New therapy shows promise in LCA disease models
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 in Leber congenital amaurosis type 10 (LCA10). Their results have been published in the journal Molecular Therapy: Nucleic Acids. LCA10 is a severe, early-onset inherited …
Continue reading “New therapy shows promise in LCA disease models”
MeiraGTx Receives Orphan Drug Designation from the U.S. FDA for the Treatment of Achromatopsia
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) caused by mutations in the CNGA3 gene. The FDA’s Orphan Drug Designation programme provides orphan status to drugs and biologics which are defined as those …
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. …
Continue reading “AGTC enrols first patient of second cohort for XLRP trial”
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 …
Continue reading “Gene therapy for dogs could help RP patients”
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 …
Continue reading “First Patient Dosed in XLRP Treatment Trial”
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 …
Continue reading “ReNeuron wins grant to advance development of stem cell therapy”
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 …
Continue reading “ProQR Usher clinical development programme”
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 …
Continue reading “Gene Therapy Trials for Best Disease could start in two years”
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 …
Continue reading “Immune cells in the retina can spontaneously regenerate”