HFR will support the manufacture of a groundbreaking new treatment for cystinosis, following the award of £3.9 million from the Medical Research Council (MRC) to progress the drug into clinical trials.
The therapy, known as CF10, was discovered by the late Professor Roz Anderson at the University of Sunderland and is designed to improve treatment for patients living with the rare, life-threatening genetic condition.
Cystinosis causes a build-up of cystine within cells, leading to progressive organ damage. Current treatment options are limited and often associated with significant side effects. CF10 has been developed to deliver treatment directly into cells, with the potential to reduce these side effects and improve patient outcomes.
HFR will manufacture CF10 for use in the first-in-human trial, working as part of a wider collaboration that includes University Hospitals Birmingham NHS Foundation Trust and the University of Birmingham.
The trial marks a major milestone in the development of CF10 and represents a significant step forward in the search for improved treatments for cystinosis.
Read the full press release for more details.
https://www.sunderland.ac.uk/news/2025/new-cystinosis-drug-clinical-trials
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