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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 Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 07, 2021
Details:
Illuminate is a Phase 2/3 trial, which randomized 36 patients aged eight years or older to receive either sepofarsen at the target registration dose, a low dose, or sham treatment.
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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 Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable 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.
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