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BioWorld - Thursday, March 19, 2026
Home » Topics » RNAi, BioWorld Science

RNAi, BioWorld Science
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3D illustration of RNA
Endocrine/metabolic

China’s NMPA clears China Medical System’s CMS-D008 for clinic

March 6, 2026
No Comments
China Medical System Holdings Ltd. has received clinical trial approval from China’s National Medical Products Administration (NMPA) for CMS-D008 injection for overweight or obese individuals.
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Illustration of human heart with DNA structure background
Cardiovascular

Atrium Tx launches with Avidity's RNA ticker and cardio assets

March 2, 2026
By Karen Carey
No Comments
Targeting rare cardiomyopathy conditions with no approved disease-modifying therapies, Atrium Therapeutics Inc. started operations in San Diego, assuming the public listing and technology once held by Avidity Biosciences Inc., an RNA company acquired by Novartis AG for about $12 billion. Novartis closed the M&A, announced last October, as Atrium unveiled its pipeline and a $270 million cash position.
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Illustration of kidney with DNA structures
Nephrology

GSK licenses Frontier Biotechnologies’ siRNA therapeutics

Feb. 25, 2026
No Comments
Frontier Biotechnologies Inc. has entered into an exclusive licensing agreement with GSK plc whereby GSK will obtain exclusive worldwide rights to develop, manufacture and commercialize two of Frontier’s small interfering RNA (siRNA) pipeline products.
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siRNA bound to mRNA
Gastrointestinal

Madrigal licenses six Ribo siRNA programs

Feb. 11, 2026
No Comments
Madrigal Pharmaceuticals Inc. has signed an exclusive global license agreement with Suzhou Ribo Life Science Co. Ltd. and its subsidiary Ribocure Pharmaceuticals AB for six preclinical small interfering RNA (siRNA) programs.
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Interfering RNA (RNAi)
Drug design, drug delivery & technologies

Genentech licenses Sanegene’s RNAi asset in $1.7B deal

Feb. 3, 2026
By Marian (YoonJee) Chu
No Comments
Genentech Inc. is paying $200 million up front and up to $1.5 billion in milestone payments to license one of Suzhou Sanegene Bio Inc.’s RNAi programs. Metabolic and autoimmune-focused Sanegene did not disclose specifics around the licensed candidate, except that it was derived from its LEAD (Ligand and Enhancer Assisted Delivery) platform.
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Microscope with laptop displaying histology image.
Cancer

Blocking TET3 disrupts OTX2 effects in group 3 medulloblastoma

Jan. 15, 2026
No Comments
Medulloblastoma is one of the most common pediatric brain malignancies. A deeper understanding of the mechanisms underlying tumor cell fate determination is crucial to develop novel, effective and safe therapeutic strategies. With this aim, researchers from the University of Chinese Academy of Sciences and collaborating institutions recently conducted a study integrating whole-genome bisulfite sequencing and bulk RNA sequencing (RNA-seq) data from 189 human medulloblastoma samples.
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3D illustration of RNA and proteins
Neurology/psychiatric

Sarepta Therapeutics seeks clinical trial clearance for SRP-1005

Jan. 8, 2026
No Comments
Sarepta Therapeutics Inc. has filed a clinical trial application (CTA) in New Zealand seeking clearance to initiate a first-in-human trial of SRP-1005 (formerly ARO-HTT). Pending approval, the INSIGHTT trial is anticipated to begin in the second quarter of this year.
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Cardiovascular

Rona Therapeutics seeks clinical trial clearance for RN-5681

Dec. 18, 2025
No Comments
Rona Therapeutics Co. Ltd. has announced the submission of RN-5681 to the Australian Human Research Ethics Committee (HREC), and anticipates dosing to begin in a phase I trial in the first quarter of next year.
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Liver over digital lens background
Gastrointestinal

Bispecific siRNA knocks down YAP1, WWTR1 with single guide strand

Dec. 15, 2025
No Comments
Yes-associated protein (YAP), encoded by Yes1-associated transcriptional regulator (YAP1) and transcriptional coactivator with PDZ-binding motif (TAZ), encoded by WW domain containing transcription regulator 1 (WWTR1), are two crucial paralog transcriptional regulators of the Hippo pathway regulating organ size, tissue homeostasis and disease progression.
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Illustration of siRNA structure
Drug design, drug delivery & technologies

Peptidream reveals milestone in Alnylam collaboration

Dec. 15, 2025
No Comments
Peptidream Inc. has announced a preclinical development milestone in its collaboration with Alnylam Pharmaceuticals Inc. under their siRNA conjugate discovery collaboration.
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