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BioWorld - Thursday, April 23, 2026
Breaking News: Best of BioWorld Science: Q1Breaking News: Best of BioWorld: Q1Breaking News: Best of BioWorld: Q1
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Kidney, pills, bottle
Nephrology

Further financing at Mironid to support lead program through IND-enabling studies for ADPKD

Sep. 13, 2023
Mironid Ltd. has announced an extension of its series A financing round to support its development of small-molecule therapeutics for the treatment of autosomal dominant polycystic kidney disease (ADPKD). The company will use the proceeds of the financing to advance its lead discovery program through IND-enabling studies.
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Neurology/Psychiatric

Gain Therapeutics receives Australian HREC approval to begin phase I study of GT-02287 for GBA1-Parkinson’s disease

Sep. 13, 2023
Gain Therapeutics Inc. has received human research ethics committee (HREC) approval in Australia to initiate a phase I study of its lead drug candidate GT-02287 in development for GBA1-Parkinson’s disease. The study is expected to begin in the near term.
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Acute myeloid leukemia illustration
Cancer

OXS-007464 shows potency against a wide range of AML subtypes

Sep. 13, 2023
Researchers from the University of Oxford and Oxstem Ltd. have discovered small-molecule candidates with the ability to induce differentiation of acute myeloid leukemia (AML) cells.
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Musculoskeletal

Rome Therapeutics raises new funds to advance LINE-1 reverse transcriptase inhibitor into clinic

Sep. 13, 2023
Rome Therapeutics Inc. has completed a $72 million series B extension financing, bringing the total series B amount raised to $149 million.
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Pancreas
Cancer

Novel radioimmunotherapy [225Ac]-Ac-DOTA-C595 for pancreatic ductal adenocarcinoma presented

Sep. 13, 2023
Pancreatic ductal adenocarcinoma (PDAC) is an exocrine pancreatic cancer with a poor prognosis and limited treatment options. There is a strong demand for new treatment strategies to overcome these challenges.
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Cancer

Riana collaborates with LDC on discovery of inhibitors targeting oncogenic PPI interactions

Sep. 13, 2023
Riana Therapeutics GmbH, a spin-off from Vetmeduni Vienna founded this year, has entered into a collaborative agreement with the Lead Discovery Center GmbH (LDC) to support its efforts to discover and develop novel cancer therapeutics that disrupt oncogenic protein-protein interactions (PPIs).
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Scientist looking in microscope, chemical structure concept image
Drug Design, Drug Delivery & Technologies

Biomarin outlines progress of preclinical pipeline

Sep. 13, 2023
Biomarin Pharmaceutical Inc. has reported progress across its research and development portfolio, including early-stage product candidates.
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Lungs
Respiratory

HL-001 demonstrates antifibrotic effects in murine and human organoid models of IPF

Sep. 13, 2023
At the recent ERS meeting, researchers from Hilung Inc. and Ube Corp. presented preclinical data for the novel lysophospholipid LPA1 receptor antagonist, HL-001, being developed for the treatment of idiopathic pulmonary fibrosis (IPF).
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Respiratory

ARV-825 shows both antifibrotic and senolytic effects in lung fibrosis model

Sep. 13, 2023
Senescence of lung epithelial tissue translates into fibrinolysis reduction and loss of tissue repair, both drivers of pulmonary fibrotic diseases. Researchers from Tokushima University reported on the effect of ARV-825, a BRD4 degrader, as an antifibrotic agent in lung fibrosis.
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Other news to note for Sept. 12, 2023

Sep. 12, 2023
Additional early-stage research and drug discovery news in brief.
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