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Beskrivning

LandSverige
ListaFirst North Stockholm
SektorHälsovård
IndustriBioteknik
CombiGenes vision är att ge patienter som drabbats av svåra sjukdomar möjlighet till ett bättre liv genom genterapi och andra former av avancerade behandlingar. Bolagets verksamhet har tre fokusområden: inlicensiering av nya tillgångar, utveckling av dessa tillgångar till proof-of-concept samt utlicensiering av tillgångarna till en strategisk partner för fortsatt utveckling och kommersialisering. CombiGene has sitt säte i Stockholm.
2020-03-20 08:45:00

CombiGene recently concluded the preclinical pharmacokinetic study in the CG01 project. The outcome of the study is very promising and means that CombiGene now has the answers to key questions with respect to the long-term expression of NPY and Y2.

Successful pharmacokinetic study confirms that CG01 creates long-term expression of NPY and Y2
CombiGene's candidate drug, CG01, is being developed for treatment of patients with drug-resistant focal epilepsy. The aim is to enable a very long-term therapeutic effect through a one-time administration. To determine how the body takes up a drug and how lasting the effect is, so-called pharmacokinetic studies are conducted. CombiGene recently concluded a study of this kind, the results of which are extremely encouraging.

The concluded study clearly shows that the expression (occurrence) of neuropeptide Y (NPY) and its receptor, Y2, increases markedly after only one week following administration of CG01, and then continues to increase during the following two weeks, reaching a plateau after three weeks. It is particularly pleasing to note that the level achieved after three weeks remained stable throughout the duration of the study, i.e., six months.
“One question that often arises in our discussions with physicians is just how long we can expect to see an effect in humans after an injection and whether several injections will be necessary to maintain the effect. This study shows that the expression or occurrence of NPY/Y2 persists over a long period. This provides further evidence that a single injection should have an effect that lasts for many years. A rule of thumb is that six months in the experimental model we have used for our pharmacokinetic study corresponds to 15 years in humans.

The results from the pharmacokinetic study are extremely encouraging,” says a very satisfied Annika Ericsson, Senior Project Manager, CombiGene. Findings from the pharmacokinetic study are gratifying in other respects as well. Understanding how CG01 acts in the brain is also an important piece of knowledge that will aid planning of the coming safety studies (toxicology and biodistribution) in an optimal way. Knowledge gained from the pharmacokinetic study will also constitute a central component in the design of the first study in humans.