Scientists Reverse Congenital Blindness in Mice
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 …
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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
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
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 …
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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) …
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 …
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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 …
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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 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 …
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. …
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 …
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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 …
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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 …
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