Gene Therapy Takes Center Stage at 2019 NORD Summit

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…

Isis Pharma Receives $5 Million Milestone Payment From GlaxoSmithKline

Isis Pharmaceuticals received a $5 million milestone payment from partner GlaxoSmithKline following news of its investigational drug ISIS-GSK5Rx being granted the development candidate designation. ISIS-GSK5Rx is the second medication created as a result of the collaboration between the two companies and is an antisense drug, indicated for the treatment of an undisclosed ocular disease. Among other…