ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need.
Today we are delighted to announce the further expansion of our collaboration with Lilly. Lilly is a leader in innovative RNA science, making them a great partner for our Axiomer technology.
Bart Klein was at the forefront of ProQR's Axiomer® RNA editing technology development. This platform technology makes it possible to use the cells own machinery to reverse mutations that cause a genetic disease. Read the interview.
ProQR has announced the completion of an in-depth strategic review designed to deliver on our commitment to advance RNA therapies for diseases with high unmet need. We are also sharing additional analyses of the sepofarsen Illuminate trial.
He is one of the founders of ProQR, but even long before that Gerard Platenburg was involved in the development of RNA therapies. Even so, after decades of working on them he still gets enthusiastic when talking about RNA.