Applicability of a structure-based modelling approach as a tool for in silico screening of oligonucleotides for targeted RNA editing presented at Oligonucleotide Therapeutics Society (OTS) 2018 by Julien Boudet
2017 may become a very exciting year for Patricia Biasutto, sepofarsen captain. Her work, developing a novel RNA therapy for Leber congenital amaurosis 10, has progressed steadily. The team expects to start a first clinical trial this year.
There are currently around 160 people working at ProQR, but that wasn’t always the case. When Hee Lam Chan joined in 2013, she was only employee number six. As a result, she has seen the company grow and develop.
… long before that Gerard Platenburg was involved in the development of RNA therapies. Even so, after decades of … RNA therapies.” But what about gene therapy, isn’t that an option? “There are several reasons to choose RNA therapy over … Can we learn more about our molecules, and does it help to optimize our protocols? Those are just some of the questions …
… with Lilly and Company . The partnership focuses on developing potential medicines using our Axiomer™ RNA editing … is to progress new medicine technology towards clinical development and, ultimately, to patients via … into diseases of which there are little to no treatment options. In our vision partnerships are true catalyst for the …
… collaboration with Lilly. The partnership focuses on developing potential medicines using our Axiomer RNA editing … beginning, ProQR has been focused on RNA technologies to develop medicines for people and families with high unmet medical need. In August …
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.
This presentation highlights the therapeutic possibilities of Axiomer platform that are not limited to disease causing mutations and can potentially address high unmet medical needs by editing wild-type RNA to engineer proteins or modify their function as well as creating de novo mutations.
This virtual event on March 29, 2023 featured presentations by ProQR Management on the company's proprietary Axiomer® RNA editing technology platform, detailed the initial pipeline targets, and provided guidance on the advancement of programs toward the clinic. ProQR also reported its 2022 year-end financials and the extension of its cash runway guidance.
