The company Azafaros was established in 2018 to translate innovative science from Leiden University and Amsterdam University Medical Center into novel disease-modifying treatment modalities for rare genetic metabolic disorders such as lysosomal storage diseases.

A broad group of rare diseases

Lysosomal storage diseases such as Gaucher, Tay-Sachs, Fabry, Sanfilippo or Pompe disease, are a group of rare inherited metabolic disorders caused by lysosomal function defects. Lysosomes are cellular compartments filled with a variety of enzymes that are involved in the turn-over and degradation of proteins, polysaccharides, nucleic acids, or lipids. 
For example, dysfunctions in the aforementioned enzymes in the brain can result in the cytotoxic accumulation of degradation products, resulting in severe symptoms including developmental delays, seizures, respiratory infections, loss of vision and hearing, and cognitive functions.

Potential treatments based on patented compounds

Azafaros is developing a pipeline of LED3-sourced small molecules that have great potential to alter these disorders. Based on their mode of action, these compounds provide the prospect for a disease-modifying treatment combined with a convenient oral administration route that allows a life-long treatment. 

First Azafaros compound makes it to clinical trials

The company announced in 2021 that the first cohort of healthy subjects has been dosed in a two-part Phase 1 clinical study with clinical candidate AZ-3102, a first-in-class small molecule compound being developed for the treatment of rare neurogenetic disorders. Azafaros’ initial targets with AZ-3102 are GM1 and GM2 gangliosidoses, two rare life-threatening neurogenetic lysosomal storage disorders affecting infants, adolescents, and adults, for which there is currently only palliative care.