Gene editing – the practice of adding, removing, or altering genetic data in specific locations in the genome – is a relatively new area of genetic engineering that has demonstrated great therapeutic potential when it comes to treating or preventing genetic diseases.
One gene editing tool, CRISPR, has generated particular excitement in the scientific community due to its ability to easily alter DNA sequences and modify gene functions. Though we are yet to see an approved CRISPR-based therapy for humans, the technique holds great promise for the future of genetic medicine.
Spanish biotech Integra Therapeutics, founded in 2020, is building on this ground-breaking technology to improve the safety and effectiveness of advanced therapies – that is, treatments involving gene therapy, cell therapy and tissue engineering.
The company is developing a therapeutic gene writing platform that it says will overcome the current limitations with gene therapies. The technology, which combines CRISPR techniques with the gene transfer efficiency of viral integrases and transposases, can be used to paste both small and large DNA sequences into genomes with high precision.
CEO Avencia Sánchez-Mejías speaks to Pharmaceutical Technology about the young company’s plans for the future and how Integra’s gene editing platform could improve advanced therapies for patients.
Darcy Jimenez: Integra Therapeutics spun out of Pompeu Fabra University. What led to the company being formed there and how is it being built from those beginnings?
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Avencia Sánchez-Mejías: My co-founder, Marc Güell, started a lab at the university four years ago and I joined him to develop this technology. Once we had a good prototype that was performing quite well at inserting small and large payloads into the genome, it was a good time to incorporate the company and bring the traction we needed for this to become a therapeutic product.
DJ: Integra is creating new gene editing tools to make advanced therapies safer and more effective. What are the limitations of the advanced therapies currently being delivered to patients?
ASM: In many the diseases, the affected gene is relatively large. At the moment, if you need to insert a full-length gene or large coding sequence that does not fit into the current viral vectors, you’re left with no solutions.
Also, with the the viral vectors, such as retroviruses, that are currently used in the pharma industry, the gene is inserted in a non-controllable way. We wanted to have a machinery that we can program to insert only in the target area of the genome that we want. In that way, we can select a safe place to put the gene and then control its insertion at that location.
DJ: Can you talk me through how Integra’s platform works? What sets you apart from other established companies in the space?
ASM: With our platform, we combined the specificity of CRISPR-Cas9 systems with the efficiency and capacity of integrases and transposases that have evolved to write a large message into the genome. Our objective is to bring to the market the ability to do programmable transposition of large and small sequences.
We plan to do an analysis of which will be the best first indication we want to move into the regulatory phase. While it’s not settled yet, we will make a decision in the next year together with the funding partners.
DJ: The company recently raised €4.5 million in funding. What will this mean for Integra and its gene editing platform?
ASM: We are very proud that the investors trusted us and wanted to come on board to build this company. These funds will be used to finalise the prototype and do its final development which we will move into the regulatory phase. They will also be used to generate strong proof-of-concept data in relevant animal models. And thirdly, it will allow us to manage our intellectual property (IP) portfolio.
DJ: What does Integra have planned for next year? How do you envision the company’s platform impacting the advanced therapy space in 2022?
ASM: We want to show what the platform is capable of doing in a relevant therapeutic setup. For now, that will be in animal models, but we envision ourselves being used as a reference in the ecosystem for advanced therapies.
For next year, we will focus on having a really strong proof of concept and a final prototype, and then probably in a few years, work to get approvals to do our first in-human clinical trials.
Cell & Gene Therapy Coverage on Pharmaceutical Technology supported by Cytiva.
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