AVROBIO has announced positive follow-up data from a collaborator-sponsored Phase I/II trial investigating its gene therapy in patients with cystinosis. The trial is sponsored by the University of California, US.

The fully enrolled clinical trial (NCT03897361) monitored long-term safety and efficacy in six adults affected by the most severe and common dorm of cystinosis who were previously treated with cysteamine, the existing standard of care (SOC).

Cystinosis is a genetic condition characterised by a build-up of cystine crystals in the body. The build-up can cause progressive organ and corneal damage, swallowing dysfunction, chronic kidney disease and muscle wasting.

Patients underwent hematopoietic stem cell (HSC) mobilisation and collection, which were then genetically modified to express a functional version of cystinosin, a protein that is deficient in this patient population.

The follow-up data analysis showed that after receiving HSC gene therapy, patients were able to produce functional cystinosin throughout the body. This resulted in decreased levels of leukocyte cystine in the blood below baseline in all six patients and stabilised up to 36 months after the treatment.

Additionally, skin, and gastrointestinal mucosa biopsies showed a decline in tissue cystine crystals below baseline in the first four patients, who were observed after at least 12 months and two patients, who were observed up to 24 months.

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The first four patients treated with the gene therapy demonstrated an improvement or stabilisation of scores on the Beery – Buktenica Developmental Test of visual motor integration 36 months after treatment. This finding suggests a potential impact on the neuropathy of the disease.

The preliminary data from the trial suggested that the HSC gene therapy was well tolerated, with no adverse events (AEs) related to the drug product. All AEs were related to myeloablative conditioning, stem cell mobilisation, the underlying disease, co-morbid or pre-existing conditions. Avrobio first reported positive interim data in May 2022.

Avrobio also announced it had positive and productive meetings with the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and the US Food and Drug Administration (FDA) to align on regulatory pathways and obtain feedback on the program. Avrobio previously teased about a company-sponsored clinical trial which is expected to start in 2023.

Last year, Avrobio were not so successful in the rest of its gene therapy pipeline. Despite earlier promising results, the company cancelled its Fabry disease gene therapy after disappointing data from a Phase II trial. Two years earlier, experts were divided if then-known data in one patient was clinically relevant.

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