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            Lead Product(s): QR-421a

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 31, 2020

            Details:

            QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse events.

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            Lead Product(s): QR-421a

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 30, 2020

            Details:

            QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa.