A 6.5-month-old boy with the rare inherited urea cycle disorder ornithine transcarbamylase (OTC) deficiency has responded positively in a targeted in vivo gene editing trial, in which a correct copy of a defective gene was inserted at a precise locus in the genome.

Iecure Inc. has received clearance from the Australian Therapeutic Goods Administration (TGA) of the company’s clinical trial notification (CTN) for ECUR-506 (formerly GTP-506), an investigational therapy in development for the treatment of ornithine transcarbamylase (OTC) deficiency in pediatric (or neonatal) patients.

The U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) and the Gene Therapy Advisory Committee (GTAC) have approved a clinical trial application (CTA) submitted by University College London (UCL) to initiate a phase I/II trial of BGT-OTCD, Bloomsbury Genetic Therapies Ltd.’s liver-targeted AAV-LK03 gene therapy, in pediatric patients with ornithine transcarbamylase deficiency (OTCD).

Ornithine transcarbamylase deficiency (OTCD) is an X-linked genetic condition, the most common...

The prospects for Ultragenyx Pharmaceutical Inc.'s adeno-associated virus (AAV) gene therapy, DTX-301, in patients with ornithine transcarbamylase (OTC) deficiency weren't looking too great after treatment of the first two cohorts in a phase I/II study.