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[{"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Receives Rare Pediatric Disease Designation from FDA for QR-421a","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"ProQR Therapeutics"},{"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Announces Positive Findings From an Interim Analysis in the Phase 1\/2 trial of QR-421","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"ProQR Therapeutics"}]

ProQR Therapeutics

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ProQR Announces Positive Findings From an Interim Analysis in the Phase 1/2 trial of QR-421

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.

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

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ProQR Therapeutics

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ProQR Receives Rare Pediatric Disease Designation from FDA for QR-421a

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.

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

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