Inscripta… is developing a high-throughput, multiplexed genome editing platform that will, Ness asserts, allow scientists to perform tens of thousands of insertions, deletions, and swaps in one experiment.
“The traditional CRISPR/Cas9 approach performs one edit, querying one area of the genome, one edit at a time,” Ness notes by way of contrast. “That’s a very limited, laborious way to interrogate a genome or to create a specific cell.”
“CRISPR and gene editing tools are very powerful,” says Andre Watson, founder, chairman, and CEO of Ligandal. “But you still have to get the edits into the right cells.” Otherwise, gene editing won’t make the transition to gene therapy. Ligandal uses ligands to form nanoparticles and package genetic materials without viruses, causing cells to “eat” (endocytose) the nanoscale packages and deliver precise genetic instructions to target cells…the goal is to predictively manufacture optimized nanoparticles for given cell types, payloads, and therapeutic applications. In preclinical studies, Ligandal’s technology has successfully targeted specific cells in the blood and bone marrow. It appears to have applications in a wide range of gene therapies.