Category: Research news

Ophthalmology clinical research activity on the increase in the UK

November 22, 2018

Ophthalmology research in the NHS has grown and become more accessible to patients over the last eight years, according to a newly published report in the journal Eye. This is …

Widespread errors in “proofreading” cause inherited blindness

October 12, 2018

Mistakes in “proofreading” the genetic code of retinal cells is the cause of a form of inherited blindness, retinitis pigmentosa (RP) caused by mutations in splicing factors. This new understanding …

First gene therapy for inherited retinal disease takes a step closer to European approval

September 23, 2018

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended approval of the gene therapy Luxterna for the treatment of inherited retinal dystrophy caused by mutations …

Investigating a revolutionary gene therapy technique for Usher syndrome

September 14, 2018

Thanks to support from our wonderful community, including those who participated in the Big Give Christmas Challenge last year, we have been able to commit funding to a three-year research …

Valproic acid no better than placebo for autosomal dominant retinitis pigmentosa

September 11, 2018

The results of a clinical trial did not provide support for the use of orally administered valproic acid for the treatment of autosomal dominant retinitis pigmentosa, as it did not …

Scientists Reverse Congenital Blindness in Mice

September 7, 2018

A team of scientists led by the Icahn School of Medicine at Mount Sinai has successfully reversed congenital blindness in mice by changing supportive cells in the retina called Müller …

ProQR Announces Positive Interim Results from Phase I/II Clinical Trial of QR-110 in LCA10 Patients, and Plans to Start a Phase II/III Pivotal Trial

September 5, 2018

ProQR Therapeutics, developers of transformative medicines for the treatment of rare genetic diseases, today announced results from its Phase I/II trial of therapy QR-110 in patients with Leber’s congenital amaurosis …

New therapy shows promise in LCA disease models

August 17, 2018

Researchers in London and the Netherlands have demonstrated the ability of a potential therapeutic molecule, known as QR-110, to help correct the defects associated with the most common disease-causing mutation …

MeiraGTx Receives Orphan Drug Designation from the U.S. FDA for the Treatment of Achromatopsia

August 15, 2018

MeiraGTx has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for its AAV-CNGA3 gene therapy product candidate for the treatment of achromatopsia (ACHM) …

AGTC enrols first patient of second cohort for XLRP trial

July 25, 2018

Applied Genetic Technologies has enrolled the first patient of the second cohort in a Phase l/ll clinical trial to examine the safety and efficacy of an unnamed investigational AAV-based gene …

Gene therapy for dogs could help RP patients

June 8, 2018

A US clinician has received a five-year, £6.1 million grant to investigate the potential of advancing a gene therapy currently used in dogs to help retinitis pigmentosa patients. Michigan State …

Researchers develop a new CRISPR technique to restore retinal function in mice with RP

May 29, 2018

Researchers from Columbia University have developed a new technique for the powerful gene editing tool CRISPR to restore retinal function in mice with retinitis pigmentosa. This is the first time …