In a deal potentially worth $810 million for Regenxbio Inc., Nippon Shinyaku Co. Ltd. is partnering on the U.S. and Asian development and commercialization of iduronate-2-sulfatase enzyme RGX-121 for Hunter syndrome and RGX-111 for Hurler syndrome.
Canary Cure Therapeutics (Canary Global Inc.) has released preclinical results for its lead candidate, CCT-217, an siRNA therapeutic targeting cannabinoid CB1 receptor (CB1R) and zinc finger protein 423 (ZFP423) to treat obesity.
Dyne Therapeutics Inc. is eyeing accelerated approval for its myotonic dystrophy type 1 treatment after reviewing new results from a phase I/II study. DYNE-101, an oligonucleotide antisense and DMPK gene modulator, produced results on disease biomarkers that included DMPK and splicing correction, disease progression reversal on several functional endpoints and a favorable safety profile. The accelerated approval submission could come in the first half of 2026.
New Anglo/U.S. obesity and cardiometabolic specialist Verdiva Bio Ltd. has launched with a massive $411 million series A and a portfolio of GLP-1 and amylin agonists in-licensed from China.
Alesta Therapeutics BV has closed an oversubscribed €65 million (US$67 million) series A financing round designed to support development of its two small-molecule therapeutics.
With obesity drugs taking the U.S. and global markets by storm and more than 100 clinical programs currently in progress for the drugs, the U.S. FDA released a draft guidance Jan. 7 to help sponsors develop drugs and biologics for weight reduction and long-term maintenance of body weight.
Sana Biotechnology Inc. CEO Steve Harr said “cool and transformative science” led to his firm’s latest strides in type 1 diabetes, and the “very generalizable [clinical] result” cheered Wall Street, which led shares of the Seattle-based firm (NASDAQ:SANA) to close Jan. 8 at $4.30 up $2.65, or 160.6%. “I believe we now have all of the pieces in place to move forward rapidly,” Harr said.
Sun Pharmaceutical Industries Ltd. has divulged incretin analogues acting as dual agonists of gastric inhibitory polypeptide (GIPR) receptor/GLP-1 or GLP-1/glucagon receptor (GCGR) or triple agonists of GIPR/GLP-1/GCGR reported to be useful for the treatment of type 2 diabetes, dyslipidemia, obesity, metabolic syndrome, neurodegeneration, fibrosis and cardiovascular disorders.
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