UNC Chapel Hill Angelman gene therapy rescues motor function; plus data backing planned Pacylex, Kronos clinical trials and more

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...