Patient One is a collaboration of academia, pharma, development and clinical experts, patient representatives and social investors, developing a novel formulation for the treatment of cystinosis, for a fair price that guarantees patient access across Europe.


Cystinosis is a rare, inherited, autosomal recessive disease that effects around 1 in 100.000 to 1 in 200.000 persons in the world. Patients with cystinosis are unable to transport cystine out of their cells, which leads to progressive cell-damage throughout their body over time.

Cystinosis patients are often diagnosed in the first year of life with symptoms such as slow growth, fragile bones, frequent urination, and dehydration.

Without treatment patients develop renal failure around the age of ten and loss of vision later in life.


While no cure exists, treatment with cysteamine bitartate can help manage patients’ symptoms and slow the disease’s progression.

There are two challenges, though:

1) Current treatments’ high frequency and volume make it hard to incorporate it into patients’ daily lives, while missing dosages allows for irreversible damage.

2) Having very few patients in the world, treatment pricing for rare diseases tends to be very high, challenging the accessibility for patients.


Patient One is developing a novel sustained-release cysteamine bitartrate formulation for the treatment of Cystinosis, for a fair price that guarantees patient access across Europe.

After years of development, Patient One has developed a novel formulation that shows great potential. To further investigate the product, additional clinical research is needed.


Our novel sustained-release cysteamine bitartrate formulation for and accessible price that guarantees patient access across Europe.


Between 2017 and the end of 2022, a group of clinical experts and social investors have invested in the development of this formulation, including a proof of concept trial and re-formulation.

To finance the next necessary pharmacokinetic and clinical trials, Patient One seeks new investors and experts who want to join our collaboration.


As Patient One we are working towards a world where everyone has access to the care they need, made possible by close collaboration and with the patient as priority.

higher sensitivity

Striving for accessibility for all patients without compromising on high quality care

higher sensitivity

Close collaboration with patient representatives and clinical experts

higher sensitivity

Fair pricing model that guarantees patient access across Europe


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