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Translating science into treatments of rare metabolic disorders

Leiden biotech startup Azafaros has successfully completed a funding round, raising 25 million euros of investments for developing treatments of rare metabolic disorders. The company holds exclusive license to a library of novel patented compounds discovered by experts from Leiden University.

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. The now completed so-called series A financing round was led by Forbion, with participation from BioMedPartners and founding investor BioGeneration Ventures.

'The Series A financing round is a very significant achievement and endorsement for Azafaros. It allows us to build our organisation and expand our executive team with passionate and experienced people and to advance our rare metabolic disorders pipeline,' commented Olivier Morand, Chief Executive Officer at Azafaros.

Exclusive license

Azafaros holds an exclusive license from Leiden University and Amsterdam UMC to a library of novel patented compounds discovered by internationally recognised experts in the field: Professors Hans Aerts, Hermen Overkleeft and Stan van Boeckel, and their co-workers at the Leiden Institute of Chemistry, Leiden University.

Professor Hans Aerts stated: 'Azafaros has secured the capital to develop a pipeline of first-in-class small molecules that have great potential for treating patients with rare diseases. 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.'

Meaningfully impacting patients' lives

Carlo Incerti, Chairman of Azafaros, added: 'I dedicated most of the last three decades of my professional life to the discovery and development of drugs for rare disorders. Azafaros represents the ideal continuation of this journey as it is developing new therapies that have the potential to meaningfully impact patients suffering from lysosomal storage disorders through a unique mode of action. These are major unmet medical needs, particularly in such diseases affecting the central nervous system. Azafaros’ lead oral small molecule holds the promise of becoming an innovative approach to treating these conditions.'

Read the full press statement

About Lysosomal storage disorders

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.

About Azafaros

Azafaros was founded in 2018 by a team of experienced industry professionals and scientists aspiring to address rare genetic metabolic disorders through a pipeline of oral small molecules with disease-modifying potential. Based on discoveries from Leiden University and Amsterdam UMC, Azafaros' proprietary lead compound AZ-3102 will initially address inherited life-threatening lysosomal storage diseases for which there are no effective therapies today. This orally available azasugar compound interferes with the metabolism of glycolipids and uniquely affects several key disease pathways through a dual mode of action. Leveraging the know-how of its team and partners in orphan drug development, the company is advancing its lead program toward first-in-man studies while further expanding its product pipeline into other rare metabolic diseases through its drug discovery efforts.

For more information, visit azafaros.com

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