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Displaying 421–430 of 732 for “Visions 2020”
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Dec 7, 2018
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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Dec 4, 2018
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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Nov 21, 2018
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.