NTCP

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

Retrieved on: 
Friday, January 19, 2024

“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: 
Tuesday, November 7, 2023

(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: 
Monday, October 2, 2023

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: 
Tuesday, September 19, 2023

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: 
Thursday, August 3, 2023

(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.

Assembly Biosciences Presents New Data Highlighting Entry Inhibitor and Core Inhibitor Programs at EASL’s International Liver Congress™ 2023

Retrieved on: 
Wednesday, June 21, 2023

“We are excited to share the latest data on our promising HDV/HBV entry inhibitor program and next-generation HBV core inhibitors with the scientific community,” said William Delaney, PhD, chief scientific officer of Assembly Bio.

Key Points: 
  • “We are excited to share the latest data on our promising HDV/HBV entry inhibitor program and next-generation HBV core inhibitors with the scientific community,” said William Delaney, PhD, chief scientific officer of Assembly Bio.
  • “Data presented from our entry inhibitor program, from which we expect to nominate a development candidate this year, highlight our rapid progress in developing a small molecule inhibitor of this validated mechanism.
  • ABI-H3733 (3733) and ABI-4334 (4334) are novel, structurally distinct, orally bioavailable investigational core inhibitors that exhibit nM potency against pgRNA encapsidation and covalently closed circular (ccc)DNA formation.
  • Subsequent to presentation at EASL’s International Liver Congress™ 2023, Assembly Bio intends to make the posters available on the “Events & Presentations” page in the “Investors” section of its website at www.assemblybio.com .

ProQR Announces First Quarter 2023 Operating and Financial Results

Retrieved on: 
Tuesday, May 16, 2023

(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, 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 first quarter ended March 31, 2023, and provided a business update.
  • Research and development (R&D) costs were €6.1 million for the quarter ended March 31, 2023 compared to €13.4 million for the same period last year.
  • General and administrative costs were €4.0 million for the quarter ended March 31, 2023 compared to €4.9 million for the same period last year.
  • For further financial information for the period ended March 31, 2023, please refer to the financial statements appearing at the end of this release, or the Q1 financial report filing.

ProQR Announces Initial Pipeline Targets and Highlights Axiomer® RNA Editing Platform Technology at R&D Event

Retrieved on: 
Wednesday, March 29, 2023

(Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today announced initial pipeline programs focused on diseases that originate in the liver.

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 announced initial pipeline programs focused on diseases that originate in the liver.
  • ProQR will host a virtual R&D event today, during which the Company will showcase its proprietary Axiomer RNA-editing technology platform, detail the pipeline, and provide guidance on the advancement of programs toward the clinic.
  • ProQR today announced AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease targeting B4GALT1 as initial pipeline programs.
  • Beyond this, ProQR has several unpublished patent applications and continuously invests in expanding its IP estate around ADAR-mediated RNA editing.

Ipsen completes acquisition of Albireo, expanding the scope of its Rare Disease portfolio

Retrieved on: 
Thursday, March 2, 2023

The acquisition enriches Ipsen’s Rare Disease portfolio, with promising therapeutics for pediatric and adult rare cholestatic-liver diseases, innovative pipeline potential, as well as scientific and commercial capabilities.

Key Points: 
  • The acquisition enriches Ipsen’s Rare Disease portfolio, with promising therapeutics for pediatric and adult rare cholestatic-liver diseases, innovative pipeline potential, as well as scientific and commercial capabilities.
  • “The acquisition of Albireo will greatly strengthen our portfolio in rare diseases,” said David Loew, Chief Executive Officer of Ipsen.
  • “I am excited to welcome new colleagues to Ipsen, who led the innovation on the development of novel bile acid modulators, like Bylvay, to treat rare liver diseases in children and adults.
  • As part of the transaction, Ipsen has also acquired A3907 and A2342, two clinical-stage assets in Albireo’s pipeline.

Ipsen to Acquire Albireo Accelerating Growth in Rare Disease With Treatments for Several Pediatric Liver Diseases

Retrieved on: 
Monday, January 9, 2023

Ipsen (Euronext: IPN: ADR: IPSEY) and Albireo (Nasdaq: ALBO) today announced that they have entered into a definitive merger agreement under which Ipsen will acquire Albireo, a leading innovator in bile-acid modulators to treat pediatric and adult cholestatic liver diseases.

Key Points: 
  • Ipsen (Euronext: IPN: ADR: IPSEY) and Albireo (Nasdaq: ALBO) today announced that they have entered into a definitive merger agreement under which Ipsen will acquire Albireo, a leading innovator in bile-acid modulators to treat pediatric and adult cholestatic liver diseases.
  • “Our Rare Disease franchise is strengthened with Bylvay, which, in addition to being the first-approved treatment in PFIC, has two further indications being investigated in rare liver conditions that are underserved.
  • “Our talented team at Albireo have advanced the first Phase III studies in three different pediatric liver diseases while discovering two promising new clinical stage bile acid modulators.
  • Goldman Sachs is acting as exclusive financial advisor to Ipsen and Orrick Herrington & Sutcliffe LLP as legal counsel to Ipsen.