Pseudouridine

ProQR Announces First Quarter 2024 Operating and Financial Results

Retrieved on: 
Thursday, May 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 Year End 2023 Operating and Financial Results

Retrieved on: 
Wednesday, March 13, 2024
Trident, Eli Lilly, Exercise, ProQR, Patient, IP, Therapy, Lilly, RNA editing, Cholestasis, Rett syndrome, Research, Cardiovascular disease, Partnership, Pseudouridine, NTCP, RNA, Functional, Uridine, MECP2, Oligonucleotide, ACTB, LPL, Annual report, Conference, Patent, Biomarker, Mouse, RSRT, Eli Lilly and Company, Liver, ANGPTL3

(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.

Drugs of the future will be easier and faster to make, thanks to mRNA – after researchers work out a few remaining kinks

Retrieved on: 
Thursday, January 4, 2024
RNA, Pseudouridine, Liver, Pfizer, University of Massachusetts, Cholesterol, Protein, Phospholipid, Immune system, Infection, Drug development, Messenger, Messenger RNA, CRISPR, Vaccine, Method, Disease, Half-life, Tissue, Endosome, DNA, Heart, Nobel Prize, Program, COVID-19, Engineering, Gene, Cell, Time, Physiology, Medicine, Therapy, Uridine, DSM-IV codes, Algorithm, Orthornavirae

Until the COVID-19 pandemic, however, vaccine development was still a long and idiosyncratic process.

Key Points: 
  • Until the COVID-19 pandemic, however, vaccine development was still a long and idiosyncratic process.
  • But the COVID-19 mRNA vaccines brought a new approach to vaccine development that has far-reaching implications for how researchers make drugs to treat many other diseases.

Some basics of mRNA drugs

  • An mRNA drug comprises two essential components: mRNA molecules, which code for desired proteins, and the lipid molecules – such as phospholipids and cholesterol – that encapsulate them.
  • From a drug development perspective, mRNA drugs offer significant advantages over traditional drugs because they are easily programmable.
  • More importantly, different mRNA drugs produced by the same set of methods will have similar properties.
  • This predictability significantly reduces the development risks and financial costs of developing mRNA drugs.

Self vs. nonself

  • This may sound paradoxical – after all, your cells already contain large amounts of mRNAs.
  • How does your immune system distinguish between self and nonself mRNAs?
  • Therapeutic mRNAs enter cells using endosomes – sacs made of the cell’s membrane that take in materials from the cell’s environment.
  • The 2023 Nobel Prize in physiology or medicine was awarded to the scientists who made this breakthrough discovery.
  • RNA viruses also form double-stranded RNA when they replicate, and exposing cells to double-stranded RNA can lead to a strong immune response.
  • Fortuitously, for mRNA vaccines, the residual amount of double-stranded RNA can stimulate the immune system to enhance antibody responses.

Moving beyond vaccines

  • One promising example in development is using mRNA that encodes CRISPR-Cas9 gene-editing proteins to knock out genes that cause specific diseases.
  • This disease is an ideal target for mRNA-based CRISPR gene therapy because the target protein is produced by the liver.
  • Notable new developments in these areas include using computational algorithms to optimize mRNA sequences in ways that enhance their stability and engineering RNA polymerases that introduce fewer side products that may cause an immune response.
  • Further advancements have the potential to enable a new generation of safe, durable and effective mRNA therapeutics for applications beyond vaccines.


Li Li receives funding from NIH.

Drugs of the future will be easier and faster to make, thanks to mRNA − after researchers work out a few remaining kinks

Retrieved on: 
Thursday, January 4, 2024
RNA, Pseudouridine, Liver, Pfizer, University of Massachusetts, Cholesterol, Protein, Phospholipid, Immune system, Infection, Drug development, Messenger, Messenger RNA, CRISPR, Vaccine, Method, Disease, Half-life, Tissue, Endosome, DNA, Heart, Nobel Prize, Program, COVID-19, Engineering, Gene, Cell, Time, Physiology, Medicine, Therapy, Uridine, DSM-IV codes, Algorithm, Orthornavirae

Until the COVID-19 pandemic, however, vaccine development was still a long and idiosyncratic process.

Key Points: 
  • Until the COVID-19 pandemic, however, vaccine development was still a long and idiosyncratic process.
  • But the COVID-19 mRNA vaccines brought a new approach to vaccine development that has far-reaching implications for how researchers make drugs to treat many other diseases.

Some basics of mRNA drugs

  • An mRNA drug comprises two essential components: mRNA molecules, which code for desired proteins, and the lipid molecules – such as phospholipids and cholesterol – that encapsulate them.
  • From a drug development perspective, mRNA drugs offer significant advantages over traditional drugs because they are easily programmable.
  • More importantly, different mRNA drugs produced by the same set of methods will have similar properties.
  • This predictability significantly reduces the development risks and financial costs of developing mRNA drugs.

Self vs. nonself

  • This may sound paradoxical – after all, your cells already contain large amounts of mRNAs.
  • How does your immune system distinguish between self and nonself mRNAs?
  • Therapeutic mRNAs enter cells using endosomes – sacs made of the cell’s membrane that take in materials from the cell’s environment.
  • The 2023 Nobel Prize in physiology or medicine was awarded to the scientists who made this breakthrough discovery.
  • RNA viruses also form double-stranded RNA when they replicate, and exposing cells to double-stranded RNA can lead to a strong immune response.
  • Fortuitously, for mRNA vaccines, the residual amount of double-stranded RNA can stimulate the immune system to enhance antibody responses.

Moving beyond vaccines

  • One promising example in development is using mRNA that encodes CRISPR-Cas9 gene-editing proteins to knock out genes that cause specific diseases.
  • This disease is an ideal target for mRNA-based CRISPR gene therapy because the target protein is produced by the liver.
  • Notable new developments in these areas include using computational algorithms to optimize mRNA sequences in ways that enhance their stability and engineering RNA polymerases that introduce fewer side products that may cause an immune response.
  • Further advancements have the potential to enable a new generation of safe, durable and effective mRNA therapeutics for applications beyond vaccines.


Li Li receives funding from NIH.

EnginZyme Produces Key mRNA Vaccine Ingredient Using Biocatalysis

Retrieved on: 
Wednesday, October 25, 2023
Chemicals, Plastics, Retail, Manufacturing, Technology, Pharmaceutical, Supply Chain Management, Infectious Diseases, Artificial Intelligence, Transport, Science, Biotechnology, Logistics, Health, Research, Medical Supplies, Physiology, Uridine, FDA, CGMP, Pfizer–BioNTech COVID-19 vaccine, Therapy, Vaccine, RNA, CDMO, Enzyme, R, Ajinomoto, Hoarding, COVID-19, World Health Organization, Medicine, Messenger, Nobel Prize in Physiology or Medicine, Nobel Prize, Pseudouridine, Fine chemical, Pharmaceutical industry

The companies said they had already synthesized enough pseudouridine for more than half a billion doses of vaccine in a facility that is fully cGMP* compliant.

Key Points: 
  • The companies said they had already synthesized enough pseudouridine for more than half a billion doses of vaccine in a facility that is fully cGMP* compliant.
  • The enzymatic process EnginZyme has perfected is cleaner and more efficient than the chemical synthesis methods currently used.
  • A critical impurity, alpha-pseudouridine, which is a by-product of the chemical synthesis of beta-pseudouridine, is eliminated in EnginZyme's patented enzymatic synthesis.
  • This project with EnginZyme has been particularly rewarding because of its potential to improve the global supply chain for vaccine ingredients.”
    N1-methylpseudouridine-5’-triphosphate, which is derived from pseudouridine, helps stabilize and reduce the immunogenicity of mRNA.

Nobel prize in medicine awarded to mRNA pioneers – here's how their discovery was integral to COVID vaccine development

Retrieved on: 
Monday, October 2, 2023
Vaccine, BioNTech, Nobel Prize, DNA, University, Cell membrane, HIV, Guanine, Pseudouridine, Oxygen, Infection, COVID-19, Cancer, Lipid, Adenine, Physiology, Research, COVID, Cell, Zika virus, Messenger, Nucleoside, Cytosine, Uridine, Immune system, Viral, ARD International Music Competition, Mouse, Weissmann, Pfizer, Pharmaceutical industry

The rapid development of these vaccines changed the course of the pandemic, providing protection against the SARS-CoV-2 virus.

Key Points: 
  • The rapid development of these vaccines changed the course of the pandemic, providing protection against the SARS-CoV-2 virus.
  • Their discovery was not only integral to COVID-19 vaccine development, but may also lead to the development of many other therapies – such as vaccines for cancer.

Life’s work

    • It’s made in the body from our very own DNA in a process called translation.
    • DNA is our special encoded handbook of instructions for manufacturing proteins, which are the building blocks for material in the body.
    • The cells then make whatever protein they’ve been instructed to, such as haemoglobin for helping red blood cells carry oxygen around the body.
    • The researchers faced two major challenges as they began their work.
    • Karikó and Weissman’s research had successfully eliminated the obstacles that had previously stood in the way of using mRNA clinically.

COVID vaccines

    • Researchers had already been working on developing mRNA vaccines before the pandemic, such as a vaccine for Ebola that didn’t receive much commercial interest.
    • This produced a harmless COVID particle which our cells then replicated, allowing our bodies to protect us from severe COVID infections when it encountered the real virus.
    • Studies have also shown mRNA vaccines might be useful in treating certain types of cancer.