Category: Research news

ABCA4 midigenes reveal the full splice spectrum of all reported noncanonical splice site variants in Stargardt disease

December 5, 2017

Based on the work of Riccardo Sangermano, Mubeen Khan, Stephanie Cornelis, PhD candidates in the group of prof. Frans Cremers in the Department of Human Genetics, Radboudumc, Nijmegen, The Netherlands, …

ProQR Therapeutics announces QR-110 trial

November 21, 2017

ProQR Therapeutics N.V. has announced that the first patient has been dosed in the Phase I / II open-label trial assessing the safety, tolerability, pharmacokinetics and efficacy of QR-110. The …

French Biotech firm raises €19M for gene therapy trial RP

November 13, 2017

The French company Horama has announced the closing of its Series B round, which has ammassed a total of €19M. The funds will be used to finance the development of …

ReNeuron makes progress with hRPC trials

November 3, 2017

ReNeuron said its hRPC clinical trial’s data safety monitoring board has given approval for the study to progress into its Phase II element. That decision was said to be based …

Scientists demonstrate powerful ability to manipulate building blocks of life in two separate studies

October 27, 2017

One study altered the order of atoms in DNA to rewrite the human genetic code and the instructions for life. The other edited RNA, which is a chemical cousin of …