ACTB

ProQR Announces Year End 2023 Operating and Financial Results

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Wednesday, March 13, 2024
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(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 year ended December 31, 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 year ended December 31, 2023, and provided a business update.
  • At December 31, 2023, ProQR held cash and cash equivalents of €118.9 million, compared to €94.8 million at December 31, 2022.
  • The Company experienced a net positive cash flow from operating activities in 2023 primarily due to the receipt of the Lilly up-front payment of $60 million in February 2023.
  • For further financial information for the period ended December 31, 2023, please refer to our 2023 Annual Report on Form 20-F and our Statutory Annual Report which will be available on our website, www.

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

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Friday, January 19, 2024
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“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.

Wave Life Sciences Announces Nature Biotechnology Publication Highlighting First RNA Base Editing in Non-Human Primates Using an Endogenous Enzyme

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Monday, March 7, 2022
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CAMBRIDGE, Mass., March 07, 2022 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced the publication of preclinical proof-of-concept data for the company’s novel ADAR-mediated RNA base editing modality in the journal Nature Biotechnology. Data reported include an in vivo study where Wave’s GalNAc-conjugated A-to-I(G) RNA base editing oligonucleotides (“AIMers”) yielded up to 50% editing of ACTB (Beta-actin) transcript in the liver of non-human primates (NHPs), with editing levels persisting as high as 40% for more than one month. This is the first scientific publication to report that RNA base editing in NHPs can be achieved with a simplified oligonucleotide approach. The paper, titled “Endogenous ADAR-mediated RNA editing in non-human primates using stereopure chemically modified oligonucleotides,” is available here.

Key Points: 
  • This is the first scientific publication to report that RNA base editing in NHPs can be achieved with a simplified oligonucleotide approach.
  • The paper, titled Endogenous ADAR-mediated RNA editing in non-human primates using stereopure chemically modified oligonucleotides, is available here .
  • We are proud to be contributing these important learnings to the scientific community through this seminal publication in Nature Biotechnology.
  • Waves A-to-I RNA base editing oligonucleotides (AIMers) are designed to recruit these ADAR enzymes to direct efficient and highly specific editing of RNA transcripts.

Wave Life Sciences Reports Third Quarter 2021 Financial Results and Provides Business Update

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Wednesday, November 10, 2021
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CAMBRIDGE, Mass., Nov. 10, 2021 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced financial results for the third quarter ended September 30, 2021 and provided a business update.

Key Points: 
  • ET today
    CAMBRIDGE, Mass., Nov. 10, 2021 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced financial results for the third quarter ended September 30, 2021 and provided a business update.
  • ET to discuss the companys third quarter and 2021 financial results and provide a business update.
  • The live webcast may be accessed from the Investor Relations section of the Wave Life Sciences corporate website at ir.wavelifesciences.com .
  • Wave Life Sciences (Nasdaq: WVE) is a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases.

Wave Life Sciences Announces New Data for Leading RNA Editing Capability Across Multiple Tissues and Provides Update on AATD Program During Analyst and Investor Research Webcast

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Tuesday, September 28, 2021
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CAMBRIDGE, Mass., Sept. 28, 2021 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage genetic medicines company committed to delivering life-changing treatments for people battling devastating diseases, today presented new data for its ADAR-mediated RNA editing capability (ADAR editing), including new preclinical editing data across multiple tissues, as well as an update on its discovery-stage alpha-1 antitrypsin deficiency (AATD) program during the company’s 2021 Analyst and Investor Research Webcast. The webcast also included updates on the company’s PRISM™ platform and initial results from the application of ADAR editing to neurology targets.

Key Points: 
  • The webcast also included updates on the companys PRISMplatform and initial results from the application of ADAR editing to neurology targets.
  • A replay of the Analyst and Investor Research Webcast is available on Waves Investor Relations website.
  • Additionally, in vitro data were presented demonstrating the potential to target protein-protein interactions and upregulate downstream gene expression with AIMers.
  • Wave undertakes no obligation to update the information contained in this press release to reflect subsequently occurring events or circumstances.