NTCP

Assembly Biosciences Presents New Data Highlighting Hepatitis D Virus Entry Inhibitor ABI-6250 at the EASL Congress™ 2024

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Mittwoch, Juni 5, 2024
Cell, Hepatitis B virus, NTCP, Doctor of Philosophy, Cirrhosis, SAN, EASL, Safety, Poster, Biomarker, Liver disease, Viral hepatitis, Death, Therapy, Viral, HDV, Liver cancer, Sodium, Biotechnology, HIV, Hepatitis, Assembly, Vaccine

SOUTH SAN FRANCISCO, Calif., June 05, 2024 (GLOBE NEWSWIRE) -- Assembly Biosciences, Inc. (Nasdaq: ASMB), a biotechnology company developing innovative therapeutics targeting serious viral diseases, today announced new data for ABI-6250, the company’s orally bioavailable, small molecule hepatitis D virus (HDV) entry inhibitor candidate, featured in a poster presentation at the European Association for the Study of the Liver (EASL) Congress™, taking place June 5-8, 2024, in Milan, Italy.

Key Points: 
  • The poster presentation “Preclinical profiling of ABI-6250, a novel orally bioavailable small-molecule therapeutic candidate for the treatment of chronic hepatitis D” will highlight preclinical data that support the advancement of ABI-6250 into Phase 1 clinical studies.
  • Chronic HDV infection is considered the most serious form of viral hepatitis, and can result in liver cirrhosis, liver cancer, decompensated liver disease or death.
  • ABI-6250 also effectively inhibited NTCP-mediated bile acid uptake and demonstrated selectivity for the NTCP bile transporter versus other transporters in vitro.
  • ABI-6250 is an investigational product candidate that has not been approved anywhere globally, and its safety and efficacy have not been established.

ProQR Announces First Quarter 2024 Operating and Financial Results

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Donnerstag, 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.

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

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Mittwoch, Mai 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.

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

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Montag, April 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 Announces Year End 2023 Operating and Financial Results

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Mittwoch, März 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.

ProQR Highlights New Platform Data from Presentation on Axiomer™ RNA Editing Technology at Deaminet 2024

Retrieved on: 
Freitag, Januar 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.

ProQR Announces Third Quarter 2023 Operating and Financial Results

Retrieved on: 
Dienstag, November 7, 2023
NTCP, B4GALT1, ADAR, Research, Lilly, Patent, RNA, Eli Lilly, Adenosine, Liver, Conference, Oligonucleotide, Bank statement, ProQR, Partnership, Inosine, IP, Patient, NHP, Airline, Cryptocurrency, 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 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.

Assembly Biosciences Nominates Development Candidate ABI-6250, an Orally Bioavailable HDV Entry Inhibitor

Retrieved on: 
Montag, Oktober 2, 2023
Injection, Viremia, NTCP, Assembly, Bio, Biotechnology, HDV, Viral, Liver injury, Hepatitis B virus, Doctor of Philosophy, Liver cancer, Patient, SAN, Viral hepatitis, Cirrhosis, IND, HBV, Vaccine

SOUTH SAN FRANCISCO, Calif., Oct. 02, 2023 (GLOBE NEWSWIRE) -- Assembly Biosciences, Inc. (Nasdaq: ASMB), a biotechnology company developing innovative antiviral therapeutics targeting serious viral diseases, today announced the selection of development candidate ABI-6250 to progress to IND-enabling studies. ABI-6250, an orally bioavailable small molecule entry inhibitor, is the first development candidate nominated by the company for the treatment of chronic hepatitis D virus (HDV) infection, and the second development candidate selected in 2023, following ABI-5366 for high-recurrence genital herpes.

Key Points: 
  • ABI-6250, an orally bioavailable small molecule entry inhibitor, is the first development candidate nominated by the company for the treatment of chronic hepatitis D virus (HDV) infection, and the second development candidate selected in 2023, following ABI-5366 for high-recurrence genital herpes.
  • “We’re pleased to nominate ABI-6250 as a development candidate targeting chronic HDV infection, the most severe form of viral hepatitis,” said William Delaney, PhD, chief scientific officer of Assembly Bio.
  • Patients with chronic HDV infection experience much higher rates of cirrhosis and liver cancer than those with chronic HBV infection only.
  • Inhibiting viral entry by blocking NTCP is a clinically validated target for reducing HDV viremia and liver injury in patients.

Assembly Biosciences to Highlight Pipeline Progress in HBV and HDV at the 2023 International HBV Meeting

Retrieved on: 
Dienstag, September 19, 2023
PK, DNA, NTCP, International, Assembly, DL, Biotechnology, HDV, CAM, Nm, Viral, Hepatitis B, Hepatitis B virus, Doctor of Philosophy, Liver cancer, Plasma, Patient, Liver, Poster, Viral hepatitis, HBV, Mutation, Vaccine

SOUTH SAN FRANCISCO, Calif., Sept. 19, 2023 (GLOBE NEWSWIRE) -- Assembly Biosciences, Inc. (Nasdaq: ASMB), a biotechnology company developing innovative antiviral therapeutics targeting serious viral diseases, today announced that the company will present new preclinical data from multiple hepatitis B virus (HBV) and hepatitis D virus (HDV) pipeline programs at the 2023 International HBV Meeting taking place in Kobe, Japan, September 19-23, 2023.

Key Points: 
  • At the meeting, one oral presentation will highlight progress in the company’s HBV/HDV oral entry inhibitor program including activity, selectivity and pharmacokinetic (PK) data that support advancement towards development candidate nomination.
  • A second oral presentation reports new data for ABI-4334 (4334), Assembly Bio’s most potent capsid assembly modulator (CAM) with a potential best-in-class profile, describing the prevention of HBV DNA integration in vitro.
  • The poster presentation, from the company’s interferon-α receptor (IFNAR) agonist program, characterizes small molecule IFNAR agonists that closely mimic interferon-α’s signaling.
  • In patients with chronic HBV infection, HBV DNA integration has been linked to the development of liver cancer.

ProQR Announces Second Quarter 2023 Operating and Financial Results

Retrieved on: 
Donnerstag, August 3, 2023
Partnership, NTCP, ADAR, Bank statement, Research, IP, RNA, Eli Lilly, Publishing, Lilly, Patient, Liver, NHP, Therapy, B4GALT1, ProQR, Bank, Vaccine, Pharmaceutical industry, Cryptocurrency, EU, Axiom Space

(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 second quarter ended June 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 second quarter ended June 30, 2023, and provided a business update.
  • The transaction is expected to close in the third quarter of 2023, subject to the satisfaction of customary closing conditions.
  • General and administrative costs were €4.1 million for the quarter ended June 30, 2023 compared to €5.4 million for the quarter ended June 30, 2022.
  • For further financial information for the period ending June 30, 2023, please refer to the financial statements appearing at the end of this release.