Adenosine deaminase

Korro Reports Full Year 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Tuesday, March 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.

Key Proteo Announces Clinical Laboratory Expansion Plans Including New CLIA-Certified Laboratory

Retrieved on: 
Thursday, March 7, 2024
Wilson's disease, Growth, Wiskott–Aldrich syndrome, Adenosine deaminase, Classification, DSM-IV codes, Research, IVD, Proteomics, Pharmaceutical industry

SEATTLE, March 7, 2024 /PRNewswire/ -- Key Proteo, a pioneering proteomics diagnostics company specializing in the early identification of rare but treatable genetic disorders, announced it has initiated plans to build a new CLIA-certified laboratory as part of its initial commercialization and growth strategy to enhance newborn screening programs. The new larger facility and expanded scope of operations will allow Key Proteo to serve unaddressed newborn screening needs among public health laboratories, reference laboratories, hospital laboratories and other providers associated with neonatal care, while maintaining dedicated lab operations for ongoing clinical research and other collaborative opportunities.

Key Points: 
  • The new larger facility and expanded scope of operations will allow Key Proteo to serve unaddressed newborn screening needs among public health laboratories, reference laboratories, hospital laboratories and other providers associated with neonatal care, while maintaining dedicated lab operations for ongoing clinical research and other collaborative opportunities.
  • Based in Seattle, the new 5,000-square-foot facility will include the addition of a state-of-the art CLIA-certified clinical laboratory alongside Key Proteo's corporate headquarters and dedicated clinical research facilities.
  • Buildout of the lab has commenced, and, upon launch, Key Proteo's first newborn screening panel will target four treatable genetic orders including Wilson's disease, Wiskott-Aldrich syndrome, X-linked a-y-globulinemia and adenosine deaminase (ADA) deficiency.
  • In parallel path, Key Proteo is pursuing FDA De Novo classification of an in vitro diagnostic (IVD) test kit for the same four disorders.

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, 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.

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

Retrieved on: 
Monday, November 6, 2023
Adenosine deaminase, ADAR, Patent, RNA, Adenosine, Intellectual property, Oligonucleotide, ProQR, RNA editing, European Patent Office, United States Patent and Trademark Office, Inosine, Therapy, European Patent Convention, IP, General counsel, Japan Patent Office, 3D printing, Vaccine

“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.

FONDAZIONE TELETHON AND ORCHARD THERAPEUTICS COMPLETE TRANSFER OF MARKETING AUTHORIZATION OF STRIMVELIS FOR ADA-SCID IN EUROPE

Retrieved on: 
Tuesday, September 12, 2023
Gene, Civil service commission, Marketing, SAN, European Medicines Agency, Research, EMA, Patient, San Raffaele Hospital, ADA-SCID, Therapy, European Commission, Adenosine deaminase, Vaccine, Medical device

MILAN, BOSTON, and LONDON, Sept. 12, 2023 /PRNewswire/ -- Fondazione Telethon, one of the main Italian biomedical charities, and Orchard Therapeutics, a global gene therapy leader, today announced the completion of the transfer of the marketing authorization for Strimvelis, a gene therapy approved by the European Medicines Agency in 2016 for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).

Key Points: 
  • The marketing authorization transfer was approved on July 17th by the European Commission[1] following a positive opinion from the European Medicines Agency (EMA).
  • The European manufacturing and distribution rights have been fully transferred to Fondazione Telethon from its former holder, Orchard Therapeutics, which previously announced it would discontinue investment in and seek strategic alternatives for its programs in rare primary immune deficiencies, including Strimvelis.
  • A total of 45 patients from over 20 countries worldwide have been treated with Strimvelis in clinical trials and commercially, to date.
  • Fondazione Telethon will continue to make Strimvelis available to eligible patients through the San Raffaele Hospital in Milan, Italy.

FONDAZIONE TELETHON AND ORCHARD THERAPEUTICS COMPLETE TRANSFER OF MARKETING AUTHORIZATION OF STRIMVELIS FOR ADA-SCID IN EUROPE

Retrieved on: 
Tuesday, September 12, 2023
Gene, Civil service commission, Marketing, SAN, European Medicines Agency, Research, EMA, Patient, San Raffaele Hospital, ADA-SCID, Therapy, European Commission, Adenosine deaminase, Vaccine, Medical device

MILAN, BOSTON, and LONDON, Sept. 12, 2023 /PRNewswire/ -- Fondazione Telethon, one of the main Italian biomedical charities, and Orchard Therapeutics, a global gene therapy leader, today announced the completion of the transfer of the marketing authorization for Strimvelis, a gene therapy approved by the European Medicines Agency in 2016 for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).

Key Points: 
  • The marketing authorization transfer was approved on July 17th by the European Commission[1] following a positive opinion from the European Medicines Agency (EMA).
  • The European manufacturing and distribution rights have been fully transferred to Fondazione Telethon from its former holder, Orchard Therapeutics, which previously announced it would discontinue investment in and seek strategic alternatives for its programs in rare primary immune deficiencies, including Strimvelis.
  • A total of 45 patients from over 20 countries worldwide have been treated with Strimvelis in clinical trials and commercially, to date.
  • Fondazione Telethon will continue to make Strimvelis available to eligible patients through the San Raffaele Hospital in Milan, Italy.

HuidaGene Therapeutics Announces Publication of the World's First Guanine Base-Editor

Retrieved on: 
Thursday, May 18, 2023
SAB, CBQT-FM, DNA, Guanosine, Guanine, Gene editing, C. J. T. Thamotheram, Deamination, Nucleoside, National Science Review, Patent, Thymidine, CBE, Nature Biotechnology, Entrepreneurship, Cytidine deaminase, Therapy, Adenosine deaminase, Publishing, Vaccine

SHANGHAI and CLINTON, N.J., May 18, 2023 /PRNewswire/ -- HuidaGene Therapeutics (辉大基因; HuidaGene), a clinical-stage genome-editing company, today announced the online National Science Review published the comprehensive data from a study of the world's first DNA base editor converting G-to-Y, glycosylase-based guanine base editor (gGBE).

Key Points: 
  • SHANGHAI and CLINTON, N.J., May 18, 2023 /PRNewswire/ -- HuidaGene Therapeutics (辉大基因; HuidaGene), a clinical-stage genome-editing company, today announced the online National Science Review published the comprehensive data from a study of the world's first DNA base editor converting G-to-Y, glycosylase-based guanine base editor (gGBE).
  • Company has filed an international patent application for this base editor and owns the exclusive global rights to the underlying patent.
  • Current widely-used DNA base editors mainly integrate programmable DNA binding proteins (Cas9, Cas12, or TALE protein variants) with base deaminases (cytidine deaminase or adenosine deaminase variants).
  • The new class of engineered glycosylases base editors may further enrich the base editing tool, establish disease models, and develop gene editing therapies."

HuidaGene Therapeutics Announces Publication of the World's First Guanine Base-Editor

Retrieved on: 
Thursday, May 18, 2023
SAB, CBQT-FM, DNA, Guanosine, Guanine, Gene editing, C. J. T. Thamotheram, Deamination, Nucleoside, National Science Review, Patent, Thymidine, CBE, Nature Biotechnology, Entrepreneurship, Cytidine deaminase, Therapy, Adenosine deaminase, Publishing, Vaccine

SHANGHAI and CLINTON, N.J., May 18, 2023 /PRNewswire/ -- HuidaGene Therapeutics (辉大基因; HuidaGene), a clinical-stage genome-editing company, today announced the online National Science Review published the comprehensive data from a study of the world's first DNA base editor converting G-to-Y, glycosylase-based guanine base editor (gGBE).

Key Points: 
  • SHANGHAI and CLINTON, N.J., May 18, 2023 /PRNewswire/ -- HuidaGene Therapeutics (辉大基因; HuidaGene), a clinical-stage genome-editing company, today announced the online National Science Review published the comprehensive data from a study of the world's first DNA base editor converting G-to-Y, glycosylase-based guanine base editor (gGBE).
  • Company has filed an international patent application for this base editor and owns the exclusive global rights to the underlying patent.
  • Current widely-used DNA base editors mainly integrate programmable DNA binding proteins (Cas9, Cas12, or TALE protein variants) with base deaminases (cytidine deaminase or adenosine deaminase variants).
  • The new class of engineered glycosylases base editors may further enrich the base editing tool, establish disease models, and develop gene editing therapies."

ProQR to Present at Upcoming Scientific and Industry Conferences

Retrieved on: 
Tuesday, March 7, 2023
Performance, Discovery, RNA editing, ADAR, Gene, Therapy, GRC, Safety, RNA, Knowledge, Conference, Congress, ProQR, Eon, Oligonucleotide, Disease, OPT, Adenosine deaminase, Delivery, Fine chemical, Vaccine, Equivalents of the Axiom of Choice

Session: Oligonucleotide Discovery and Delivery - Optimizing Design, Delivery and Performance

Key Points: 
  • Session: Oligonucleotide Discovery and Delivery - Optimizing Design, Delivery and Performance
    Abstract: Axiomer, an RNA editing technology, uses Editing Oligonucleotides (EONs) to target specific RNA for A-to-I editing by recruiting endogenous Adenosine Deaminase Acting on RNA (ADAR).
  • This presentation will highlight the therapeutic possibilities of this platform that are not limited to disease causing mutations and can potentially address high unmet medical needs by editing wild-type RNA to engineer proteins or modify their function as well as creating de novo mutations.
  • Abstract: Axiomer, an RNA editing platform, uses chemically modified and complementary single stranded editing oligonucleotides (EONs) to target specific RNA for A-to-I editing by recruiting endogenous Adenosine Deaminase Acting on RNA (ADAR).
  • Through now further developed design principles there has been an established improvement of specificity and efficacity of EONs, and we have tested the RNA editing efficacy and short-term safety of EONs acting on various genes and models.

ProQR to Present its Axiomer® RNA Editing Technology at the Oligonucleotide and Peptide Therapeutics Conference (TIDES Europe) 2022

Retrieved on: 
Monday, November 14, 2022
Mutation, Therapy, ADAR, Adenosine deaminase, Patient, Oligonucleotide, Annual report, Security (finance), Disease, RNA, Eon, Vaccine, Adenosine, Guanosine, Inosine

ProQR is pioneering a next-generation RNA base editing technology called Axiomer, which could potentially yield a new class of medicines for diverse types of diseases.

Key Points: 
  • ProQR is pioneering a next-generation RNA base editing technology called Axiomer, which could potentially yield a new class of medicines for diverse types of diseases.
  • Axiomer Editing Oligonucleotides, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA).
  • ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies.
  • Such forward-looking statements include, but are not limited to, statements regarding our participation in this conference and the potential of our technologies and platforms (including Axiomer).