
Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target

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Lead Product(s) : AX-0810
Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)
Study Phase : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
ProQR Announces CTA Submission for Phase 1 Study of AX-0810 Targeting NTCP
Details : AX-0810 is an investigational GalNac-delivered RNA editing oligonucleotide designed to treat cholestatic liver disease by targeting NTCP.
Product Name : AX-0810
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
June 26, 2025
Lead Product(s) : AX-0810
Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)
Highest Development Status : IND Enabling
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : AX-2402
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery
Sponsor : RSRT
Deal Size : $9.1 million
Deal Type : Collaboration
ProQR Gets $8.1M from Rett Trust for RNA Editing Collaboration Expansion
Details : The collaboration will focus on designing and developing AX-2402, a Methyl CpG Binding Protein 2 (MECP2) therapy, for the treatment of Rett syndrome.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Undisclosed
December 11, 2024
Lead Product(s) : AX-2402
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery
Sponsor : RSRT
Deal Size : $9.1 million
Deal Type : Collaboration

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Lead Product(s) : AX-0810
Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)
Study Phase : Preclinical
Sponsor : Evercore ISI
Deal Size : $63.0 million
Deal Type : Public Offering
ProQR Announces Closing of Underwritten Public Offering and Concurrent Private Placement
Details : The net proceeds from the offering will primarily be used to fund the advancement of current or future product candidates, including AX-0810 for the treatment of primary sclerosing cholangitis.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Undisclosed
October 25, 2024
Lead Product(s) : AX-0810
Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)
Highest Development Status : Preclinical
Sponsor : Evercore ISI
Deal Size : $63.0 million
Deal Type : Public Offering

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Lead Product(s) : AX-0810
Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)
Study Phase : Preclinical
Sponsor : Evercore ISI
Deal Size : Undisclosed
Deal Type : Public Offering
ProQR Announces Proposed Underwritten Public Offering of Ordinary Shares
Details : The net proceeds from the offering will primarily be used to fund the advancement of current or future product candidates, including AX-0810 for the treatment of primary sclerosing cholangitis.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Undisclosed
October 22, 2024
Lead Product(s) : AX-0810
Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)
Highest Development Status : Preclinical
Sponsor : Evercore ISI
Deal Size : Undisclosed
Deal Type : Public Offering

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Lead Product(s) : AX-0810
Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)
Study Phase : Preclinical
Sponsor : Evercore ISI
Deal Size : $75.0 million
Deal Type : Public Offering
ProQR Prices $75 Million Underwritten Public Offering and Concurrent Private Placement
Details : The net proceeds from the offering will primarily be used to fund the advancement of current or future product candidates, including AX-0810 for the treatment of primary sclerosing cholangitis.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Undisclosed
October 22, 2024
Lead Product(s) : AX-0810
Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)
Highest Development Status : Preclinical
Sponsor : Evercore ISI
Deal Size : $75.0 million
Deal Type : Public Offering

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Lead Product(s) : AX-0810
Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)
Study Phase : Preclinical
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
ProQR Announces Preclinical Proof Of Concept for AX-0810 RNA Editing Program
Details : AX-0810 is an editing oligonucleotide development program targeting SLC10A1 RNA, providing a controlled approach aimed at reducing bile acids concentration in the liver for cholestatic diseases.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
May 08, 2024
Lead Product(s) : AX-0810
Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)
Highest Development Status : Preclinical
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Discovery Platform
Sponsor : Rett Syndrome Research Trust
Deal Size : $1.0 million
Deal Type : Collaboration
ProQR and Rett Syndrome Research Trust Join Forces with Axiomer™ RNA Editing Collaboration
Details : The collaboration focuses on developing editing oligonucleotides (EONs) using ProQR’s Axiomer technology to target MECP2 and correct specific mutations.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Undisclosed
January 05, 2024
Lead Product(s) : Undisclosed
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Discovery Platform
Sponsor : Rett Syndrome Research Trust
Deal Size : $1.0 million
Deal Type : Collaboration

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Lead Product(s) : Sepofarsen
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase II/ Phase III
Sponsor : Laboratoires Thea
Deal Size : $186.3 million
Deal Type : Divestment
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.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : $8.6 million
December 08, 2023
Lead Product(s) : Sepofarsen
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Sponsor : Laboratoires Thea
Deal Size : $186.3 million
Deal Type : Divestment

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Lead Product(s) : Sepofarsen
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase II/ Phase III
Sponsor : Laboratoires Thea
Deal Size : $162.0 million
Deal Type : Divestment
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.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : $13.7 million
August 01, 2023
Lead Product(s) : Sepofarsen
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase II/ Phase III
Sponsor : Laboratoires Thea
Deal Size : $162.0 million
Deal Type : Divestment

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Lead Product(s) : Undisclosed
Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)
Study Phase : Discovery Platform
Sponsor : Eli Lilly
Deal Size : $3,750.0 million
Deal Type : Expanded Collaboration
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.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : $75.0 million
December 22, 2022
Lead Product(s) : Undisclosed
Therapeutic Area : Hepatology (Liver, Pancreatic, Gall Bladder)
Highest Development Status : Discovery Platform
Sponsor : Eli Lilly
Deal Size : $3,750.0 million
Deal Type : Expanded Collaboration
