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Home » Topics » Drugs » RNAi

RNAi
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Cancer cell, dropper, test tubes
Cancer

Silexion’s SIL-204 shows promise in pancreatic, colorectal and lung cancer cell lines

May 30, 2025
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Silexion Therapeutics Corp. has released preclinical data demonstrating the efficacy of its next-generation RNA interference (RNAi) therapeutic candidate, SIL-204, against human pancreatic, colorectal and lung cancer cell lines.


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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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Biotech deal illustration

City hits the town with $1B-plus deal with Biogen

May 27, 2025
By Lee Landenberger
No Comments
A development deal with Biogen Inc. could eventually bring City Therapeutics Inc. about $1 billion in milestone payments. It’s a step in the direction the company had been going: looking for the right partners, including those from big pharma. Privately held City Therapeutics is getting $46 million in the deal. That includes $16 million as an up-front payment and an investment of $30 million in exchange for a City Therapeutics convertible note.
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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
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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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Illustration of siRNA structure

Sirius bags $50M series B2 for cardiovascular siRNA pipeline

May 13, 2025
By Marian (YoonJee) Chu
No Comments
Sirius Therapeutics Inc. raised nearly $50 million in a series B2 financing round May 9 to support its pipeline of small interfering RNA (siRNA) molecules for cardiovascular disease indications. SRSD-107, a long-acting Factor XI anticoagulant for thromboembolic disorders, is its lead candidate waiting on EMA clearance to begin a phase II study in Europe. 
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Illustration of siRNA structure

Sirius bags $50M series B2 for cardiovascular siRNA pipeline

May 12, 2025
By Marian (YoonJee) Chu
No Comments
Sirius Therapeutics Inc. raised nearly $50 million in a series B2 financing round May 9 to support its pipeline of small interfering RNA (siRNA) molecules for cardiovascular disease indications. SRSD-107, a long-acting Factor XI anticoagulant for thromboembolic disorders, is its lead candidate waiting on EMA clearance to begin a phase II study in Europe. 
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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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Illustration of hole in vessel wall with repair process in progress

Sanofi adds to hemophilia arsenal with FDA nod for Qfitlia

March 31, 2025
By Jennifer Boggs
Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment to prevent or reduce bleeding episodes in people with hemophilia A or B, with or without inhibitors.
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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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