Category: Research news

Blood-Retina Chip research

January 25, 2018

Researchers from Microelectronics Institute of Barcelona, Vall d’Hebron Research Institute, and Universitat Autònoma de Barcelona have developed a microfluidic chip that mimics the human blood-retinal barrier in the eye. The …

GenSight to start clinical testing combination of gene therapy and wearable device

January 11, 2018

GenSight will start a clinical trial in the UK testing a combination of gene therapy and a wearable device to restore sight in patients with retinitis pigmentosa. The UK Medicines …

Agreement formed for development of AAV vectors for ocular gene therapy

January 9, 2018

Sirion Biotech GmbH, a virus-based technology and gene delivery company, announced that it has signed a two-year development agreement with Acucela, a clinical-stage ophthalmology company. Recombinant adeno associated virus (rAAV) …

FDA approves Luxturna gene therapy treatment

December 20, 2017

The U.S. Food and Drug Administration has approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may …

jCyte Presents Results of Clinical Testing in RP

December 14, 2017

Cell therapy company jCyte has announced results from a phase 1/2a clinical trial for its investigational product, jCell, in retinitis pigmentosa. Presented by David S. Boyer, MD, at the annual …

Scientists explore how a pain medicine also protects vision in blinding conditions

December 8, 2017

Scientists have shown that a medicine prescribed for severe pain can help preserve vision in a model of severe, blinding retinal degeneration. Now they want to know more about how …

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 …