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.
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.
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.
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).
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.
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.
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.
Sirnaomics Ltd. is advancing lead siRNA candidate STP-705 for the treatment of squamous cell carcinoma in situ into late-stage clinical testing after sharing encouraging phase II trial results with the U.S. FDA in an end-of-phase II meeting. The company plans to move forward in 2023 with a single-dose study as a subgroup of subjects in a large phase III study. Positive results will provide the basis for completion of this large registrational phase III trial.
Comanche Biopharma Corp. has received IND clearance from the FDA to conduct a first-in-human study of CBP-4888, its novel, small interfering ribonucleic acid (siRNA) therapy to treat pre-eclampsia.
