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AVROBIO Announces Neuronopathic Mucopolysaccharidosis Type II (nMPS-II) Or Hunter Syndrome Clinical Trial Application (CTA) Accepted By U.K. Regulatory Agency To Initiate Pediatric Phase 1/2 Gene Therapy Trial

First in-human pediatric Phase 1/2 study to evaluate the safety and tolerability of first-in-class gene therapy targeting infants age ≥3 months and ≤12 months, diagnosed with nMPS-II University of Manchester

Benzinga · 09/14/2022 07:29

First in-human pediatric Phase 1/2 study to evaluate the safety and tolerability of first-in-class gene therapy targeting infants age ≥3 months and ≤12 months, diagnosed with nMPS-II

University of Manchester (U.K.) expects to initiate clinical trial later this year

U.S. Food and Drug Administration (FDA) previously granted rare pediatric disease and orphan drug designations for AVR-RD-05, AVROBIO's investigational gene therapy for nMPS-II or Hunter syndrome

AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that the U.K. Medicines and Healthcare Products Regulatory Agency (MHRA), Research Ethics Committee (REC) and Health Research Authority (HRA) have approved the clinical trial application (CTA) submitted by AVROBIO's collaborators at the University of Manchester, U.K. (UoM) for initiation of the Phase 1/2 clinical trial of investigational autologous hematopoietic stem cell (HSC) gene therapy in infants diagnosed with neuronopathic mucopolysaccharidosis type II (nMPS-II) or Hunter syndrome. This rare and seriously debilitating lysosomal disorder primarily affects young males.