Category: Research news

AGTC enrols first patient of second cohort for XLRP trial

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 therapy for the treatment of X-linked retinitis pigmentosa (XLRP). The open-label, dose-escalation trial is being conducted as part of a collaboration between AGTC and Biogen. …

Gene therapy for dogs could help RP patients

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 University veterinary ophthalmologist, Simon Petersen-Jones, has highlighted in research published in The Journal of Clinical Investigation that the treatment restored night vision and stopped the …

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

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 researchers have successfully applied CRISPR technology to a type of inherited disease known as a dominant disorder. Dr Stephen H. Tsang, and his colleagues sought …

Researchers create a retina cell network on a chip modelled after a humans neural network

Scientists have developed a new lab-on-a-chip technology that could quickly screen potential drugs to repair damaged neurons and retinal connections. Such damage is seen in people with RP, macular degeneration or those who have had too much exposure to the glare of electronic screens, according to the study published in the journal Science Advances. Researchers …

Increasing Eye Cells’ Ability to Process Misfolded Proteins May Prevent Retinal Degeneration

Researchers at Duke University believe they have developed an approach to treat retinal conditions (including retinitis pigmentosa), all of which create misfolded proteins that cells in the eye cannot process. The scientists have shown that boosting the cells’ ability to process misfolded proteins could keep them from aggregating inside the cell. They devised and tested …

First Patient Dosed in XLRP Treatment Trial

Applied Genetic Technologies Corporation has announced that the first patient was dosed in its Phase 1/2 clinical trial evaluating the safety and efficacy of AGTC-501 for the treatment of X-linked retinitis pigmentosa (XLRP). XLRP is an inherited condition that affects young boys and men. It causes the development of night blindness and eventually leads to …

ReNeuron wins grant to advance development of stem cell therapy

UK-based developer of cell-based therapeutics, ReNeuron Group, has won a grant to advance the development of its hRPC stem cell therapy candidate for blindness-causing degenerative diseases of the retina. The grant, which has been awarded under Innovate UK’s Medicines and Manufacturing Round 1: Challenge Fund, is worth £1.5 million. It will be used to co-fund …

ProQR Usher clinical development programme

ProQR is a young biotech firm based in the Netherlands that is in the early stages of planning a number of clinical trials targeting vision loss in Usher 2A patients (due to mutations in Exon 13 of Ush2A gene). Their approach uses RNA oligonucleotide therapy to make changes to the RNA of these patients, with …

Gene Therapy Trials for Best Disease could start in two years

Scientists in the U.S. say human trials of gene therapy for the inherited form of blindness known as vitelliform macular degeneration, or Best disease, could be less than two years away, following successful use of the treatment in a canine model of the disease. Developed through a longstanding collaboration between scientists at the University of …

Immune cells in the retina can spontaneously regenerate

Immune cells called microglia can completely repopulate themselves in the retina after being nearly eliminated, according to a new study in mice from scientists at the National Eye Institute (NEI). The cells also re-establish their normal organisation and function. The findings point to potential therapies for controlling inflammation and slowing progression of rare retinal diseases …

Nightstar Therapeutics announces initiation of STAR Phase III Registrational Trial for NSR-REP1 in Choroideremia

Nightstar Therapeutics PLC, a clinical-stage gene therapy company developing treatments for rare inherited retinal diseases, has announced the initiation of the company’s STAR Phase III registrational trial to study the safety and efficacy of NSR-REP1 in patients with choroideremia. In data from 32 patients treated with NSR-REP1 across four open-label Phase I/II clinical trials, over …

MeiraGTx Receives EMA PRIME Designation for Achromatopsia Gene Therapy Candidate

MeiraGTx, a London and New York based gene therapy company, has announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to its gene therapy product candidate A002 for the treatment of patients with achromatopsia due to mutations in the CNGB3 gene. PRIME is an initiative launched by EMA in 2016 to enhance …