The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has opened a consultation on changes it is proposing to the legal definition of gene therapies, to reflect the advances in technology and manufacturing over the two decades since the current legislation was drawn up. The aim is to correct the mismatch between regulatory terminology and modern science, which MHRA says “can lead to uncertainty” over how a product is classified and to “inconsistent oversight and potential barriers for developers."
Three decades of trial-and-error, and the resulting safety data, in the oligonucleotide-based therapeutic space have paved way for the present-day innovations and the promise of “programmable,” precision medicine for patients, speakers at Bio Korea 2026 said April 28.
Three years after Johnson & Johnson handed back rights to an siRNA candidate targeting a genetic driver of metabolic dysfunction-associated steatohepatitis (MASH), Arrowhead Pharmaceuticals Inc. found a new partner in Madrigal Pharmaceuticals Inc., a firm that markets the first U.S. FDA-approved MASH drug, Rezdiffra (resmetirom), and has been building a pipeline in the space.
Three decades of trial-and-error, and the resulting safety data, in the oligonucleotide-based therapeutic space have paved way for the present-day innovations and the promise of “programmable,” precision medicine for patients, speakers at Bio Korea 2026 said April 28.
Vivatides Therapeutics Inc. has closed an oversubscribed $54 million series A financing to advance its work in extrahepatic RNA delivery technologies. Proceeds from the financing will be used to further advance the company’s extrahepatic delivery platform and accelerate pipeline programs.
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.
GSK plc will pay Frontier Biotechnologies Inc. $40 million up front and up to $963 million in milestone payments to license two of Frontier’s small interfering RNA-based assets in the field of immunology.
GSK plc will pay Frontier Biotechnologies Inc. $40 million up front and up to $963 million in milestone payments to license two of Frontier’s small interfering RNA-based assets in the field of immunology.
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.
Researchers from Imperial College London and collaborators further investigated C/EBPβ as a target for MASLD and evaluated the efficacy of a C/EBPβ-targeted siRNA approach to reverse metabolic dysfunction and restore liver function in high-fat-diet-induced steatosis, which can be precursor to MASH and hepatocellular carcinoma.
