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.
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.
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).
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.
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.
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 a study published in the journal Science Advances.
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 the strategy in mice, significantly delaying the onset of blindness.
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).
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.
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).