Gene Editing Startups Fabricate Industrial-Grade CRISPR Tools

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.


Delivery is a specific challenge for the CRISPR/Cas9 gene editing complex, too, Marco de Boer, PhD, CEO of Ntrans Technologies, points out. His firm has developed a virus-free cell targeting method called iTOP, which uses small molecules to induce the uptake of therapeutic proteins into the target cell. It may be used for in vivo or ex vivo applications.

Because the complex is broken down after a relatively short time, the risk of off-target effects is reduced. In addition, delivery in the form of RNP complex avoids the immune responses possible with viral delivery.”

Additional advantages include ex vivo and in vivo delivery of gene editing systems, virus-free delivery, cost-effective therapeutic formulation, consistency, GMP compatibility, and delivery of naïve, unmodified proteins.


Thank you @twinklestars .
You are always at the frontier of breakthrough science.
That’s a nice interest to have, and you make an immense contribution to the forum with your news posts.
I am less interested in science, I am more of a sports guy, but that’s OK I guess.
Considering that I am schizophrenic, I am glad that I found a niche for myself.


Science says you are doing great things for yourself by exercising. Keep up the good work!

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