Home Analyses Status report CombiGene – In the front line of gene therapy

Status report CombiGene – In the front line of gene therapy

Status report CombiGene – In the front line of gene therapy

26 November, 2020

In recent years, gene therapies have emerged as one of the hottest areas within life science and the temperature is rising as the first treatments are now available to patients in the US and Europe. During the 2020s, several new therapies are expected to be marketed, a breakthrough period for these therapies that through one or a few treatments have the potential not only to relieve but to cure diseases. The attention focused on gene therapies has lately resulted in several acquisitions and licensing deals. On the Swedish stock market, there is only one listed gene therapy company, CombiGene. BioStock has now published a status report of the company, which can be downloaded below.

Lund-based company CombiGene is developing a new therapy for treating epilepsy with their drug candidate CG01 and they are also developing the candidate CGT2 in lipodystrophy, a rare metabolic disease.

Just to mention a few of the milestones during 2020, the company completed the manufacturing process of CG01 and the first large scale production of CG01 has also been finished in collaboration with CombiGene’s partner, gene therapy specialist Viralgen. This means that two critical steps in the development work have been completed.

In 2021, CombiGene will finalise the preclinical phase and move into the first human trials in 2022. The market potential for CG01 is significant, with estimated annual sales of 750 – 1500 million USD.

Furthermore, the CGT2 project has also advanced and recently CombiGene announced that the first in vivo study has been initiated for evaluating the different gene therapy vectors. Currently, there is no adequate treatment of partial lipodystrophy and CombiGene can potentially apply for orphan drug designation for CGT2, which would mean several strategic advantages.

With CGT2 CombiGene is aiming at a market with significant value, estimated to 700 – 1450 million USD annually and this is a market that the company can reach on its own.

Download the status report below.



This analysis is a Status Report by BioStock, conducted on commission by the analysed company. BioStock’s remuneration for the work has been agreed upon beforehand and is independent of the content and conclusions reached in the analysis. The content of this analysis is based solely on publicly available information gathered through research by the analyst from sources such as e.g. financial reports and statements, the company’s web page, public presentations and in dialogue with representatives of the company’s executive management. The information about the company was made available to the company for fact-checking and was published subsequent approval by the company. The analysis contains subjective assessments regarding future events and outcomes which should be considered to render such assessments to be uncertain. Any valuation of the company, products and/or markets has been made by BioStock’s analyst. The analyst does not own shares in the company. This analysis should not be considered as a recommendation or advice to invest in the company nor shall it be interpreted as financial advice of any sort. BioStock can not and does not give any guarantees that the conclusions or forecasts presented in this analysis will be fulfilled. BioStock can not be held responsible or liable for any damage, direct or indirect, caused by decisions made based on the information in this analysis. BioStock is not subject to the oversight of the Swedish financial authority Finansinspektionen and is thus not required to comply with the specific rules and guidelines that apply to analysis firms subject to the oversight of Finansinspektionen.

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