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            Lead Product(s): Sepofarsen

            Therapeutic Area: Genetic Disease Product Name: QR-110

            Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Available

            Deal Size: Not Available Upfront Cash: Not Available

            Deal Type: Not Available November 02, 2020

            Details:

            Sepofarsen (QR-110) is being evaluated in the pivotal Phase 2/3 Illuminate trial and is a first-in-class investigational RNA therapy designed to address the underlying cause of Leber congenital amaurosis 10 due to the p.Cys998X mutation in the CEP290 gene.

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            Lead Product(s): Sepofarsen

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 08, 2020

            Details:

            The presentation will highlight results of a phase 1b/2 trial of intravitreal (IVT) sepofarsen (QR-110) antisense oligonucleotide in Leber congenital amaurosis 10 (LCA10) due to p.Cys998X mutation in the CEP290 gene.