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News & Events

See what’s new in the field of hereditary eye diseases

Blog managed by the DBGen team, with updated therapies, news and events involving hereditary eye diseases. We invite you to take part in this forum.

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Leber Congenital Amaurosis DBGen Macular degeneration Genetic Diagnosis Retinal Dystrophy Gene editing Research Mutation Patients Stargardt Therapy Gene Therapy

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    New RNA therapy for autosomal dominant retinitis pigmentosa

    27/08/19 - Retinal Dystrophy, Gene Therapy,

    The United States Food and Drug Administration (FDA) has just approved the designation of the QR-1123 therapy by the Dutch company ProQR as a new investigational drug. This drug aims to treat a form of autosomal dominant retinitis pigmentosa (RPad) caused by the P23H mutation in the rhodopsin gene (RHO). QR-1123 is an antisense oligonucleotide, that is, a short RNA...

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    A race against the clock for Leber congenital amaurosis gene therapy

    14/08/19 - Leber Congenital Amaurosis, Retinal Dystrophy, Gene Therapy,

    ProQR Sepofarsen therapy On July 29, the Dutch company ProQR announced that the European Medicines Agency (EMA) has granted access to the Agency's priority medicine program (PRIME) to its Sepofarsen therapy (QR-110), designed for the treatment of Leber congenital amaurosis (LCA10), a childhood blinding disorder, caused by the c.2991+1655A>G (p.Cys998 *) mutation in the CEP290 gene. The EMA PRIME program...

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