Oligonucleotide

ProQR Announces First Quarter 2024 Operating and Financial Results

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星期四, 五月 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.

ProQR Announces Preclinical Proof of Concept Data for AX-0810 Axiomer™ RNA Editing Program Targeting NTCP for Cholestatic Diseases

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星期三, 五月 8, 2024
EON, NTCP, EDT, Portal vein, RNA, Cell, Webcast, Cholestasis, SLC10A1, Oligonucleotide, Liver, Gene, Eli Lilly, Eon, Society, Serum, Metabolism, Cardiovascular disease, Biomarker, NHP, Clinical trial, Annual general meeting, ProQR, B4GALT1, Q&A, Vaccine

LEIDEN, Netherlands & CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced new preclinical data for its proprietary Axiomer™ RNA editing technology platform, including the first preclinical proof of concept data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP.

Key Points: 
  • LEIDEN, Netherlands & CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced new preclinical data for its proprietary Axiomer™ RNA editing technology platform, including the first preclinical proof of concept data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP.
  • “In our AX-0810 program targeting NTCP for cholestatic diseases, we have demonstrated preclinical proof of concept showing that Axiomer RNA editing oligonucleotides can have a meaningful effect on levels of serum biomarker bile acid.
  • ProQR is developing its AX-0810 program targeting NTCP for Cholestatic Diseases and plans to advance the program to the clinic in late 2024/early 2025.
  • AX-1412 preclinical proof of concept data and translational data to be reported in H2 2024, with program to advance to the clinic in late 2024/early 2025.

Alltrna Applies Machine Learning to Engineer tRNA Oligonucleotides with Significantly Improved Activity and Demonstrates In Vivo Readthrough of the Two Most Common Premature Termination Codons in Genetic Disease

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星期二, 五月 7, 2024
Small RNA, Mutation, Flagship Pioneering, LNP, Machine learning, RNA, Cell, Molecular biology, Computation, Disease, Oligonucleotide, Therapy, Gene, Society, Biology, Annual general meeting, PTC

"The data demonstrate the power of Alltrna's platform to identify key combinations of tRNA sequences and modifications and precisely design tRNA oligonucleotides with significantly improved in vivo activity.

Key Points: 
  • "The data demonstrate the power of Alltrna's platform to identify key combinations of tRNA sequences and modifications and precisely design tRNA oligonucleotides with significantly improved in vivo activity.
  • With optimized engineered tRNAs for the two most prevalent premature termination codons, we are advancing preclinical studies for our first Stop Codon Disease indications."
  • Delivered using a liver-directed lipid nanoparticle (LNP), both optimized tRNAs showed robust in vivo activity in two transgenic mouse models.
  • "We've also demonstrated that we can engineer robust tRNA activity for two different premature termination codons, each of which are highly prevalent in Stop Codon Disease."

Molecular Assemblies Launches Partner Program for Onsite Synthesis of Long, Pure, Accurate DNA

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星期二, 五月 7, 2024
Doctor of Philosophy, Enzyme, Gene editing, Partnership, Research, MAI, UC, NGS, Protein engineering, Oligonucleotide, DNA, Therapy, Booth, Synthetic biology, University of California, Berkeley, Biotechnology, SAN, Fine chemical

SAN DIEGO, May 7, 2024 /PRNewswire/ -- Molecular Assemblies, Inc., ("MAI" or the "Company") a pioneer and leader in the field of enzymatic DNA synthesis, today announced the launch of its Partnering Program to license Molecular Assemblies' Fully Enzymatic SynthesisTM (FESTM) technology for onsite synthesis. FES technology accelerates the production of long, pure, and accurate DNA to power a new rapidly emerging generation of therapeutics and diagnostics.

Key Points: 
  • SAN DIEGO, May 7, 2024 /PRNewswire/ -- Molecular Assemblies, Inc. , ("MAI" or the "Company") a pioneer and leader in the field of enzymatic DNA synthesis, today announced the launch of its Partnering Program to license Molecular Assemblies' Fully Enzymatic SynthesisTM (FESTM) technology for onsite synthesis.
  • FES technology accelerates the production of long, pure, and accurate DNA to power a new rapidly emerging generation of therapeutics and diagnostics.
  • Molecular Assemblies also announced today that the company has opened early access to order oligonucleotides up to 400mer in length, limited to the first 20 customers.
  • Molecular Assemblies' Fully Enzymatic SynthesisTM (FESTM) is the foundation of their approach to create long, high quality, sequence specific DNA composed of more than 300 base pairs.

Wave Life Sciences Announces Continued Momentum in GSK Collaboration and Advancements in siRNA and RNA Editing

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星期二, 四月 23, 2024
Sequence, Small RNA, RNA editing, Potency, Durability, Health, ...Continued, RNA, MD, Doctor of Philosophy, Weight loss, IND, CNS, Obesity, Oligonucleotide, GSK, Patient, Medicinal chemistry, MBA, Liu Wei, Clinical trial, Pharmaceutical industry

CAMBRIDGE, Mass., April 23, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today provided an update on its best-in-class small interfering RNA (siRNA) and RNA editing platform capabilities.

Key Points: 
  • As part of Wave’s ongoing collaboration with GSK, GSK has selected its first two programs to advance to development candidates following achievement of target validation.
  • GSK will provide an aggregate initiation payment of $12 million to Wave for these two oligonucleotide programs.
  • Under the agreement, GSK can advance up to eight programs leveraging Wave’s PRISM™ platform and multiple RNA-targeting modalities (RNA editing, splicing, siRNA, and antisense) with target validation work ongoing across multiple therapy areas.
  • Beyond these programs, our collaboration is focusing on all Wave modalities, including RNA editing.

ProQR Highlights Upcoming Presentations on Axiomer™ RNA Editing at ASGCT 27th Annual Meeting

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星期一, 四月 22, 2024
EON, NTCP, RNA, Cell, Cholestasis, Oligonucleotide, ACTB, Gene, Eli Lilly, Society, Metabolism, Biomarker, NHP, Annual general meeting, ProQR, Vaccine

LEIDEN, Netherlands & CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it will present new preclinical data for its proprietary Axiomer™ RNA editing technology platform, including the first preclinical data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, May 7-11, 2024, in Baltimore, Maryland.

Key Points: 
  • LEIDEN, Netherlands & CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it will present new preclinical data for its proprietary Axiomer™ RNA editing technology platform, including the first preclinical data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, May 7-11, 2024, in Baltimore, Maryland.
  • “The in vitro and in vivo data that we will be presenting at ASGCT further build upon our experience with Axiomer and highlight the significant potential we see with the platform, including now preclinical proof of concept for cholestatic diseases targeting NTCP,” said Gerard Platenburg, Chief Scientific Officer and co-founder, ProQR.
  • “We continue to be impressed by the robust editing efficiency of Axiomer editing oligonucleotides and look forward to leveraging these data as we advance AX-0810 toward the clinic.”
    Building on its robust platform data, the Company will present additional non-human primate (NHP) data including with ACTB and NTCP and initial in vitro and in vivo preclinical proof of concept for its AX-0810 program targeting NTCP for cholestatic diseases, including hepatic editing in NHP with strong correlating impact on disease-relevant biomarkers.
  • P-726, partner poster presentation by Eli Lilly: “Complex Metabolism and Prolonged PK/PD of a GalNAc-Conjugated Editing Oligonucleotide (EON) in Mice”

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

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星期五, 四月 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.

New President Appointed for Nitto Denko Avecia Inc.

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星期五, 四月 26, 2024
Corporation, Business development, Female, Culture, Oligonucleotide, Patient, Marketing, Growth, Management

MILFORD, Mass., April 26, 2024 /PRNewswire/ -- Nitto Denko Avecia Inc. and Nitto Denko Avecia Pharma Services (Nitto Avecia), a global leader in contract development and manufacturing of oligonucleotides for the therapeutic market announced today that Tammy Cooper will be appointed President of Nitto Avecia effective June 21, 2024.

Key Points: 
  • MILFORD, Mass., April 26, 2024 /PRNewswire/ -- Nitto Denko Avecia Inc. and Nitto Denko Avecia Pharma Services (Nitto Avecia), a global leader in contract development and manufacturing of oligonucleotides for the therapeutic market announced today that Tammy Cooper will be appointed President of Nitto Avecia effective June 21, 2024.
  • As President, she will report to Yoshihiko Terada who will be appointed Executive Chairman of Nitto Avecia.
  • After June 21, 2024, Mr. Fujioka will continue as a Corporate Executive Vice President of Nitto Denko Corporation (Nitto Denko) leading Nitto Denko's South Asia Region including India and Oceania.
  • Mr. Terada has been with Nitto Denko for nine years and has served as Chief of Staff at Nitto Avecia and Corporate Vice President of Nitto Denko.

New President Appointed for Nitto Denko Avecia Inc.

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星期五, 四月 26, 2024
Corporation, Business development, Female, Culture, Oligonucleotide, Patient, Marketing, Growth, Management

MILFORD, Mass., April 26, 2024 /PRNewswire/ -- Nitto Denko Avecia Inc. and Nitto Denko Avecia Pharma Services (Nitto Avecia), a global leader in contract development and manufacturing of oligonucleotides for the therapeutic market announced today that Tammy Cooper will be appointed President of Nitto Avecia effective June 21, 2024.

Key Points: 
  • MILFORD, Mass., April 26, 2024 /PRNewswire/ -- Nitto Denko Avecia Inc. and Nitto Denko Avecia Pharma Services (Nitto Avecia), a global leader in contract development and manufacturing of oligonucleotides for the therapeutic market announced today that Tammy Cooper will be appointed President of Nitto Avecia effective June 21, 2024.
  • As President, she will report to Yoshihiko Terada who will be appointed Executive Chairman of Nitto Avecia.
  • After June 21, 2024, Mr. Fujioka will continue as a Corporate Executive Vice President of Nitto Denko Corporation (Nitto Denko) leading Nitto Denko's South Asia Region including India and Oceania.
  • Mr. Terada has been with Nitto Denko for nine years and has served as Chief of Staff at Nitto Avecia and Corporate Vice President of Nitto Denko.

Clearside Biomedical Appoints Victor Chong, M.D., MBA as Chief Medical Officer

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星期一, 三月 18, 2024
DAS, Retina, University, Moorfields Eye Hospital, Pathology, Oligonucleotide, Ranibizumab, Data analysis, Partnership, Business, Boehringer Ingelheim, DME, Patient, Pharming, Research, Royal College of Ophthalmologists, Cell biology, Diabetic retinopathy, C3, University College London, MBA, Eye, Johnson & Johnson, Janssen, Drug development, John Radcliffe Hospital, AMD, CLS, University of Oxford, Royal college, Geographic atrophy, Stargardt disease, DMI, Pharmaceutical industry

ALPHARETTA, Ga., March 18, 2024 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), announced today that Victor Chong, M.D., MBA was appointed Chief Medical Officer on March 14, 2024.

Key Points: 
  • ALPHARETTA, Ga., March 18, 2024 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), announced today that Victor Chong, M.D., MBA was appointed Chief Medical Officer on March 14, 2024.
  • George Lasezkay, Pharm.D., J.D., President and Chief Executive Officer of Clearside, commented, “Victor is a well-known and well-respected retinal clinician and scientist, who is a strategically focused and visionary leader.
  • We are delighted to have him join the Clearside team.
  • “With the upcoming ODYSSEY data and extensive partnership portfolio, this is an exciting time to join Clearside.