ADAR

Korro Reports First Quarter 2024 Financial Results and Appointment of Kemi Olugemo, M.D. as Chief Medical Officer

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Mardi, mai 14, 2024
Atlas Venture, ADAR, University, Protein, Clinical trial, Drug development, Administration, Health, Maryland School, PIPE, Adenosine deaminase, AAT, NEA, RNA, Research, Research and development, Atlas, G&A, Ultragenyx, Pharmaceutical industry

CAMBRIDGE, Mass., May 14, 2024 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, today reported financial results for the first quarter of 2024 and provided an update on its recent progress and anticipated milestones.

Key Points: 
  • Today, we are excited to announce the addition of Dr. Kemi Olugemo as Korro’s Chief Medical Officer.
  • In April 2024, Korro announced a PIPE of approximately $70.0 million with participation from new and existing investors.
  • First Quarter 2024 Financial Results:
    Cash Position: Cash and cash equivalents were $138.8 million as of March 31, 2024, compared to $166.1 million as of December 31, 2023.
  • Net Loss: Korro’s net loss was $19.6 million for each of the three months ended March 31, 2024, and 2023.

Radar Therapeutics Raises $13.4M in Seed Funding to Develop Smart Programmable Medicines Using Molecular RNA Sensors

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Jeudi, mai 23, 2024
Hardware, FDA, Data Management, Other Health, Technology, Pharmaceutical, Oncology, Genetics, Clinical Trials, Science, Biotechnology, Software, Other Science, Health, Research, Radar, Chemical engineering, Doctor of Philosophy, Biology, Data, Eli Lilly and Company, ADAR, MicroRNA, RNA, Stanford University, Messenger, Therapy, Safety, MIT, Vaccine, Nature Biotechnology, Chemical substance, Gene, Entrepreneurship, CSO, Medicine, Associate, RNA editing, Multimedia, RADAR, Enzyme

Radar Therapeutics , a biotech company developing smart programmable medicines, today announced the completion of an oversubscribed $13.4 million in seed financing led by NfX Bio.

Key Points: 
  • Radar Therapeutics , a biotech company developing smart programmable medicines, today announced the completion of an oversubscribed $13.4 million in seed financing led by NfX Bio.
  • View the full release here: https://www.businesswire.com/news/home/20240523395742/en/
    Radar Therapeutics founders Sophia Lugo, CEO and Eerik Kaseniit, PhD, CSO & President.
  • Photo credit: Radar Therapeutics
    Current genetic medicines, including mRNA therapeutics, are not targeted and typically rely on cell surface proteins to confer targeting, which limits application.
  • “Like a safety switch, our payload is always off, and only gets turned on in the right cell,” said Sophia Lugo, CEO & Co-Founder, Radar Therapeutics.

ProQR Announces First Quarter 2024 Operating and Financial Results

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Jeudi, mai 9, 2024
EON, Trident, Annual general meeting, Research, Partnership, Doctor of Philosophy, Webcast, Biomarker, Knowledge, Cholestasis, NTCP, RNA editing, Oncology, European Patent Convention, Oligonucleotide, Serum, Lilly, Eli Lilly and Company, Ribose, Pseudouridine, Poster, Alnylam Pharmaceuticals, Eli Lilly, RNA, Exercise, NHP, Clinical trial, ProQR, ADAR, B4GALT1, Adenosine, EDT, Science, Patent, Patient, Liver, Eon, Metabolism, Uridine, Q&A, Pharmaceutical industry

(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 first quarter ended March 31, 2024, 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 first quarter ended March 31, 2024, and provided a business update.
  • At March 31, 2024, ProQR held cash and cash equivalents and short term financial assets of €102.7 million, compared to €118.9 million cash and cash equivalents at December 31, 2023.
  • General and administrative costs were €3.5 million for the quarter ended March 31, 2024 compared to €4.0 million for the same period last year.
  • For further financial information for the period ended March 31, 2024, please refer to the Q1 financial report filing.

Shape Therapeutics RNAfix® Editing Platform Achieves >90% Editing in Mouse and Non-Human Primate Brain

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Mercredi, mai 8, 2024
Brain, Transcriptome, AI, Rett syndrome, RNA, MD, Doctor of Philosophy, Cell, AAV, CNS, DNA, Patient, Gene, Society, Central nervous system, NHP, Annual general meeting, ADAR, Vaccine

SEATTLE, May 08, 2024 (GLOBE NEWSWIRE) -- Shape Therapeutics Inc. (ShapeTX), a leader at the convergence of AI and RNA editing to revolutionize genomic medicine, today presented data at the 27th American Society for Gene and Cell Therapy (ASGCT) Annual Meeting in Baltimore, MD showcasing the high therapeutic potential of its RNAfix platform in for the treatment of central nervous system (CNS) disorders.

Key Points: 
  • Potential therapeutic applications of RNAfix will be highlighted in Rett syndrome, Parkinson’s disease, and Alzheimer’s disease, showcasing a diversity of therapeutic mechanisms enabled by nucleotide-level precision RNA editing.
  • A superior safety profile will also be presented, with no significant off-target editing across the entire transcriptome.
  • The data presented at ASGCT highlight levels of editing in the CNS unparalleled by any other editing technology, unlocking vast therapeutic opportunities.
  • Importantly, the RNAfix platform leaves genomic DNA intact and does not require the delivery of foreign proteins with immunogenicity risks.

ProQR Achieves Successful Defense of New Challenge to its Axiomer™ IP Portfolio

Retrieved on: 
Vendredi, avril 19, 2024
Adenosine, IP, General counsel, European Patent Convention, RNA, AON, Oligonucleotide, Deamination, Science, Patent, Ribose, Therapy, ProQR, ADAR, EPO, Vaccine

This patent is part of ProQR’s intellectual property estate surrounding its Axiomer RNA editing platform and the current generation of its molecules.

Key Points: 
  • This patent is part of ProQR’s intellectual property estate surrounding its Axiomer RNA editing platform and the current generation of its molecules.
  • Since then, ProQR has filed multiple additional patent applications on further improvements to form a leading patent estate that supports ProQR’s ADAR-mediated RNA editing platform Axiomer.
  • Today ProQR has extensive patent protection related to the Axiomer RNA editing platform, including 15 published patent families, that currently comprise a total of 28 patents.
  • Beyond this, ProQR has several unpublished patent applications and continuously invests in expanding its IP estate around ADAR-mediated RNA editing.

Korro Reports Full Year 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Mardi, mars 26, 2024
Research, Piz, Adenosine deaminase, Half-life, Patient, Alnylam Pharmaceuticals, AAT, ADAR, Mouse, RNA, Chord, N-Acetylgalactosamine, Hepatocyte, Investment, Customization, Protein, Therapy, Research and development, Administration, Liver, Vaccine

KRRO-110 is the first RNA editing oligonucleotide product candidate from Korro’s platform called Oligonucleotide Promoted Editing of RNA (OPERA™).

Key Points: 
  • KRRO-110 is the first RNA editing oligonucleotide product candidate from Korro’s platform called Oligonucleotide Promoted Editing of RNA (OPERA™).
  • Using CHORDs, Korro achieved greater than 50% editing using GalNAc conjugates with subcutaneous administration to the liver in a preclinical mouse model.
  • Full Year 2023 Financial Results:
    Cash Position: Cash, cash equivalents and short-term investments were $166.1 million as of December 31, 2023, compared to $55.2 million as of December 31, 2022.
  • Net Loss: Korro’s net loss was $81.2 million for the year ended December 31, 2023, as compared to $58.0 million for the year ended December 31, 2022.

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

Retrieved on: 
Jeudi, février 15, 2024
Enzyme, ProQR, Patent, ADAR, General counsel, RNA, Therapy, Oligonucleotide, IP

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: 
Vendredi, janvier 19, 2024
EON, RNA-Seq, Oligonucleotide, Knowledge, RNA, Science, Circulatory system, Mouse, NHP, NTCP, ACTB, Cholestasis, ANGPTL3, Cardiovascular disease, Plasma, Conference, B4GALT1, LDL, Liver, Eon, ProQR, ADAR, Nested polymerase chain reaction, CNS, Video game

“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

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Jeudi, janvier 18, 2024
AATD, FTD, Chord, Patient, Chronic obstructive pulmonary disease, Cirrhosis, Fibrosis, Oligonucleotide, Emphysema, RNA, Mouse, RNA editing, AAT, COPD, Dicarboxylic acid, De novo, Conference, Central nervous system, Calcium channel, Piz, Protein, Inflammation, ADAR, Adenosine deaminase, Machine learning, Customization, Genetic disorder, ALS, Liver disease, Nav1.7, Vaccine

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: 
Jeudi, décembre 7, 2023
LNP, RNA, Liver, ADAR, Pizzicato, Disease, SNV, Mouse, Doctor of Philosophy, Human, A1AT, Mutation, AATD, Patient, Piz, Vaccine

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.