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BioWorld - Thursday, December 25, 2025
Home » Topics » Drugs » RNAi

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Red blood cells in an artery with cholesterol

Kalexo enters dyslipidemia fray with preclinical siRNA candidate

Sep. 23, 2025
By Marian (YoonJee) Chu
Mabwell Bioscience Co. Ltd. and Aditum Bio Management Co. LLC announced, in after-market hours Sept. 17, an agreement to forge a new company called Kalexo Bio Inc. and load the biotech with a preclinical dyslipidemia asset via a potential $1 billion global license deal.
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Red blood cells in an artery with cholesterol

Kalexo enters dyslipidemia fray with preclinical siRNA candidate

Sep. 18, 2025
By Marian (YoonJee) Chu
No Comments
Mabwell Bioscience Co. Ltd. and Aditum Bio Management Co. LLC announced, in after-market hours Sept. 17, an agreement to forge a new company called Kalexo Bio Inc. and load the biotech with a preclinical dyslipidemia asset via a potential $1 billion global license deal.
Read More
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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Deal illustration

Novartis taps Argo again, inks potential $5.2B collaboration

Sep. 9, 2025
By Jennifer Boggs
No Comments
Argo Biopharmaceutical Co. Ltd. and its RNAi work drew Novartis AG back to the table for a third time, as the companies entered a potential $5.2 billion deal involving cardiovascular-focused assets, including a right to first negotiation for BW-00112, an angiopoietin-like protein 3-targeting siRNA in phase II testing in severe hypertriglyceridemia.
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Deal illustration

Novartis taps Argo again, inks potential $5.2B collaboration

Sep. 3, 2025
By Jennifer Boggs
No Comments
Argo Biopharmaceutical Co. Ltd. and its RNAi work drew Novartis AG back to the table for a third time, as the companies entered a potential $5.2 billion deal involving cardiovascular-focused assets, including a right to first negotiation for BW-00112, an angiopoietin-like protein 3-targeting siRNA in phase II testing in severe hypertriglyceridemia.
Read More
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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Clinical trial virtual display

Regeneron's cemdisiran hits phase III endpoints in myasthenia gravis

Aug. 26, 2025
By Karen Carey
No Comments
Amid the increasingly competitive myasthenia gravis drug development space, siRNA candidate cemdisiran met phase III endpoints, with the monotherapy showing numerically higher results than a combination product. Regeneron Pharmaceuticals Inc., which has a worldwide license to cemdisiran from Alnylam Pharmaceuticals Inc., plans to file for U.S. approval in generalized myasthenia gravis, a rare and chronic autoimmune disease leading to life-threatening muscle weakness, in the first quarter of 2026.
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3D illustration of RNA
Newco news

Axelyf emerges to tackle RNA delivery, advance autoimmune pipeline

Aug. 25, 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.
Read More
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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