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Multiple System Atrophy (MSA) is a rare neurodegenerative disorder that belongs to the group of synucleinopathies and is also classified as an “atypical parkinsonism” disorder. Pathologically, MSA is characterized by abnormal deposits of the α-synuclein protein mainly in oligodendroglial cells and also in certain nerve cells. Typically, there is a dysfunction of the autonomic nervous system, i.e. disturbances of bladder function, erectile function, intestinal mobility, or the regulation of blood pressure and body temperature. Often, additional PD-like symptoms, such as slowness of movement and stiffness of the muscles, or a disturbance of cerebellar function with a progressive loss of balance or speech problems are experienced by the patient.

Parkinson`s disease (PD) is one of the most common diseases of the central nervous system. It is usually diagnosed between the ages of 50 and 79, with increasing incidences at an advanced age; men are affected more often than women. Drugs and supportive therapies can alleviate motor symptoms, but to date, there is no cure for PD.

Certain proteins including α-synuclein can form disease-specific fibrillar amyloid aggregates. The misfolding/ aggregation pathway resulting in amyloid fibrils involves characteristic oligomer species which have been shown to be the most relevant toxic protein species. These pathological oligomers exhibit structural epitopes that are not found in physiologically folded proteins or physiological protein assemblies, providing a specific structural target for anle138b, our lead candidate.