The now submitted PCT application builds on the
“The PCT application is important to protect the work we have put into the project so far in a cost-effective way” says
About CGT2
The goal of the CGT2 project is to develop a gene therapy treatment for partial lipodystrophy, a rare disease characterized by altered fat distribution on the body. Patients suffer from body fat atrophy. In the absence of normal body fat, various organs begin to accumulate fat, leading on to serious metabolic complications, including extreme insulin resistance, hypertriglyceridemia (elevated values of blood fat triglyceride) and liver steatosis (fatty liver). There are currently a few symptom-relieving treatments for lipodystrophy, but no therapy that targets the root cause of the disease. For patients suffering from partial lipodystrophy, there are currently no treatments at all.
Eurostars Programme
About
CombiGene’s vision is to offer patients affected by severe life-changing diseases opportunities for a better life through innovative gene therapies. CombiGene’s business concept is to develop effective gene therapies for serious diseases that today lack adequate treatment methods. Research assets are taken in from a network of external researchers and developed further up to clinical concept verification. Drug candidates for common diseases will be co-developed and commercialized through strategic partnerships, while
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