UNC Chapel Hill Angelman gene therapy rescues motor function; plus data backing planned Pacylex, Kronos clinical trials and more
BioCentury’s roundup of translational news
AAV gene editing therapy for Angelman syndrome
University of North Carolina at Chapel Hill scientists have created a CRISPR gene therapy for Angelman syndrome that ameliorated motor function deficits in a mouse model of the maternally inherited disorder. The AAV-delivered therapy targeting about 75 SNORD115 genes, described in Nature, restores expression of the paternal UBE3A allele by reducing expression of the non-coding RNAs.
Pacylex lipid modification inhibitor for blood cancers
A study led by Pacylex Pharmaceuticals Inc. and University of Alberta researchers has revealed that the company’s PCLX-001 had dose-dependent antitumor activity in xenograft mouse models of lymphoma, including patient-derived xenograft models. Pacylex said the proof-of-concept data reported in Nature Communications support a Phase I trial of the pan-N-myristoyltransferase inhibitor that is slated to start in early 2021...