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Research News
Applied Genetic Technologies Corporation (AGTC), a leading developer of gene therapies, has reported the six-month follow-up data in all patients treated with its gene therapy for X-linked retinoschisis (XLRS) in its Phase I/II clinical trial.
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FDA Authorizes Clinical Trial for CRISPR/Cas9 Therapy for LCA 10
Research NewsEditas Medicine, a company developing gene-editing treatments, has received authorization from the US Food and Drug Administration to launch a clinical trial for its emerging CRISPR/Cas9 therapy for people with a mutation in the gene CEP290, which causes Leber congenital amaurosis 10 (LCA10). LCA causes severe vision loss or blindness at birth.
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ProQR Receives FDA Authorization to Launch Clinical Trial for USH2A Therapy
Research NewsProQR, a biotech in the Netherlands developing therapies for rare diseases, has received authorization from the US Food and Drug Administration to launch a Phase I/II clinical trial for QR-421a, its treatment targeting mutations in exon 13 of the USH2A gene.
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Acucela Enrolling Patients in Phase 3 Trial for Stargardt Disease Treatment
Research NewsThe Seattle biotech Acucela is now enrolling participants in its Phase 3 clinical trial for emixustat hydrochloride, an emerging oral drug for slowing vision loss in people with Stargardt disease, an inherited form of a macular degeneration.
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Ophthotech to Boost Development of Gene Therapy for Best Disease
Research NewsOphthotech Corporation, a biopharmaceutical company focused on the development of therapies for orphan retinal diseases and age-related macular degeneration, has announced an evolving, commercial partnership with the University of Pennsylvania and University of Florida to develop a gene therapy for Best disease caused by BEST1 mutations.
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Foundation Invests $2.5 Million in Search for Elusive Retinal Disease Genes and Mutations
Research NewsSince 1989 genetic researchers, many funded by the Foundation, have identified approximately 270 genes linked to IRDs. In most cases, defects in a single gene can cause a retinal disease and vision loss.
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Recording Available for Achromatopsia Teleconference Hosted by AGTC, Foundation, and Achroma Corp
Research NewsApplied Genetic Technologies Corporation (AGTC), Achroma Corp, and the Foundation Fighting Blindness hosted a one-hour teleconference on the condition. The call highlighted the difficulties patients have in getting a diagnosis for achromatopsia, the importance of genetic testing, and gene therapy clinical trials underway.
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FFB Congratulates RPE65 Gene Therapy Researchers for Champalimaud Award
Foundation NewsMore than two decades ago, the Foundation Fighting Blindness (FFB) began funding RPE65 gene therapy research that led in late 2017 to LUXTURNA™, the first FDA-approved gene therapy for the eye or an inherited condition. Ultimately, the Foundation provided more than $10 million in funding for the groundbreaking effort.
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Beacon Stories
Kai Wang was 18 months old when he was diagnosed with the condition. His parents never imagined the extraordinary journey they would take with their son when they learned he had a condition that would render him blind.
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Apellis Launches Phase 3 Clinical Trial Program for Advanced Dry AMD Treatment
Research NewsTrial participants will receive intravitreal injections of APL-2, or a sham procedure (placebo), monthly or every other month. The injections are made into the vitreous, the soft gel in the middle of the eye.
