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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 PlatformProduct Type: Large molecule
Partner/Sponsor/Collaborator: Rett Syndrome Research Trust
Deal Size: $1.0 million Upfront Cash: Undisclosed
Deal Type: Collaboration January 05, 2024
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 IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Laboratoires Thea
Deal Size: $186.3 million Upfront Cash: $8.6 million
Deal Type: Divestment December 08, 2023
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 IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Laboratoires Thea
Deal Size: $162.0 million Upfront Cash: $13.7 million
Deal Type: Divestment August 01, 2023
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 PlatformProduct 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
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 IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 11, 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: PreclinicalProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 09, 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 IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 26, 2022
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: PreclinicalProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 04, 2022
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 IIIProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 11, 2022
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: DiscoveryProduct 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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