E!114714, CGT2, Combigene AB

Purpose and goal:

The project aimed to develop a novel gene therapy for partial lipodystrophy (PL). We intended to select a drug candidate with a unique mode of action in the treatment of PL and test its efficacy and safety in animal models. While the experiments were completed as planned, the project goal could not be met due to the lack of conclusive data on the effect of the CGT2 protein on liver lipids in animal models.

As a result, we could not achieve the expected outcomes needed for obtaining the proof of concept with the selected drug candidate and therefore decided to discontinue the project. Expected results and effects:

The expected outcome of the CGT2 project was a proof of concept for the lead gene therapy candidate for partial lipodystrophy (PL). Even with repeated studies and mitigation measures implemented, no conclusive results on the effect of the CGT2 protein on liver lipids in animal models were observed, which led to the conclusion that the ultimate outcome could not be achieved.

Instead, the completed work and the data generated in the project resulted in interesting scientific progress related to lipid metabolism, which is now being prepared for publication by our research collaborators. Approach and implementation:

CombiGene led the consortium with University Medical Center Hamburg-Eppendorf and Accelero Bioanalytics that later on declared bankruptcy and terminated its participation. The work included 3 scientific Work Packages. WP1 was dedicated to the evaluation of gene therapy vectors in animal models.

The lack of conclusive results on the effect of the CGT2 protein on liver lipids in animal models delayed further work in WP2 Expression of CGT2 in human liver cells and WP3 In vivo validation, leading to the inability to complete Proof of concept in its full intended scope.