The market for CombiGene’s lipodystrophy project

Partial lipodystrophy is a very rare disorder. Estimates place the number of patients in the USA and Europe at about 2,000. Therefore, according to CombiGene’s assessment, there are good prospects for obtaining orphan drug designation for the lipodystrophy project’s candidate drug. If orphan drug designation is granted for the candidate drug, CombiGene will have several significant advantages in the form of regulatory advice, tax relief, public funding, reduced fees and longer market exclusivity. Given the advantages of an orphan drug designation, CombiGene may potentially develop this project all the way to market under its own management.

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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