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Home » Topics » Drugs » RNAi

RNAi
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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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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 hole in vessel wall with repair process in progress

Sanofi adds to hemophilia arsenal with FDA nod for Qfitlia

March 28, 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, and joins the small but growing group of non-factor therapy options, with the advantage of a broad label and a convenient dosing regimen.
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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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Liver illustration

Olix wins $630M deal from Eli Lilly for phase I MASH asset

Feb. 11, 2025
By Marian (YoonJee) Chu
Olix Pharmaceuticals Inc. walked the talk in realizing a new $630 million licensing deal with Eli Lilly and Co. for its cardiovascular and metabolic disease asset, OLX-702A (OLX-75016), rallying stock by 30% after it had largely recovered from a terminated deal with France’s Théa Open Innovation last year.
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