AccumTM platform greatly enhances CRISPR gene-editing technology

© iStock/Bill Oxford

Defence Therapeutics has announced that its AccumTM platform can potently enhance Cas9 delivery in target cells, significantly advancing CRISPR gene-editing technology.

The CRISPR technology offers the potential to transform medicine, enabling both the treatment or prevention of many illnesses by deleting or inserting genes in a targeted manner. CRISPR is central to the biotech industry as it can be used to treat multiple illnesses, such as cancer, viral diseases (HIV and Hepatitis B), and several genetic disorders.

The essence of this technology is simple and requires two important tools. Firstly, a piece of genetic material to guide the editing process, and secondly, the co-delivery of a Cas protein to cut/edit the DNA. Of all the Cas proteins currently identified, Cas9 is the most widely used by scientists as it can easily find and bind to almost any desired target sequence simply by giving a piece of RNA to guide it in its search.

Delivery of the Cas9 protein instead of a DNA-encoding sequence gives the advantage of decreasing the off-target activity of Cas9. Unfortunately, the current and existing protein delivery tools available today are inefficient at delivering proteins inside the nucleus of target cells. More specifically, any delivered protein ends up being entrapped and degraded within small endosome-lysosome vesicles. Consequently, the pool of cytosolic Cas9 is low in magnitude, making it difficult to reach the nucleus for efficient DNA editing activity. As such, multiple deliveries have to be attempted to reach enough nuclear Cas9 material.

The AccumTM platform is designed to accumulate a given molecule in target cells. The Defence team has tested AccumTM‘s ability to improve Cas9 delivery to cells. With only a single AccumTM dose and without any added use of a complex lipid formulation, the obtained results are astonishing. The accumulation of AccumTM-linked Cas9 inside the nucleus of the cells was at least 9-fold higher than the free Cas9 when added to mammalian cells in vitro.

Sébastien Plouffe, the CEO of Defence Therapeutics, said: “We successfully provide another strong example of how versatile and powerful AccumTM is for delivering proteins of crucial importance. This observation is key to Defence as it’s paving the path to strategic partnerships with major pharmaceutical companies in the field looking to improve their CRISPR/Cas9 editing approach.”

With such impressive data in hand, Defence will actively be looking to partner with major players in the field in order to move forward on CRISPR-based treatments designed for specific indications.

As per the report published by Fior Markets, the global gene editing market is expected to grow from $4.2bn in 2020 to $13bn by 2028, at a CAGR of 15.2% during the forecast period 2021-2028.


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