The potential of this new technology for RNA therapeutics is immense, says Bart Klein, who was at the forefront of the Axiomer® technology development. ProQR’s Sr. Vice President Innovation makes his point about the promise of Axiomer® technology: “This opens up a whole new way of designing medicines for genetic diseases that were previously ‘out of reach’ for existing technologies.”
2017 may become a very exciting year for Patricia Biasutto, sepofarsen captain. The work of her department, aimed at developing a novel drug for Leber congenital amaurosis 10 (LCA10) patients, has progressed steadily. The team is expecting to start a first clinical trial in patients this year.
