Scientists behind Purespring Therapeutics Ltd. have made progress in overcoming the problems of accessibility, complexity of structure and diversity of cell types that have held back the use of gene therapy in the kidneys, reporting success in treating nephrotic syndrome in a mouse model of the inherited form of the renal disease.
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).
