The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has granted approval for Avrobio’s clinical trial application (CTA) to begin a Phase I/II clinical trial of gene therapy AVR-RD-05 in infants with Hunter syndrome.
Submitted by collaborators of Avrobio at the University of Manchester (UoM), UK, the CTA also received approval from the Research Ethics Committee (REC) and the Health Research Authority (HRA).
The first-in-human, non-randomised, 24-month, open-label, paediatric trial will enrol up to five children aged between three months or above and 12 months or below.
It will assess the tolerability, safety, pharmacodynamic, and clinical efficacy of the gene therapy.
The first trial subject is anticipated to be dosed in the first half of next year.
AVR-RD-05 is an investigational, autologous hematopoietic stem cell (HSC) gene therapy.
It can convert autologous HSCs ex vivo using a lentiviral vector that encodes for a brain-targeted iduronate-2-sulfatase (IDS) enzyme.
This gene therapy has obtained rare pediatric disease and orphan drug designations from the US Food and Drug Administration (FDA).
Avrobio president and CEO Geoff MacKay said: “Hunter syndrome causes complications throughout the body, including severe neurological, cardiac, and respiratory dysfunction, skeletal malformations, and hearing impairment.
“This investigational HSC gene therapy, which includes CNS-targeted gene expression, has been designed to express supra-physiological levels of the IDS enzyme in transduced HSCs, with the intent to correct both the systemic and neurological manifestations of this disease.”
Also called neuronopathic mucopolysaccharidosis type II (nMPS-II), Hunter syndrome is a rare ailment that mainly impacts young males.
In May this year, the company reported positive interim data from a Phase I/II trial of investigational gene therapy AVR-RD-04 for cystinosis.
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