Cystinosis

Patient One

Developing PO-003, a sustained-release cysteamine bitartrate formulation designed to reduce treatment burden while keeping cystinosis therapy accessible.

Fair Medicine spin-off PO-003 Clinical development
Patient One cystinosis programme
Academic programme
Patient One
01
Opportunity + development

A proven medicine.
A better treatment experience.

Cysteamine can slow cystinosis progression, but immediate-release treatment requires strict, frequent dosing that is difficult to sustain in everyday life.

Patient One is developing PO-003, a sustained-release formulation intended for twice-daily dosing. Following proof-of-concept work and reformulation, the programme is preparing for further clinical testing.

Academic
science
Programme
structure
Development
Clinical
progression
Patient
access
01

Patient One

Cystinosis

02
What Orfenix adds

The structure around the science.

03
Access + next step

Designed for development.
Designed for access.

Access by design

Access should not become a question only after approval.

Patient One combines pharmaceutical development with a pricing and investment model intended to keep the treatment accessible once it reaches patients.

01 Cost-price-plus pricing
02 Transparent development costs
03 Pre-defined investor return
NEXT ↗
PK + clinical trials

Testing the final PO-003 formulation.

The next step is pharmacokinetic testing in healthy volunteers, followed by a patient study if the required profile is confirmed.

Built with Fair Medicine Foundation Tiofarma Nierstichting Nederland Cystinose Groep NL & Vlaanderen