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BioWorld - Monday, April 20, 2026
Home » siRNA

Articles Tagged with ''siRNA''

siRNA bound to mRNA

Boehringer strikes deals with Ribo, 3T Biosciences with combined $2.5B value

Jan. 4, 2024
By Tamra Sami
Boehringer Ingelheim GmbH’s start to the new year includes two fresh deals across two continents. BI struck one deal with Kunshan, China-based Suzhou Ribo Life Science Co. Ltd. and its Mölndal, Sweden-based subsidiary, Ribocure Pharmaceuticals AB, to develop small interfering RNA (siRNA) treatments for nonalcoholic or metabolic dysfunction-associated steatohepatitis. It struck a second deal with San Francisco-based 3T Biosciences Inc. to develop cancer immunotherapies, which builds on an earlier collaboration formed last year. Combined, the two deals are worth more than $2.5 billion.
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DNA in drug capsules
Neurology/Psychiatric

Voyager selects lead development candidate for SOD-mutated ALS gene therapy program

Dec. 7, 2023
Voyager Therapeutics Inc. has selected a lead development candidate for its superoxide dismutase 1 (SOD1)-mutated amyotrophic lateral sclerosis (ALS) gene therapy program. The company anticipates filing an IND application for the candidate with the FDA in mid-2025.
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Digital spine concept art
Neurology/Psychiatric

Nurexone reports new siRNA sequences with potential for spinal cord injury

Dec. 1, 2023
Nurexone Biologic Inc. has reported results from laboratory tests of its secondary two proprietary sequences, showing promise for the treatment of spinal cord injuries.
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Endocrine/Metabolic

CSPC Pharmaceutical’s SYH-2053 receives NMPA clearance for clinical studies in China

Nov. 30, 2023
CSPC Pharmaceutical Group Ltd.’s SYH-2053 injection has obtained clearance from China’s National Medical Products Administration (NMPA) to enter clinical trials in China.
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Blue heart and data grid

Avidity, BMS sign $2.3B cardiovascular deal for new RNA class

Nov. 28, 2023
By Karen Carey
In a deal that could bring Avidity Biosciences Inc. $2.3 billion if all milestones are met, Bristol Myers Squibb Co. gained global rights to Avidity’s antibody oligonucleotide conjugates platform to advance up to five genetic cardiovascular targets. Avidity’s technology, which combines the specificity of monoclonal antibodies with the precision of oligonucleotide therapies, aims to address the root cause of diseases that are untreatable with current RNA therapeutics. Its lead internal programs are based on the targeted delivery of RNA into muscle.
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3D heart in chest
Cardiovascular

Activity of novel GalNAc-siRNA option in models supports advancement into the clinic

Sep. 1, 2023
Apolipoprotein C3 (APOC3) is one of the main regulators of triglyceride metabolism. Hypertriglyceridemia (HTG) is a major risk factor for cardiovascular disease and there is evidence that loss-of-function mutations in APOC3 correlate with decreased plasma triglyceride levels and subsequent reduced cardiometabolic dysfunction.
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Red blood cells illustration.
Hematologic

Agios obtains license to novel preclinical siRNA targeting TMPRSS6 from Alnylam

Aug. 3, 2023
Agios Pharmaceuticals Inc. and Alnylam Pharmaceuticals Inc. have entered into an exclusive worldwide license agreement under which Agios will acquire the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting TMPRSS6, as a potential disease-modifying treatment for patients with polycythemia vera (PV).
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Dtx brings siRNA candidates to Novartis in $1B merger

July 17, 2023
By Lee Landenberger
Novartis AG just added muscle to its siRNA development effort by buying Dtx Pharma Inc. in an M&A deal that could total $1 billion. Novartis is getting a preclinical asset, DTx-1252, which just received the U.S. FDA’s orphan drug designation on June 8. Dtx is developing the siRNA candidate for treating the neuromuscular disease Charcot-Marie-Tooth disease type 1A (CMT1A). Basel, Switzerland-based Novartis will pay Dtx $500 million up front and make payments of up to $500 million in milestones completions.
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Infection

siRNA PD-L1 inhibition leads to HBV infection clearance

June 28, 2023
One of the characteristics of chronic hepatitis B virus (HBV) infection is T-cell exhaustion, which, in turn, is mediated by the PD-1/PD-L1 axis. Researchers at Oligo Therapeutics Inc. reported on ALG-072571, a potent siRNA PD-L1 inhibitor active against HBV infection mouse models.
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Transmission electron micrograph of hepatitis B virus particles
Gastrointestinal

RBD-1016, a GalNAc-siRNA therapy effective in hepatitis B models

June 23, 2023
At the ongoing European Association for the Study of the Liver meeting, researchers from Suzhou Ribo Life Science Co. Ltd. presented preclinical data on RBD-1016 in two models of chronic hepatitis B virus (HBV) infection.
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