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BioWorld - Tuesday, March 17, 2026
Home » Topics » RNAi, BioWorld Science

RNAi, BioWorld Science
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Artery and plaque
Cardiovascular

Rona Therapeutics reports preclinical findings with RNAi therapy for cardiovascular disease

May 30, 2025
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Rona Therapeutics Co. Ltd. has reported preclinical data for its RNA interference (RNAi) therapy RN-026 targeting lipoprotein(a) (Lp[a]).
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Nuvec gene delivery system
Gastrointestinal

N4 Pharma completes in vivo study of siRNA and mRNA-loaded oral Nuvec formulations

May 21, 2025
No Comments
N4 Pharma plc has successfully completed the first in vivo study of orally delivered Nuvec using its lead program N4 101, an orally administered anti-inflammatory treatment for inflammatory bowel disease (IBD).
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Blood pressure monitor
Cardiovascular

Chinese IND clearance for Rona′s AGT-targeting siRNA drug for hypertension

April 25, 2025
Rona Therapeutics Co. Ltd. has obtained IND clearance from China′s National Medical Products Administration (NMPA) for RN-1871, a small interfering RNA (siRNA) drug targeting angiotensinogen (AGT) for hypertension.
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Hands holding brain
Neurology/psychiatric

C3-targeting siRNA shows promise for Alzheimer’s: findings from cynomolgus monkey study

April 3, 2025
Previous work uncovered the role of the complement system in neuroinflammation and synaptic loss in neurodegenerative disorders such as Alzheimer’s disease (AD). The third component, C3, central in all complement activation pathways, has been proposed as a potential therapeutic target in AD.
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Liver illustration
Gastrointestinal

ABHD17B identified as a target in liver fibrosis

March 31, 2025
Scientists at Massachusetts General Hospital have revealed how chronic liver injury alters hepatic stellate cells (HSCs), triggering the formation of fibrotic scarring in the liver. The researchers have shown that this cell type transdifferentiates into myofibroblasts, which generate excess extracellular matrix, driven by the activation of ABHD17B, an enzyme that could be investigated as a therapeutic target to inhibit liver fibrosis.
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3D illustration of pancreatic cancer
Cancer

Silexion plans dual-route strategy for SIL-204 for KRAS-driven pancreatic cancer

March 31, 2025
Silexion Therapeutics Corp. has announced an expanded development plan for its next-generation siRNA candidate SIL-204. The new dual-route strategy will integrate intratumoral and systemic administration to target both primary tumors and metastases, respectively, to treat KRAS-driven pancreatic cancer.
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Illustration of cancer in the pancreas
Cancer

Silexion completes study of SIL-204 in orthotopic pancreatic cancer models

Feb. 26, 2025
Silexion Therapeutics Corp. has completed its initial study evaluating SIL-204 in orthotopic pancreatic cancer models. SIL-204 is a next-generation siRNA candidate designed to target a broad range of KRAS mutations.
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RNA
Cancer

Akribion harnessing G-dase E nuclease, with initial focus in cancer

Feb. 5, 2025
By Nuala Moran
Newco Akribion Therapeutics GmbH has raised €8 million (US$8.3 million) in a seed round to develop a new and potent class of RNA-targeted CRISPR nucleases, which, rather than cleaving specific nucleic acids, can destroy every type of nucleic acid in a cell.
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Illustration of siRNA structure
Cardiovascular

Huadong Medicine and Synerk to jointly develop siRNA drug

Dec. 30, 2024
Hangzhou Sino-US Huadong Medicine Co. Ltd. and Synerk Pharmatech (Suzhou) Co. Ltd. have entered into a strategic collaboration in which the two parties will jointly develop the small nucleic acid (siRNA) drug SNK-2726.
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Cardiovascular

SYH-2062 cleared for clinic in China for hypertension

Dec. 24, 2024
CSPC Pharmaceutical Group Ltd. has gained clinical trial approval from China’s National Medical Products Administration (NMPA) for SYH-2062 injection, a double-stranded small interfering RNA (siRNA) drug to treat hypertension.
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