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    [{"orgOrder":0,"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":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"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":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Announces Virtual Presentation of Phase 1\/2 Sepofarsen Data Through the Association for Research in Vision and Ophthalmology (ARVO)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Announces Virtual Presentations at American Academy of Ophthalmology (AAO)","therapeuticArea":"Rare Diseases and 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Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR to Focus Exclusively on Axiomer RNA-editing Technology and Partner Ophthalmology Programs","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Eli Lilly","pharmaFlowCategory":"D","amount":"$3,750.0 million","upfrontCash":"$75.0 million","newsHeadline":"Lilly and ProQR to Expand RNA Editing Collaboration","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","highestDevelopmentStatus":"Discovery Platform","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 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Ultevursen Ophthalmic Assets","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Rett Syndrome Research Trust","pharmaFlowCategory":"D","amount":"$1.0 million","upfrontCash":"Undisclosed","newsHeadline":"ProQR and Rett Syndrome Research Trust Join Forces with Axiomer\u2122 RNA Editing Collaboration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery Platform","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery Platform"}]

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              ProQR Therapeutics

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              ProQR and Rett Syndrome Research Trust Join Forces with Axiomer™ RNA Editing Collaboration

              Details:

              The collaboration will focus on the design and development of editing oligonucleotides (EONs) using ProQR’s Axiomer technology platform targeting the transcription factor Methyl CpG binding protein 2 (MECP2) and correcting mutations of interest.

              Lead Product(s): Undisclosed

              Therapeutic Area: Genetic Disease Product Name: Undisclosed

              Highest Development Status: Discovery Platform Product Type: Large molecule

              Partner/Sponsor/Collaborator: Rett Syndrome Research Trust

              Deal Size: $1.0 million Upfront Cash: Undisclosed

              Deal Type: Collaboration January 05, 2024

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              ProQR Therapeutics

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              ProQR Therapeutics Announces Transaction Completed for Théa to Acquire Sepofarsen and Ultevursen Ophthalmic Assets

              Details:

              The divestment provides Théa with ProQR's late stage ophthalmic assets, QR-110 (sepofarsen) and QR-421a (ultevursen) for patients with rare genetic eye diseases and supports ProQR’s strategic focus on the Axiomer® RNA editing technology platform.

              Lead Product(s): Sepofarsen

              Therapeutic Area: Rare Diseases and Disorders Product Name: QR-110

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

              Partner/Sponsor/Collaborator: Laboratoires Thea

              Deal Size: $186.3 million Upfront Cash: $8.6 million

              Deal Type: Divestment December 08, 2023

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              ProQR Therapeutics

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              ProQR Therapeutics and Laboratoires Théa Announce Agreement for Théa to Acquire ProQR’s Sepofarsen and Ultevursen Ophthalmic Assets

              Details:

              The divestment provides Théa with ProQR's late stage ophthalmic assets, QR-110 (sepofarsen) and QR-421a (ultevursen) for patients with rare genetic eye diseases and supports ProQR’s strategic focus on the Axiomer® RNA editing technology platform.

              Lead Product(s): Sepofarsen

              Therapeutic Area: Rare Diseases and Disorders Product Name: QR-110

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

              Partner/Sponsor/Collaborator: Laboratoires Thea

              Deal Size: $162.0 million Upfront Cash: $13.7 million

              Deal Type: Divestment August 01, 2023

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              ProQR Therapeutics

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              Lilly and ProQR to Expand RNA Editing Collaboration

              Details:

              Under the terms of the expanded agreement, Lilly will gain access to additional targets in the central nervous system and peripheral nervous system with ProQR’s Axiomer platform.

              Lead Product(s): RNA-based Therapy

              Therapeutic Area: Hepatology (Liver, Pancreatic, Gall Bladder) Product Name: Undisclosed

              Highest Development Status: Discovery Platform Product Type: Large molecule

              Partner/Sponsor/Collaborator: Eli Lilly

              Deal Size: $3,750.0 million Upfront Cash: $75.0 million

              Deal Type: Expanded Collaboration December 22, 2022

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              ProQR Therapeutics

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              ProQR to Focus Exclusively on Axiomer RNA-editing Technology and Partner Ophthalmology Programs

              Details:

              Following the results from the QR-110 (sepofarsen) Illuminate trial, the EMA has recommended an additional clinical trial be conducted for sepofarsen prior to submitting a Marketing Authorisation Application (MAA).

              Lead Product(s): Sepofarsen

              Therapeutic Area: Rare Diseases and Disorders Product Name: QR-110

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

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable August 11, 2022

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              ProQR Therapeutics

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              ProQR to Present its Axiomer RNA Editing Technology at the Oligonucleotide and Peptide Therapeutics Conference (TIDES USA) 2022

              Details:

              RNA base editing technology called Axiomer, potentially new class of medicines for genetic diseases. Axiomer “Editing Oligonucleotides” mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells.

              Lead Product(s): Editing Oligonucleotides

              Therapeutic Area: Technology Product Name: Undisclosed

              Highest Development Status: Preclinical Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable May 09, 2022

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              ProQR Therapeutics

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              ProQR to Present at the Retinal Cell and Gene Therapy Innovation Summit and the Association for Research in Vision and Ophthalmology (ARVO) 2022

              Details:

              Sepofarsen (QR-110) is an investigational RNA therapy designed to restore vision in Leber congenital amaurosis 10 due to the c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene.

              Lead Product(s): Sepofarsen

              Therapeutic Area: Rare Diseases and Disorders Product Name: QR-110

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

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable April 26, 2022

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              ProQR Therapeutics

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              ProQR to Present Axiomer RNA Editing Platform Technology at RNA Editing Summit

              Details:

              Axiomer editing oligonucleotides are designed to recruit an endogenously expressed RNA editing system called ADAR, which can direct change of adenosine to inosine in the RNA, and Inosine is translated as guanosine.

              Lead Product(s): RNA-based Therapeutic

              Therapeutic Area: Genetic Disease Product Name: Undisclosed

              Highest Development Status: Preclinical Product Type: Large molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable April 04, 2022

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              ProQR Therapeutics

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              ProQR Announces Top-Line Results from Phase 2/3 Illuminate Trial of Sepofarsen in CEP290-mediated LCA10

              Details:

              The study did not meet primary endpoint nor notable secondary endpoints for QR-110 (sepofarsen) an investigational RNA therapy and, no benefit observed in either treatment arm versus sham for the treatment of CEP290-mediated LCA10.

              Lead Product(s): Sepofarsen

              Therapeutic Area: Rare Diseases and Disorders Product Name: QR-110

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

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable February 11, 2022

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              ProQR Therapeutics

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              ProQR Announces Axiomer® RNA Editing Licensing and Research Collaboration with Lilly

              Details:

              This collaboration with Lilly, is an important validation for Axiomer® RNA editing platform of ProQR, which enables the editing of single nucleotides in RNA in a highly targeted and specific manner.

              Lead Product(s): Editing Oligonucleotides

              Therapeutic Area: Genetic Disease Product Name: Undisclosed

              Highest Development Status: Discovery Product Type: Large molecule

              Partner/Sponsor/Collaborator: Eli Lilly

              Deal Size: $1,300.0 million Upfront Cash: $50.0 million

              Deal Type: Collaboration September 08, 2021

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