where no one has gone before
Our drug pipeline consists of preclinical small molecule drugs that are designed to hit select targets with high potency and outstanding selectivity in difficult-to-reach tissues of interest like the brain or the eye.
These novel targeted therapies are entirely based on the application of our AI and wet-lab technology platform. We leverage synergies in the physiological disease mechanisms, the molecular targets, and the compound mode of action to target unmet medical needs. Our therapeutic small molecule research and development includes the simultaneous exploration and optimization of target-specific PET tracers as preclinical tools and clinical direct biomarkers.
We pursue a variety of promising protein kinase targets that have the potential to slow down, stop, or even revert the progression of Parkinson’s Disease (PD), Alzheimer’s Disease (AD) and other forms of acute and chronic neurodegeneration. We target both, the underlying cause of the disease like spreading of a-synuclein or phosphorylation of tau and amyloid-beta, and the corresponding microglia activation, cytokine signaling, and neuroinflammation. We have built a synergistic portfolio of highly selective, brain-penetrating small molecule drugs that have a high potential to become game-changers in the treatment of the largest burden for the aging and growing population.
Leaky blood brain barrier
The blood-brain-barrier consists of a tight layer of cells that protect the brain from all kinds of toxins, proteins, viruses, and infiltrating cells that circulate in the blood stream. In the aging population and in many devastating CNS diseases the barrier becomes more porous, and immune cells slip through and attack the nerve cells as in Multiple Sclerosis (MS) or cause epilepsy in Alzheimer’s Disease (AD), and lead to Vascular Dementia (VaD).
We have identified novel small molecules that reconstitute the tight junctions and improve blood-barrier-function in vitro and in vivo.
The vast majority of the approved anticancer drugs do not sufficiently cross the blood-brain-barrier. This means that metastasizing cells can hide and spread in the brain as the drug cannot reach an effective concentration there to fight them. In addition, primary brain cancers like glioblastoma represent a high unmet medical need.
We have created a portfolio of brain-penetrating small molecules that hit validated anticancer targets in the brain and in the system, thus aiming at the redefinition of the standard-of-care in cancer treatment.
Viral infections account for the majority of the severe infections worldwide. Limited options are available to combat novel viruses. Small molecule antivirals have the great advantage to target a broader spectrum of viruses, including yet unknown viruses. We focused on viral encephalitis, which is caused by a variety of common viruses like herpes simplex virus (HSV). As viruses can retract to compartments of the body that are not reached by current antiviral compounds, we optimized our antivirals for efficient brain penetration.
Since 2017, we have been using a combination of targeted design and phenotypic screening to identify and optimize antiviral small molecules with a novel mode of action. Our inhibitors show efficacy on multiple strains of DNA and RNA viruses including clinical isolates of resistance strains.
Update – November 2021: We have successfully extended our research program to include SARS-CoV-2. Our data confirms excellent target engagement and high specificity of several classes of our lead small molecule drugs. Viral load is reduced significantly at low concentrations. The PK shows excellent exposure in the lungs and brain, and that clinically significant exposures can be achieved at low doses. Our compounds may therfore provide a therapeutic treatment for Covid-19 and other viral infections.
The eye represents a particular organ that can be targeted systemically and topically. Topical administration to the eye, in particular as eye drops, has the advantage of a minimal drug exposure and reduction of unwanted side-effects in the body. In order to be efficacious and long-lasting, the small molecule drugs must display a very specific set of properties to reach the eye tissue, which is protected by a three-layer tear film.
We pursue novel targets to combat the sight-threatening host response to eye infection by fungi, virus, and bacteria. In addition, we target other forms of eye inflammation and tissue degeneration, as well as allergic conditions, to preserve and improve patient’s precious vision.