Harnessing the Biological Power of Cells to Deliver a Therapeutic Product

Could you start by introducing yourself and describing your role within the industry?

Hi, Ryan. Thank you for the opportunity to again, talk about my passion, which is the cell and gene therapy field, the fantastic things we’re doing for patients and the value that we’re harnessing.

My role in industry has two sides. First, as a CEO of biotech companies in this field – I’ve worked with three different technologies at three companies, and I’m now participating in a VC group in Paris, AdBio, where we’re trying to bring a very interesting technology to market with gene-modified cells for a therapeutic purpose for a company to be created early next year, which is currently still in ‘start’ mode. So that’s the day job, I would say, but again at the same time something that I am very keen to do is help the field and foster the field in general.

I have roles in some of the organizations that contribute to that. On one side as a board member on the Executive Committee of the Alliance for Regenerative Medicine, but also with ISCT, the International Society of Cell and Gene Therapy, where I’ve been involved for a long time, mostly fostering the industrial community, and making sure that we bring the society to the current state of the field, which can mean taking academic ideas into translational steps to bring products to patients. I have had the pleasure to have been elected recently, President Elect of ISCT, which gives me the mandate to do such things, which is really, I would call it, industrialize the society and contribute to the field.

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What are the biggest challenges in cell and gene therapy that you’re thinking about right now?

The cell and gene therapy field is booming, in that it’s really becoming an established field with a well-defined business opportunity, with a lot of companies, a lot of clinical activity across the globe, and delivering a lot of value to patients. Everybody recognizes this as an established field, but it is a very young field, with several challenges and things that require adaptation to the technology.

Probably one of the major challenges is harnessing the industrial aspect. I tend to say that we’re currently in the ‘industrial revolution’ where we’re able to manufacture products faster, cheaper, more effectively, more consistently, and that is enabling us, but there’s also still a lot to be done in this aspect, in terms of the manufacturing.

Also, because the value of the products that we’re bringing to the market provide a significantly longer-term value, either because they correct the gene deficiency or because they are so effective they nearly cure cancer, as we’ve seen with some very significant impacts on disease recently. We need to reconsider how these products are used in the market, and so the challenges is to get people comfortable, ready, and knowledgeable to use these products.

We’re making a lot of progress there, but also a significant challenge is enabling patients to get access. We’ve seen the difficulties regarding the payment and reimbursement of these products, namely, in Europe we’re seeing a slightly different perspective in the US. But overall, I think all of us – the developers, supporting organizations, clinicians, patient bodies and payers – need to look at this, see the benefit that we’re bringing to patients and find alternative solutions to ensure the access to patients. We will continue to see discussions on this as we go to bigger and bigger populations, but we need to adapt the models all the way from development, reducing the costs up to the moment of payment, bringing new models of harnessing the value, maybe spreading the payment, maybe finding joint risk taking opportunities, or we need to find ways to adapt to the new value these products are bringing and ensure the patients get access to them.

What are you particularly excited about in the cell and gene therapy space?

I’m very excited about 2 things: the fantastic science we’re doing, and the value that is bringing to patients. What we’re now doing is engineering cells for a therapeutic purpose.

Now, I mean pharma/biotech has always been about modifying biological systems to have an impact for therapeutics. In the end it’s the cells that will deliver or get affected by that therapeutic effect. What we’re now able to do is say, ‘I would like these cells to do this in that place’, and so we’re able to engineer the cells to target them, engineer the cells to be less recognized, engineer the cells to potentiate their efficiency. So we’re engineering cells ex vivo, or in vivo, for an increased targeted therapeutic effect with long term benefit. That’s very exciting. It’s the next wave of medicine.

As I mentioned to you before, what I’m now doing is bringing a couple of technologies from the US to a couple of countries in Europe to bring them together into a new company that will take a specific cell type, and we’ll improve the targeting of that cell through gene modification, improve the efficiency and the potency of that cell, again, through gene modification. It’s this engineering of the cells, this harnessing of the biological power of the cells and magnifying it through gene editing that delivers a therapeutic product with increased benefit, that makes me so excited about the field.

Fantastic. Yeah, it sounds extremely exciting. So why are you looking forward to at Phacilitate’s Advanced Therapies Week in January?

The meeting has been kind of a tradition for me and for people in the cell and gene therapy field where you go to talks, meet your peers, make sure that you’re able to share your experiences. Get their experiences, learn what the others are doing.

It’s a growing family. It’s got quite a lot of people now, but still keeps the familiar aspects.

We’re able to network, and we’re able to all get together. I mentioned to you just before, my excitement about what I’m doing, about what we are doing, and I think it’s a moment where we can all get together and share that that that excitement.

So for me, it’s really the moment where we go to the US and have that opportunity early in the year, which gives this perspective what we’re going to see in the year to come. With Advanced Therapies Europe in the fall as well, there’s a certain cycle of being able to talk to people, being able to see what’s new and forecasting what we’ll be doing over the next few months.

It’s a tradition for all of us, and I look forward to it again!

Don’t miss Miguel’s session, ‘Decentralizing Advanced Therapies: Are We Moving Closer to Bedside Therapeutics?’ and workshop, ‘Investment in CGT – Securing Funding for your Biotech Venture’ on Thursday January, 19 2023.
Join us at Advanced Therapies Week >>