Drug development

Developing a new gene-therapeutic drug

Research begins with an idea as to how a disease can be attacked. This is often based on discoveries and research from academic laboratories. The first step is to establish the link between genes or processes in the body and a certain disease. The next step is to see how these genes/processes can change to alleviate or prevent the disease. Different substances are produced to assess which candidates have the best effect. This work is done both in vitro (e.g., biochemically or in cells) and in vivo in different animal models. Many substances are tested to determine which one is delivered to the right place in the body at a sufficient level and also binds or is expressed to an optimal degree. During the process patents are sought to protect the idea and/or the candidate drugs. The pre-clinical phase normally concludes with safety studies in animals and generally takes three to five years. Thereafter, the clinical phase, which can be further subdivided into several stages, begins.

Phase I.

Studies are done to ascertain whether the substance can be administered to humans with an acceptable safety and tolerability profile. The range of dosage is defined and how the substance is absorbed, distributed, metabolised and eliminated after dosing is studied.

Phase II.

The substance is tested on patients with the target disease to determine whether it has the postulated effect. An optimal dose is also determined. Documentation of safety and tolerability continues.

Phase III.

Large studies are often conducted to compare the substance with treatments currently used or, if the latter are lacking, with a placebo. The aim is to compare the efficacy of the substance with that of the currently available treatment. If the product has an equivalent or better effect, development continues. Concurrently, a range of other studies are conducted with respect to safety and efficacy, influence on other medicines, preparation forms, etc. After the clinical studies the documentation is compiled and submitted, together with a registration application, to the regulatory authorities in the countries in question.

Phase IV.

After market approval, so-called Phase IV studies are done to document the efficacy of the drug in a real-world, clinical setting. The authorities sometimes require approval whereby the company must, within a given period of time, generate certain specified knowledge about the medicine, something for which Phase IV studies are often used.

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 info@fnca.se.



CombiGene AB, Agavägen 52A, SE-181 55 Lidingö


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