Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York. The agency is trying to set up cell and gene companies for success and that’s a very different agency than what it was years ago, said Paul Bresge, CEO of Ray Therapeutics Inc.

While the size of the market is enormous, drug development and treatments for women’s health care still lag behind what is offered for men. There has been a renaissance in the past few years, however, led by investors and companies that have wrestled with determining exactly what encompasses women’s health and how to meet its challenges.

Nuclear medicine combines structural and functional imaging, thus detecting lesions earlier than traditional methodologies. CD147 is a transmembrane glycoprotein that is expressed at very low levels in normal tissues, but significantly overexpressed in tumoral tissues, and is tied to clinical outcome and immune infiltration in cancer.

Researchers from Vrise Therapeutics Inc. and Vegen Therapeutics Pvt Ltd. presented preclinical data for VRTX-531, an allosteric inhibitor of ubiquitin carboxyl-terminal hydrolase 1 (USP1), being developed for the treatment of cancer.

Pancreatic ductal adenocarcinoma (PDAC) has a low 5-year survival rate of <12%. Even though KRAS is mutated in about 88% of PDACs, the KRAS G12C mutation is rare, limiting the use of KRAS G12C inhibitors. Hence, there is a need for pan-RAS inhibitors to cover the broad RAS mutation spectrum in PDAC.

Gene editing strategies, from epigenetic engineering to cell reprogramming and genetic vaccines, are accelerating the development of new therapies that awaken the immune system to treat cancer, as presented last month in Rome at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). Some of these advances are taking advantage of the conditions of the tumor microenvironment, where cancer cells coexist with immune cells, microorganisms and blood vessels.

Scientists from INSERM and affiliated organizations presented findings from their work that aimed to investigate the involvement of heat shock protein 27 (HSP27) in the activation and pathological accumulation of parietal epithelial cells (PEC) during crescentic glomerulonephritis (CrGN).