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.

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.

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).

The c-MYB oncogene plays a key role in hematopoietic cell differentiation and proliferation. Genetic abnormalities and dysregulation of MYB have been found in several cancers, including adenoid cystic carcinoma (ACC) (80% of cases), making it an attractive druggable target for ACC treatment.

PARP1 is critical for repairing DNA single-strand breaks. First-generation PARP1/2 inhibitors have proven effective in the treatment of tumors with mutations in the essential homologous recombination repair (HR) genes including BRCA mutations. However, hematological toxicity associated with PARP2 emphasizes the need to find second-generation compounds with better safety profiles.

Researchers from Astrazeneca plc recently reported preclinical data for AZD-3470, a second-generation MTA-cooperative PRMT5 inhibitor currently in early clinical development for the treatment of patients with MTAP-deficient solid tumors (NCT06130553) and hematological cancers (NCT06137144).

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.