The U.S. FDA gave its go-ahead for Hemgenix (etranacogene dezaparvovec-drlb), Uniqure NV’s one-time gene therapy – the first for the treatment of adults 18 and older living with hemophilia B. Patients have been waiting “maybe beyond two decades” for a new therapy, Uniqure CEO Matthew Kapusta said. Hemgenix emerged from pioneering work by St. Jude Children’s Research Hospital and the University College London. Read More
Nearing the end of 2022, the COVID-19 pandemic clearly no longer dominates the clinical activity reported by biopharma companies. Overall clinical data tracked by BioWorld is down significantly, as is the proportion focused on the SARS-Cov-2 virus. Therapeutics and vaccines targeting cancer, infectious diseases and neurological disorders, however, remain a stronghold. Read More
Avenue Therapeutics Inc.’s takeover of Baergic Bio Inc. pursuant to the previously disclosed share contribution agreement with its parent company, Fortress Biotech Inc., highlighted the potential of targeting GABAA, an approach under investigation in various quarters. Read More
The companion diagnostic (CDx) has been a mainstay of oncology care for several years, but Richard Pazdur, director of the U.S. FDA’s Oncology Center of Excellence, said recently in a public forum recently that the notion of a single CDx for an investigational drug has not served patients well. Read More
A combination of radiation therapy and CD47 blockade induced an abscopal effect in animal studies even in animals that lacked T cells, researchers reported in the Nov. 21, 2022, online issue of Nature Cancer. The findings are “the first demonstration of T-cell-independent abscopal response,” co-corresponding author Edward Graves told BioWorld. “We’re not trying to say that all abscopal responses are macrophage-mediated. There are plenty that require T cells,” Graves clarified. But “there is another avenue of abscopal responses that has not been reported. ... All the abscopal literature is about stimulating an adaptive response.”
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BioWorld's offices will be closed in observance of Thanksgiving in the U.S. No issues will be published Thursday, Nov. 24, or Friday, Nov. 25. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Arvinas, Biosight, Intrabio, Khondrion, Merck & Co., Morphic, Oculis, Telix, VBI. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: AB Science, Argenx, Gamida, GSK, Innocare, Intelgenx, Junshi, Marker, Palisade, Regeneron, Revive, Takeda. Read More
