AVRO - Avrobio stock rises as gene therapy for rare disorder cleared to enter pediatric trial in UK

Avrobio ( NASDAQ: AVRO ) said its autologous hematopoietic stem cell (HSC) gene therapy AVR-RD-05 was cleared to enter a phase 1/2 trial in U.K. to treat infants with neuronopathic mucopolysaccharidosis type II (nMPS-II) or Hunter syndrome.

nMPS-II is a rare genetic metabolic disorder due to deficiency or improper functioning of lysosomal enzymes which then affects several organs and impairs mental development, appearance and organ function, among other things. The gene responsible for MPS II is called iduronate 2-sulfatase (IDS) gene. In Hunter syndrome (also known as nMPS-II) the body does not have enough iduronate 2-sulfatase enzyme.

The U.K. Medicines and Healthcare Products Regulatory Agency (MHRA), Research Ethics Committee (REC) and Health Research Authority (HRA) approved the clinical trial application (CTA) filed by the company's collaborators at the University of Manchester, U.K. (UoM), the company said in a Sept. 14 press release.

The two-year study will enroll up to five children aged ?3 months and ?12 months with nMPS-II to evaluate the safety and efficacy of HSC gene therapy.

The company said AVR-RD-05 is designed to transduce a patient's own HSCs ex vivo with a lentiviral vector encoding a brain-targeted IDS enzyme, which is deficient in these patients.

UoM expects to dose the first patient in the trial in H1 2023.

AVRO +6.65% to $0.92 premarket Sept. 14

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