Recent Articles in Cell & Gene Therapy

Sirius Therapeutics filed to begin a first-in-human clinical trial in Australia of SRSD107, its next-gen siRNA therapy aimed at preventing and treating thromboembolic disorders. SRSD107 is a novel double-stranded small interfering ribonucleic acid (siRNA) that targets coagulation factor XI (FXI) mRNA and inhibits FXI protein expression, blocking the coagulation pathway and promoting anticoagulant/anti-thrombotic effects. In preclinical in vivo tests, a single subcutaneous dose showed near 100% reduction of FXI levels for up to six months without bleeding events. Founded in 2021, Sirius has a discovery center in San Diego and a translational medicine center in Shanghai. More details....

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Suzhou Porton Advanced will partner with BioMap, an AI company that has developed the Xtrimo platform that offers Large Language Models to predict the behavior of proteins. The collaboration will use Porton Advanced's novel Adeno-Associated Virus (AAV) vector technology platform and relevant research data to develop AAV assembly efficiency models and an assembly distribution model. The two companies plan to build large-scale life science models that speed up the integration of experimental platforms and intelligent big data analysis platforms. BioMap is headquartered in Menlo Park, CA with operations in Singapore, Beijing and Suzhou. More details....

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Lerna Biopharma, a Singapore, Boston, Shanghai company, will present its lead drug, a first-in-class siRNA that promotes liver regeneration in patients at risk for liver failure. The candidate, LR1, takes advantage of the liver’s ability to heal itself, a long-sought goal. In preclinical testing, LR1 improved liver function and protected the liver from further degeneration. Lerna discovered the novel target using its Quartz platform, which discovers novel targets that are usually unavailable but can be addressed by siRNA technology. Lerna will describe the novel siRNA candidate at a Boston scientific convention, The Liver Meeting, later this week. More details...

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Celest Therapeutics (Shanghai) acquired China rights to a novel off-the-shelf CAR-NK cell therapy discovered by Senti Biosciences in a $156 million agreement. Celest will initially conduct a pilot trial in China for SENTI-301A, a multi-armed off-the-shelf CAR-NK cell therapy aimed at GPC3 expressing tumors. The engineered NK cells target the GPC3 antigen, which is highly expressed in 70% to 90% of HCCs with low or no expression on normal adult tissues. Celest will be responsible for paying up to $156 million in milestone payments, plus royalties. More details....

Stock Symbol: (NSDQ: SNTI)

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Argo Biopharmaceutical, a Shanghai RNAi company, raised $41 million in a Series A+ round to develop next-gen RNAi drugs for cardiovascular, congenital and immune system conditions. The company, founded by returnees, has experience in RNAi drug development including bioinformatics and nucleic acid sequence design, chemical modification, GalNAc coupling technology, extrahepatic delivery technology development, and oligonucleotide CMC. The financing was led by CS Capital, with participation from Huagai Capital and existing investor Trinity Innovation Fund. More details....

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