GeN is committed to building an end-to-end platform to develop therapies for rare diseases. We are using the entire portfolio to validate new models and for specific rare diseases usingwell-established methodologies to target specific endpoints and biomarkers.

‍GeN has developed tools for quicker and more translational preclinical studies that will help push more clinical candidates targeting rare diseases into the clinic. This includes investigating the mechanisms of action using appropriate behaviour batteries tailor to address .

Animal Models‍

•  Fragile X Syndrome mice (in different backgrounds)
•  Rett Syndrome
•  Pitt Hopkins Syndrome
•  Angelman Syndrome
•  CDKL5 Deficiency Disorder
•  Dravet Syndrome
•  Huntington′s
•  Disease Phelan McDermidASD / Shank3 Feng

A small rodent with a body length of 25 cm and a weight of 170 to 300 grams. 

Degus entered the research spotlight due to their unique relationship with sugar and diabetes, but are also studied for a wide variety of other reasons. Neuroscientists use degus for research into Alzheimer’s Disease (AD), they are the only mammals that naturally develop Alzheimer’s at 2.5-3 years of age. In elderly degus, neural markers have been discovered which are remarkably similar to those in humans with Alzheimer's, which is the first time this has been seen in a non-human mammal.

These are also animals with good eye-and-paw coordination and circadian rhythm: degus have the ability to show both diurnal and nocturnal rhythms if the environment permits, allowing a unique opportunity for study. Degus have also been found to spontaneously stack objects in order of decreasing size: it is the first time that this behaviour has been recorded in animals other than apes.Octodon degus are rodents that are becoming more widely used in the neuroscience field. Degus display several more complex behaviors than rats and mice, including complicated social behaviors, vocal communications, and tool usage, for these complex behaviors to be correlated with specific