FDA approves Oxford UshStat IND application
UshStat utilizes the company’s LentiVector platform technology to deliver a corrected version of the MYO7A gene to address the vision loss associated with the disease. The Phase I/IIa
BioMarin Pharmaceutical and n-Lorem Foundation have entered an early-stage research collaboration and exclusive global licence agreement to develop an investigational antisense oligonucleotide (ASO) therapy for ReNU syndrome.