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Displaying 31–40 of 177 research results
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FDA Authorizes Clinical Trial for CRISPR/Cas9 Therapy for LCA 10
Editas 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
ProQR, 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
The 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
Ophthotech 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
Since 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
Applied 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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Apellis Launches Phase 3 Clinical Trial Program for Advanced Dry AMD Treatment
Trial 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.
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FFB Congratulates RPE65 Gene Therapy Researchers for Champalimaud Award
On September 4, 2018, seven researchers, including six previously funded by the Foundation, were recognized with the prestigious 2018 Antonio Champalimaud Vision Award for their contributions to the advancement of blindness-reversing RPE65 gene therapies.
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Vision Improvements Reported in ProQR's Clinical Trial for LCA10 Treatment
The company reported that 60 percent of subjects in the trial demonstrated improvements in visual acuity and their ability to navigate a mobility course. The treatment was also safe for patients.
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Ophthotech is Advancing an Impressive Portfolio of Cutting-Edge Therapies for Retinal Diseases
The company is taking on a multi-track strategy that includes retinal gene-therapy development, including delivery of over-sized genes and design of a two-step process of gene knockdown and replacement for autosomal dominant conditions.
