Advanced Solutions for Gene Therapy Development with Live Q&A

Gene therapy has evolved significantly, with more therapies approved in recent years. So far, the FDA has approved over 43 gene and cell therapies, which have impacted considerably disease outcomes.

Non-viral gene editing, such as CRISPR, has emerged as a pioneering technology for developing gene therapies. CRISPR is a revolutionary gene-editing technology that allows scientists to modify genes with unprecedented precision and ease. Non-viral gene editing has several advantages over viral-based gene editing, including a lower risk of immune system reactions and less potential for the genetic material to integrate into the patient's DNA. New CRIPR-based methodologies with increased specificity, such as Base and Prime editing, have recently started gaining traction.

Another promising trend is using an mRNA format of the CRISPR enzymes bundled with an LNP delivery system to efficiently edit genes without leaving lingering reagents and potentially introducing more non-specific edits. More stable and higher-expression formats, such as self-amplifying or circular mRNA, are gaining popularity.

In this presentation we will discuss the latest advancements in the area of CRISPR gene editing and targeted delivery solutions.