestone
Covid-19 is sweeping the world. A very considerable share of healthcare resources is now being devoted to those who have fallen ill with covid-19 and enormous research efforts are being made to develop a vaccine for the disease and to find drugs that can ease symptoms and shorten the stay in hospital. At the same time the research community continues its long-term work within a range of other areas that do not receive as much media attention, but will nonetheless have great significance for the care sector and patients in the future. An example of this is CombiGene’s CGT2 project, of which the aim is to develop a gene therapeutic treatment for the rare disorder partial lipodystrophy.
CombiGene inlicensed CGT2 from the Umeå-based company Lipigon Pharmaceuticals in autumn 2019. Since the project was inlicensed the pace of development has accelerated and we are now seeing the first results in the form of a completed design of the so-called expression plasmids, which are the starting material for the gene therapy vectors that will treat the disorder. This is a significant milestone for the project.
Ingeneious contacted Annika Ericsson, Senior Project Manager at CombiGene, to discuss the CGT2 project.
Can you describe the disease partial lipodystrophy and the CGT2 project in layman’s terms?
“I’ll do my best,” replies Annika. “Lipodystrophy is a medical condition that is characterized by abnormal distribution of fat in the body. This can lead to a range of medical complications such as hepatic steatosis (fatty liver disease), diabetes and various cardiovascular diseases. With the CGT2 project we are attempting to develop a gene therapy for treating accumulation of fat in the liver. If we succeed, the treatment will probably also have a positive effect on the other conditions, i.e., diabetes and cardiovascular diseases.”
Can you give us an idea of how far you have come?
“CGT2 is in an early preclinical phase. We have conducted a number of experiments and we have designed the expression plasmids which are the starting material for the gene therapy vectors with which we intend to treat the disease. This is a major advance, although it is the first step of a long journey.”
Has the covid-19 pandemic impacted the way you work, or slowed the pace of the project?
“No, I wouldn’t say that. Our project organization was ready for a pandemic before covid-19. Four people are the core of the organization. Björn Eriksson and Ingela Bergqvist work at Lipigon’s lab in Umeå and the Brazilian researcher Ruda Feitoza is working at Stockholm University. I am the fourth member of the group and I have overall responsibility for the project. Since the project team is spread throughout the country, the obvious choice was to rely on digital communication right from the outset. So far, this has worked very well and I am satisfied that the project is off to a good start,” concludes Annika.
Brazilian researcher joins the project
In spring 2020 Brazilian researcher Ruda Feitoza joined the CGT2 project. His doctoral studies focussed on how mitochondrial function can be affected by drugs and nutrient uptake. Mitochondria are usually described as the powerhouses of the cell and transform nutrients into energy. Ruda is now working at the Wenner-Grens Institute at Stockholm University. His current work concerns detailed understanding of the mitochondrial functions and conditions in the liver, the organ which is the focus of the CGT2 project.
What are your impressions of Sweden?
“I am impressed by the nature and the way Stockholm has developed in relation to its surroundings. I think the first challenge for me will be the weather. But, since I arrived in the spring, adapting to the climate has so far been very easy.”
What has it been like for you to leave Brazil for Sweden during the ongoing pandemic?
“Of course, I have been worried and I often think of my family in Brazil. The virus is widely spread in both Sweden and Brazil. But I keep in close touch with my family and I am convinced that we are all taking the right precautions. As for my day-to-day life, I follow the authorities’ recommendations and have so far had no real difficulties. The project is in an early stage of development and we have not yet experienced any significant covid-19-related problems, as we have been able to hold our meetings digitally.”
Ingenious contacted CombiGene’s CEO, Jan Nilsson, to hear his thoughts on the development of the CGT2 project.
“I am very pleased with the way the project has developed since we inlicensed it from Lipigon,” says Jan. “We are still in an early phase, but the fact that we now have access to the starting material for production of the future gene therapy vectors is quite an important milestone. Collaboration with Lipigon is excellent and Ruda Feitoza’s work at the Wenner-Grens Institute will give us a detailed understanding of how we can control the liver’s mitochondrial functions. The CGT2 project, of which the ambition is to treat partial lipodystrophy, is very interesting. I am also convinced that the project will generate a lot of knowledge that will aid in the treatment of other metabolic disorders, which may eventually lead to other interesting projects.”
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 firstname.lastname@example.org.