Dutch biotech company Azafaros has secured significant funding to further its research into rare disease treatment.

The $28m Series A funding round, led by Netherlands-based life sciences venture capital firm Forbion, was announced on 6 February.

It also included participation from Azafaros’ founding company BioGeneration Ventures — a Dutch life sciences investor — and Swiss venture capital firm BioMedPartners.

Azafaros will use the investment to develop new ways of tackling lysosomal storage diseases (LSDs), which impair digestion and waste removal from the body, and other genetic metabolic disorders.

Dr Carlo Incerti, chairman of Azafaros, said: “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 which have the potential to meaningfully impact patients suffering from LSDs 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.”


Azafaros’ approach to rare disease treatment

Rare metabolic disorders are hereditary, life-threatening diseases that either impair the assembly of crucial metabolic end-products the body requires to function normally, or cause harmful build-ups inside the organs and other areas.

They can lead to a range of severe symptoms including organ enlargement or dysfunction, abnormal bone growth, impaired hearing and vision, developmental delay, and cognitive and neuropathological effects such as seizures and movement disorders.

Because they are triggered by genetic mutations, carriers of rare metabolic diseases can be affected from an early age. They can sometimes lead to the premature death of newborns and young children.

rare disease treatment newborn
Rare metabolic disorders can severely impact the life of a patient from a young age (Credit: US Air Force/Staff Sgt Eric T. Sheler)

Azafaros develops disease-modifying compound drugs that can treat these diseases regardless of a patient’s genotype — the individual genetic makeup of the cells in a person’s body.

In addition, Azafaros’ small-molecule compounds can readily distribute throughout the body’s organs to maximise their effectiveness.

The fact they are delivered orally makes them convenient for patients — especially younger ones — and preserves quality of life by providing long-lasting treatment at home.

They also avoid the risks associated with gene replacement therapies, such as infection, tumours, or a negative reaction from the immune system.