Epigenetic editing is a promising method for gene regulation in vitro and in vivo, allowing precise control of gene expression without altering the DNA sequence, thereby minimizing genotoxic risks.
A gene therapy based on an enhanced lipid nanoparticle (LNP) loaded with vascular endothelial growth factor (VEGF) mRNA could be developed for the treatment of preeclampsia, according to a study in mice in which it alleviated maternal hypertension until the end of gestation and improved fetal health. Preeclampsia is a disease that affects 3% to 5% of pregnant women, who suffer from hypertension and proteinuria, elevated levels of protein in the urine, during pregnancy.
Researchers from Moderna Inc. and affiliated organizations presented preclinical data for the novel mRNA-lipid nanoparticle (LNP) vaccine, mRNA-1769, against mpox virus.
Exsilio Therapeutics emerged from stealth mode on June 25, 2024, with $82 million from a series A financing that was co-led by Novartis Venture Fund and Delos Capital. The company plans to use naturally occurring, mobile genetic elements to integrate therapeutic genes at a defined location in the genome, making it safer than random integration, which can cause tumor formation.
Exsilio Therapeutics emerged from stealth mode on June 25, 2024, with $82 million from a series A financing that was co-led by Novartis Venture Fund and Delos Capital. The company plans to use naturally occurring, mobile genetic elements to integrate therapeutic genes at a defined location in the genome, making it safer than random integration, which can cause tumor formation.
Researchers from Poseida Therapeutics Inc. presented preclinical data for P-FVIII-101, a novel nonviral gene therapy being developed for the treatment of hemophilia A.
Generation Bio Co. has announced a breakthrough in its nonviral genetic medicine platform with the development of a proprietary, novel DNA called immune-quiet DNA (iqDNA).
Immetas Therapeutics Inc. and GC Biopharma Corp. have entered into a research collaboration to discover and develop novel mRNA therapeutics for the treatment of a broad range of autoimmune diseases. The collaboration combines Immetas’ proprietary platform for modulating innate immune pathways with GC Biopharma’s mRNA therapeutic and lipid nanoparticle (LNP) delivery platforms.
Messenger RNA, or mRNA, represents a relatively new class of therapeutics with the potential to prevent and treat a wide range of diseases. A well-known success story is of the mRNA vaccines that controlled the COVID-19 pandemic, which has fueled enthusiasm for the field. But biotechs are also developing mRNA candidates for several other infectious diseases, including malaria, tuberculosis and HIV, as well for cancer, autoimmune and allergic diseases. However, delivering nucleic acid therapeutics can be challenging, since mRNA cannot get into cells on its own.
