ADAR

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  • Beyond this, ProQR has several unpublished patent applications and continuously invests in expanding its IP estate around ADAR-mediated RNA editing.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.

Korro Bio Highlights Data for its Lead Program in Alpha-1 Antitrypsin Deficiency (AATD) and Progress Across its RNA Editing Portfolio

Retrieved on: 
Thursday, January 18, 2024

AATD can lead to severe progressive lung disease, including emphysema and chronic obstructive pulmonary disease (COPD), and severe liver disease leading to inflammation, cirrhosis, and fibrosis.

Key Points: 
  • AATD can lead to severe progressive lung disease, including emphysema and chronic obstructive pulmonary disease (COPD), and severe liver disease leading to inflammation, cirrhosis, and fibrosis.
  • Korro’s proprietary RNA editing platform, OPERA™, integrates a deep understanding of adenosine deaminase acting on RNA (ADAR) enzymology with expertise in oligonucleotide chemistry, machine learning optimization of oligonucleotides and fit-for-purpose delivery.
  • CHORDs™, or Customized High-fidelity Oligonucleotides for RNA Deamination, are single-stranded, anti-sense oligonucleotides designed to have high target efficiency and specificity by leveraging the pillars of OPERA.
  • The replay is available for 30 days following the January 9, 2024 conclusion of the presentation.

Korro Announces Selection of its First Development Candidate for the Potential Treatment of Alpha-1 Antitrypsin Deficiency (AATD)

Retrieved on: 
Thursday, December 7, 2023

KRRO-110 is a proprietary RNA editing oligonucleotide delivered to liver cells using clinically validated LNP technology licensed from Genevant.

Key Points: 
  • KRRO-110 is a proprietary RNA editing oligonucleotide delivered to liver cells using clinically validated LNP technology licensed from Genevant.
  • Preclinical development of KRRO-110 is ongoing in preparation for a potential regulatory filing in the second half of 2024.
  • “Selecting a development candidate for our AATD program is an important milestone for Korro, but more importantly, for patients.
  • “Korro’s RNA editing approach has the potential to be transformative for AATD patients and Korro is well-positioned to deliver a best-in-class therapeutic.

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.

Korro Bio and Frequency Therapeutics Announce Closing of Merger and Private Placement of $117 Million

Retrieved on: 
Friday, November 3, 2023

J.P. Morgan Securities LLC served as exclusive financial advisor to Korro Bio and lead placement agent on Korro Bio’s private placement.

Key Points: 
  • J.P. Morgan Securities LLC served as exclusive financial advisor to Korro Bio and lead placement agent on Korro Bio’s private placement.
  • BofA Securities, Piper Sandler and RBC Capital Markets also served as placement agents for Korro Bio’s private placement.
  • Goodwin Procter LLP served as legal counsel to Korro Bio and Davis Polk & Wardwell LLP served as the placement agents’ legal counsel.
  • TD Cowen served as exclusive financial advisor to Frequency Therapeutics and Latham & Watkins LLP served as Frequency’s legal counsel.

Locanabio Announces Presentation of Preclinical Data Demonstrating an Improved Vectorized snRNA Platform with Applications in Exon Skipping for the Potential Treatment of Duchenne Muscular Dystrophy (DMD) at the 28th Annual Congress of the World Muscle So

Retrieved on: 
Friday, October 6, 2023

“As these data demonstrate, our vectorized snRNA platform combines the therapeutic potential of exon skipping with the delivery advantages of an AAV gene therapy,” said John Leonard, Ph.D., Locanabio’s chief scientific officer.

Key Points: 
  • “As these data demonstrate, our vectorized snRNA platform combines the therapeutic potential of exon skipping with the delivery advantages of an AAV gene therapy,” said John Leonard, Ph.D., Locanabio’s chief scientific officer.
  • In contrast, exon skipping enables the production of a near full-length dystrophin from the native transcript expressed in affected tissues.
  • However, prior exon skipping approaches with first generation antisense oligonucleotides (ASOs) have been limited by poor biodistribution and tolerability resulting in negligible restoration of dystrophin protein.
  • “The ability to package multiple snRNA cassettes amplifies potency relative to delivery of single ASOs for exon skipping.

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.

Wave Life Sciences to Present at the Chardan 7th Annual Genetic Medicines Conference

Retrieved on: 
Monday, September 25, 2023

CAMBRIDGE, Mass., Sept. 25, 2023 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced that Paul Bolno, MD, MBA, President and Chief Executive Officer, is scheduled to present at the Chardan 7th Annual Genetic Medicines Conference in New York City on Monday, October 2, 2023.

Key Points: 
  • CAMBRIDGE, Mass., Sept. 25, 2023 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage RNA medicines company committed to delivering life-changing treatments for people battling devastating diseases, today announced that Paul Bolno, MD, MBA, President and Chief Executive Officer, is scheduled to present at the Chardan 7th Annual Genetic Medicines Conference in New York City on Monday, October 2, 2023.
  • Dr. Bolno will participate in the ADAR Editing Panel at 9:30 a.m.
  • A live webcast of the fireside chat will be available on the Investor Relations page of the Wave Life Sciences website at http://ir.wavelifesciences.com .
  • A replay of the ADAR Editing Panel will be available after the conclusion of the conference.