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BioWorld - Thursday, April 9, 2026
Home » Topics » Science » RNA

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Nephrology

Arrowhead files to begin phase I/IIa trial of ARO-CFB for complement-mediated renal disease

Dec. 22, 2023
Arrowhead Pharmaceuticals Inc. has filed an application in New Zealand seeking clearance to initiate a phase I/IIa trial of ARO-CFB, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for complement-mediated renal disease, such as immunoglobulin A nephropathy (IgAN).
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Bladder cancer illustration
Cancer

Ractigen files to begin phase I study in Australia with saRNA drug candidate for bladder cancer

Dec. 7, 2023
Ractigen Therapeutics Co. Ltd. has submitted a clinical trial application in Australia seeking to conduct a phase I study of RAG-01, a small activating RNA (saRNA) drug candidate, in patients with non-muscle-invasive bladder cancer who have not responded to Bacillus Calmette-Guérin (BCG) therapy.
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Digital spine concept art
Neurology/Psychiatric

Nurexone reports new siRNA sequences with potential for spinal cord injury

Dec. 1, 2023
Nurexone Biologic Inc. has reported results from laboratory tests of its secondary two proprietary sequences, showing promise for the treatment of spinal cord injuries.
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Heart illustration
Cardiovascular

Ethris and Heqet collaborate to develop RNA-based therapeutics for heart tissue regeneration

Nov. 13, 2023
Ethris GmbH and Heqet Therapeutics srl, a company spun out last year from King’s College London, have entered into a collaboration agreement to harness the potential of non-coding RNA (ncRNA) for heart tissue regeneration following acute myocardial infarction and in heart failure.
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3D rendering of a liposome containing RNA strand
Cancer

Scientists design a circular RNA against adenocarcinoma

Oct. 19, 2023
By Mar de Miguel
The mRNA technology used in vaccines against viral infections could also be developed for cancer therapies. A group of scientists has designed a circular RNA (circRNA) encapsulated in lipid nanoparticles (LNPs) that acts in the mitochondria of tumor cells through the protein gasdermin-D (GSDMD) and reduces adenocarcinoma. The work was published on Oct. 16, 2023, in Nature Cancer.
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RNA illustration
Drug Design, Drug Delivery & Technologies

Deep Genomics announces AI foundation model for RNA

Sep. 28, 2023
Deep Genomics Inc. has introduced its artificial intelligence (AI) foundation model for RNA, BigRNA, which enables discovery of disease mechanisms and candidate therapeutics.
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3D rendered illustration of a synapse cross-section
Neurology/Psychiatric

Reduced RNA editing reveals mitochondrial dysfunction in schizophrenia

April 11, 2023
By Mar de Miguel
RNA editing in schizophrenia (SCZ)-associated genes was decreased in postmortem brains of individuals of European descent, according to a study from the University of California, Los Angeles (UCLA). The scientists obtained the RNA editome from SCZ brains to detect the sequence changes in their RNA and observed hypoediting in noncoding regions related to mitochondrial function, such as the mitofusin-1 (MFN1) gene.
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Endocrine/Metabolic

Korro Bio and Genevant Sciences partner on RNA editing therapeutic for alpha-1 antitrypsin deficiency

March 8, 2023
Korro Bio Inc. and Genevant Sciences Corp. have entered into an agreement to combine Korro's...
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Neurology/Psychiatric

Blocking nuclear export of ALS/FTD troublemaker helps neurons survive

March 3, 2023
By Anette Breindl
Treatment with a cell-penetrating peptide that prevented nuclear export of unprocessed C9ORF72 RNA and its subsequent translation into neurotoxic dipeptide repeat proteins reduced motor neuron damage and death both in fruit fly models of amyotrophic lateral sclerosis (ALS), and in patient-derived induced neuronal precursor cells (iNPCs). The work suggests that targeting nuclear export could be a therapeutic option in ALS, and possibly also frontotemporal dementia (FTD), where C9ORF72 mutations also play a role.
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RNA strand
Drug Design, Drug Delivery & Technologies

Cas12a2 elicits abortive infection and can be repurposed as an RNA-guided RNA-targeting tool

Jan. 20, 2023
Bacterial abortive infection is a defense mechanism by which an infected bacterial cell enters dormancy or dies to limit phage replication and protect the clonal population. Recent studies observed that CRISPR RNA-guided adaptive immune systems that target RNA also cause abortive-infection phenotypes by activating indiscriminate nucleases.
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