ProQR

ProQR Announces Japanese Axiomer™ Patent Upheld Following Opposition Against Its Leading IP Estate for ADAR-mediated RNA Editing

Retrieved on: 
Thursday, February 15, 2024

The Japanese Patent Office rejected the strawman’s opposition and indicated that all claims were to be maintained as granted to ProQR.

Key Points: 
  • The Japanese Patent Office rejected the strawman’s opposition and indicated that all claims were to be maintained as granted to ProQR.
  • Since then, ProQR has filed multiple additional patent applications on further improvements to form a leading patent estate that makes RNA editing with oligonucleotides that recruit endogenous ADAR proprietary to ProQR.
  • Today ProQR has extensive patent protection related to its RNA editing platform, Axiomer™, including more than 13 published patent families, that currently comprise a total of 27 patents.
  • Beyond this, ProQR has several unpublished patent applications and continuously invests in expanding its IP estate around ADAR-mediated RNA editing.

ProQR Highlights New Platform Data from Presentation on Axiomer™ RNA Editing Technology at Deaminet 2024

Retrieved on: 
Friday, January 19, 2024

“We were pleased to present new in vivo data for our proprietary Axiomer RNA editing technology platform at the Deaminet 2024 meeting, demonstrating robust editing of ACTB in the liver of NHP, as well as functional protein data with the liver target ANGPTL3 in mice,” said Gerard Platenburg, Chief Scientific Officer of ProQR.

Key Points: 
  • “We were pleased to present new in vivo data for our proprietary Axiomer RNA editing technology platform at the Deaminet 2024 meeting, demonstrating robust editing of ACTB in the liver of NHP, as well as functional protein data with the liver target ANGPTL3 in mice,” said Gerard Platenburg, Chief Scientific Officer of ProQR.
  • “To date, we have generated robust in vitro and in vivo preclinical platform data across a range of liver and CNS targets.
  • Presentation of new data highlighting up to 70% editing efficiency of ACTB in liver of mice and NHPs at multiple timepoints.
  • These new preclinical platform data further highlight the potential of Axiomer EONs in preparation for clinical development and supporting discovery of potential new therapeutic applications.

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

Retrieved on: 
Friday, January 5, 2024

“The Rett Syndrome Research Trust is the leading patient advocacy group championing a cure for Rett syndrome and we look forward to collaborating with them to further expand the broad applicability of our Axiomer RNA editing technology to Rett syndrome, a rare neurodevelopment disorder with significant unmet medical need,” said Daniel A. de Boer, Chief Executive Officer of ProQR.

Key Points: 
  • “The Rett Syndrome Research Trust is the leading patient advocacy group championing a cure for Rett syndrome and we look forward to collaborating with them to further expand the broad applicability of our Axiomer RNA editing technology to Rett syndrome, a rare neurodevelopment disorder with significant unmet medical need,” said Daniel A. de Boer, Chief Executive Officer of ProQR.
  • “Axiomer has the potential to restore precise levels of the MECP2 protein, which is lacking in Rett syndrome.
  • It is the intent of the partnership to be continued by an expanded co-funding arrangement following the initial discovery work.
  • The co-funding of the next phase of the collaboration would enable clinical development of an Axiomer-based therapeutic for Rett syndrome MECP2.

SpliceBio Appoints Leading Ophthalmology Expert Aniz Girach as Chief Medical Officer

Retrieved on: 
Wednesday, January 3, 2024

BARCELONA, Spain, Jan. 3, 2024 /PRNewswire/ -- SpliceBio, a genetic medicines company harnessing Protein Splicing to develop the next generation of gene therapies, is pleased to announce the appointment of Aniz Girach, MD as Chief Medical Officer.

Key Points: 
  • BARCELONA, Spain, Jan. 3, 2024 /PRNewswire/ -- SpliceBio, a genetic medicines company harnessing Protein Splicing to develop the next generation of gene therapies, is pleased to announce the appointment of Aniz Girach, MD as Chief Medical Officer.
  • Dr Girach was Global Head of Ophthalmology at Merck & Co and Vice President, Clinical Development at Alcon Laboratories.
  • Prior to joining SpliceBio, he led the development of genetic therapies for inherited retinal diseases at ProQR Therapeutics NV as its Chief Medical Officer.
  • Aniz Girach, MD., Chief Medical Officer of SpliceBio, commented: "Inherited retinal diseases are a heterogenous group of rare diseases that can lead to severe visual disability and blindness, affecting approximately 5.5 million people worldwide.

SpliceBio Appoints Leading Ophthalmology Expert Aniz Girach as Chief Medical Officer

Retrieved on: 
Wednesday, January 3, 2024

BARCELONA, Spain, Jan. 3, 2024 /PRNewswire/ -- SpliceBio, a genetic medicines company harnessing Protein Splicing to develop the next generation of gene therapies, is pleased to announce the appointment of Aniz Girach, MD as Chief Medical Officer.

Key Points: 
  • BARCELONA, Spain, Jan. 3, 2024 /PRNewswire/ -- SpliceBio, a genetic medicines company harnessing Protein Splicing to develop the next generation of gene therapies, is pleased to announce the appointment of Aniz Girach, MD as Chief Medical Officer.
  • Dr Girach was Global Head of Ophthalmology at Merck & Co and Vice President, Clinical Development at Alcon Laboratories.
  • Prior to joining SpliceBio, he led the development of genetic therapies for inherited retinal diseases at ProQR Therapeutics NV as its Chief Medical Officer.
  • Aniz Girach, MD., Chief Medical Officer of SpliceBio, commented: "Inherited retinal diseases are a heterogenous group of rare diseases that can lead to severe visual disability and blindness, affecting approximately 5.5 million people worldwide.

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

Retrieved on: 
Friday, December 8, 2023

LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 08, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it has completed a transaction divesting late stage ophthalmic assets, sepofarsen and ultevursen, to Laboratoires Théa (Théa).

Key Points: 
  • Agreement provides ProQR with initial payment of €8M and up to €165M in earn-out payments, as well as potential double-digit royalties based on commercial sales in the US and EU
    ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it has completed a transaction divesting late stage ophthalmic assets, sepofarsen and ultevursen, to Laboratoires Théa (Théa).
  • “Following previous announcements related to this transaction, we are pleased to share that we have now completed the divestment of the sepofarsen and ultevursen programs to Théa, who will continue the development of these potentially transformational therapies for patients with LCA10 and Usher Syndrome,” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR.
  • Any information on the next development steps for these programs will be available from Théa in due course.
  • ProQR’s financial advisor on this transaction was Lazard, with Allen & Overy acting as legal advisor.

ProQR Announces Third Quarter 2023 Operating and Financial Results

Retrieved on: 
Tuesday, November 7, 2023

(Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today reported its financial and operating results for the third quarter ended September 30, 2023, and provided a business update.

Key Points: 
  • (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today reported its financial and operating results for the third quarter ended September 30, 2023, and provided a business update.
  • On September 30, 2023, ProQR held cash and cash equivalents of €120.6 million, compared to €94.8 million on December 31, 2022.
  • General and administrative costs were €3.3 million for the quarter ended September 30, 2023 compared to €5.4 million for the quarter ended September 30, 2022.
  • For further financial information for the period ending September 30, 2023, please refer to the financial statements appearing at the end of this release.

ProQR Strengthens Leading Intellectual Property Estate for ADAR-mediated RNA Editing

Retrieved on: 
Monday, November 6, 2023

“The new patent that the USPTO granted to ProQR further expands the protection of RNA editing using oligonucleotides to recruit endogenous ADAR.

Key Points: 
  • “The new patent that the USPTO granted to ProQR further expands the protection of RNA editing using oligonucleotides to recruit endogenous ADAR.
  • Our leading intellectual property portfolio protects our Axiomer® ADAR-mediated RNA editing platform technology and more fundamentally the use of an oligonucleotide to recruit endogenous deaminating enzymes in the cell,” said René Beukema, Chief Corporate Development Officer and General Counsel.
  • Since then, ProQR has filed multiple additional patent applications on further improvements to form a leading patent estate that makes RNA editing with oligonucleotides that recruit endogenous ADAR proprietary to ProQR.
  • Beyond this, ProQR has several unpublished patent applications and continuously invests in expanding its IP estate around ADAR-mediated RNA editing.

ProQR Announces Participation in the Chardan 7th Annual Genetic Medicines Conference

Retrieved on: 
Thursday, September 28, 2023

LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 28, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV.

Key Points: 
  • LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 28, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV.
  • (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today announced that Daniel A. de Boer, Founder and CEO, will participate in the ADAR Editing Panel at the Chardan 7th Annual Genetic Medicines Conference in New York City.
  • The panel is scheduled for October 2, 2023 at 9:30 am – 10:15 am EDT, and a recording of the session will be made available on the Company’s website after the conference.
  • For more information about the conference, please contact your Chardan representative.

ProQR Therapeutics Provides Update on Ophthalmic Assets

Retrieved on: 
Wednesday, September 27, 2023

LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 27, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies today provided an update that its previously announced agreement to divest the Company’s late-stage ophthalmic assets to Laboratoires Théa S.A.S.

Key Points: 
  • LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 27, 2023 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies today provided an update that its previously announced agreement to divest the Company’s late-stage ophthalmic assets to Laboratoires Théa S.A.S.
  • (Théa) has been terminated.
  • The completion of the transaction was conditional on Théa entering into employment agreements with a number of key ophthalmology personnel from ProQR.
  • “While we are disappointed that the sale of these ophthalmology programs to Théa will not proceed, our primary focus at ProQR remains on our Axiomer® RNA editing platform,” said Daniel A. de Boer, Founder and Chief Executive Officer of ProQR.