Fourteen-month-old Tomas was diagnosed with ornithine transcarbamylase (OTC) deficiency when he was a few weeks old - becoming just one of 15 each year diagnosed with the rare genetic disease.

today announced that initial data from the Phase 1/2 OTC-HOPE clinical trial evaluating in vivo gene editing candidate ECUR-506 in neonatal onset ornithine transcarbamylase (OTC) deficiency will ...

The Recombinant DNA Advisory Committee (RAC), the National Institutes of Health office responsible for public oversight of gene therapy, met on 8–10 December to examine the clinical trial in ...