The story of a unique gene editing therapy company

Last May (2019) at the BCF Career Event NTrans Technologies has been awarded the BCF Scale-up Award. What is the story behind this upcoming life science business?

The story of NTrans Technologies starts in 2015 in the laboratories of the Hubrecht Institute of the Royal Netherlands Academy of Sciences (NWO). However, its gene editing technology dates back to the moment the research group of Prof. Niels Geijsen at the Hubrecht Institute and Utrecht University discovered that a combination of two small molecules (sodium chloride and NDSB-201) triggers the cells’ natural uptake process and is particularly suitable for the application of gene editing technology. Marco de Boer, co-founder and CEO of NTrans Technologies explains: ‘Our iTOP-technology is unique, extremely effective and very different from other methods. It is one of only a few methods available for the intracellular delivery of gene editing proteins.’

Niels Geijsen directly saw the potential of his invention in gene-editing for therapeutic purposes and patent applications were filed to protect the intellectual property (IP). With the intention to develop the technology further and explore its clinical potential, Geijsen went looking for someone with business experience in the life sciences. At that time, Niels Geijsen met with Marco de Boer who has received his MBA from the Nyenrode Business University, and a background in molecular biology. Geijsen and De Boer joined forces, and they founded the company together in 2015.

NTrans Technologies develops therapeutic gene editing applications to treat genetic disorders and cancer. De Boer: ‘We were fortunate to raise over €2.5M in non-dilutive funding, which allowed us to translate the fundamental discoveries into a clinically-applicable gene editing platform. Our proof-of-concept data demonstrates in vivo gene correction. Together with physicians and patient organizations we are now working to apply this in patiënts’

Immunotherapy to fight cancer

‘The concept works for different cell types, so new applications will follow soon’, expects De Boer. ‘We already have a proof-of-concept for iTOP in skeletal muscle and hereditary eye diseases. The technology has very local effects with long-term efficacy and safety, which makes it very suitable for these kinds of applications. On the other hand, we are also developing immunotherapies with ex vivo gene editing of T-cells. Sometime, people with cancer may be cured with their own modified immune cells. The T-cells are then first genetically changed with iTOP to induce the cells to attack the tumor. We are working with labs at the Princess Máxima Center for Pediatric Oncology to develop this technology further and test the anti-tumor activity of these altered T-cells on tumor organoids.’

According to De Boer, their method can be a solid alternative to viral delivery of CRISPR/Cas. ‘Without using viruses to insert a gene we think CRISPR/Cas can be much safer. Viral delivery increases the risk of unwanted cuts in the DNA, so-called off-target editing. iTOP delivers a limited amount of protein to the cell, which after performing its job, is rapidly degraded,’ he says.

‘Our technology is fast, efficient and without the risks associated with long-term expression of gene editing systems. This inherent safety makes it very appealing for therapeutic application. The speed at which these gene editing technologies are developing is astounding. It is both exciting and a privilege to be working at the forefront of the translation of these applications into meaningful therapies.’

Dit artikel is op 9 februari gepubliceerd op bcfcareer.nl

Foto: Marco de Boer

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