Wave Life Sciences: Lead DMD Exon 51 program on track to enter clinic this year
"We recently achieved two major milestones: transitioning to a clinical stage company, with the commencement of our two phase 1b/2a trials in Huntington's Disease, PRECISION-HD1 and PRECISION-HD2; and the opening of our new cGMP manufacturing facility," said Paul Bolno, M.D., MBA, President and CEO. "Our lead DMD Exon 51 program is on track to enter the clinic later this year, and I am excited to announce our next development program targeting C9orf72 mutations in ALS and FTD. Our collaboration with Pfizer continues to advance with multiple programs progressing through in-vivo lead optimization. Within our discovery engine, we continue to generate compelling data for new modalities such as single-stranded RNAi, as well as splice correction. With the progression of our pipeline and advancement in our platform capabilities, we are closer to potentially bringing new hope and precision medicines to patients living with serious, genetically-defined diseases."