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BioWorld - Saturday, March 21, 2026
Home » Topics » RNAi, BioWorld Science

RNAi, BioWorld Science
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Illustration of glucose molecules in a bloodstream
Endocrine/metabolic

Junevity raises funds to advance siRNA metabolism program

Dec. 4, 2025
No Comments
Junevity Inc. has raised $10 million in new funding, bringing its seed financing to a total of $20 million. The new funding will enable Junevity to advance JUN-01, its lead siRNA program for type 2 diabetes and obesity, through IND-enabling and initial clinical studies.
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Liver disease
Gastrointestinal

Tangram seeks clinical trial clearance for TGM-312 for MASH

Nov. 26, 2025
No Comments
Tangram Therapeutics plc has submitted a clinical trial application (CTA) to the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II trial of TGM-312 for metabolic dysfunction-associated steatohepatitis (MASH).
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Illustration of clot forming in blood vessel
Hematologic

City Therapeutics seeks clinical trial clearance for CITY-FXI

Nov. 11, 2025
No Comments
City Therapeutics Inc. has submitted a clinical trial application (CTA) to the U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) to initiate a phase I study of CITY-FXI, an investigational RNAi therapeutic targeting factor XI (FXI) for the treatment of thromboembolic diseases.
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Microscope with slide
Cardiovascular

Corsera Health seeks clinical trial clearance for COR-1004

Oct. 28, 2025
No Comments
Corsera Health Inc. has filed a clinical trial notification (CTN) seeking to initiate a phase I trial of COR-1004 in New Zealand. COR-1004 is a novel investigational subcutaneously administered siRNA targeting proprotein convertase subtilisin/kexin type 9 (PCSK9) to reduce LDL cholesterol (LDL-C).
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Illustration of tau proteins in Alzheimer's disease
Neurology/psychiatric

Arrowhead files to begin clinical trials of ARO-MAPT

Sep. 12, 2025
No Comments
Arrowhead Pharmaceuticals Inc. has filed a request for regulatory clearance in New Zealand to initiate a phase I/IIa trial of ARO-MAPT, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for tauopathies, including Alzheimer’s disease.
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Parkinson's disease illustration showing neurons containing alpha-synuclein
Neurology/psychiatric

Arrowhead and Novartis sign agreement for ARO-SNCA

Sep. 3, 2025
No Comments
Arrowhead Pharmaceuticals Inc. has announced a global licensing and collaboration agreement with Novartis AG for ARO-SNCA, Arrowhead’s preclinical stage siRNA therapy for the treatment of synucleinopathies such as Parkinson’s disease, and for additional targets.
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3D illustration of RNA
Immune

Axelyf emerges to tackle RNA delivery, advance autoimmune pipeline

Aug. 26, 2025
By Karen Carey
No Comments
Partly focused on delivery challenges that have limited the reach of RNA medicines, new biotech company Axelyf Inc. closed a $2.6 million seed round to support development of its AXL technology and to advance lead autoimmune candidate AXL-003.
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Illustration of RNA strands being delivered by nanoparticles
Neurology/psychiatric

Nanoparticle-based intranasal siRNA delivery for Parkinson’s

Aug. 14, 2025
No Comments
In Parkinson’s disease, α-synuclein accumulates in neurons and may thereby contribute to their degeneration. Reducing expression of α-synuclein may be an effective therapy, but delivering short interfering RNA (siRNA) to the brain noninvasively is notoriously ineffective, in part because siRNA does not pass the blood-brain barrier efficiently.
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Interfering RNA (RNAi)
Cancer

New RNAi targets KRAS G12V using a cancer cell receptor

June 20, 2025
No Comments
An RNA interference (RNAi) molecule that selectively targets the KRAS G12V mutation could represent a key advance in the treatment of cancer associated with this oncogenic variant. Researchers at the University of North Carolina (UNC) at Chapel Hill, in collaboration with Enfuego Therapeutics Inc., have developed a new RNAi designed to enter cells through the epidermal growth factor receptor (EGFR), which is commonly overexpressed in tumor cells. This targeted entry pathway could minimize the side effects associated with therapies that affect KRAS.
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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
No Comments

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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