Delivering the goods

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Biotech firm Nanogenics believes that its product LipTide could solve one of the oldest problems in gene therapy – delivery. Director of business development Alan Walker and director of pre-clinical development Simon Newman talk to PharmaTimes about their vision for the company and LipTide

What is the concept behind LipTide?
Alan Walker: We’ve cracked the human genome and gene editing is starting to come in, but delivering a gene to exactly the right place and making sure it doesn’t go to any wrong places is the missing link that still hasn’t really been solved. There are lots of problems with just going in and changing people’s genomes if it’s not highly-targeted.
LipTide, though, is highly targeted because it has this particular sequence of amino acids on the end of the peptide sticking out of the nanoparticle –they are targeted to very specific receptors on very specific cells.
That’s our USP, and that’s excited many potential clients from big pharma down to smaller companies. We’re getting lots of interest in the product because this targeting aspect overcomes many problems and opens the door to effective gene therapy.
Simon Newman: The original concept for LipTide actually originated from the idea of creating an artificial virus. Viruses themselves are brilliant for getting into cells and delivering their nucleic acid into that cell. So it started out as nucleic acid wrapped in peptide-targeting receptors and this was very good at delivering the payload into the cell. The problem was that it wasn’t then released once inside the cell. That’s where the small lipid component of LipTide plays a crucial role as it allows the particles to escape the endosome. So we like to think that, despite the name, LipTide is more of an artificial virus. It certainly isn’t a standard liposome dressed with a little peptide. The product is primarily peptide, like a virus.
The other really exciting thing about LipTide technology, and the reason we’re getting a lot of interest from some of the blue-chip pharmas, is that with current viral technology you’re limited to DNA-only payloads with a size limit of about 12kb, but we can deliver in excess of 50kb of DNA or RNA. I think it’s that, combined with the ease of manufacture and the payload flexibility, which is driving a lot of interest in LipTide. Hopefully we can play a part in unlocking the pent-up demand around gene therapy, because a lot of companies have some great ideas about what they want to do but are still held back by the limitations of viral delivery systems.
Walker: Viruses have had millions of years to evolve but in response the body has developed a highly-efficient immune system, so if you go the viral route everyone is challenged with the normal basic immune responses of the body. One of the other great advantages for Liptide is that it allows for repeated delivery over periods of time with no apparent immune response – and we’ve shown that in animal models – so you overcome that basic problem.​
What are your backgrounds?
Walker: I’ve been in the pharma industry for 48 years, mostly in commercial roles, and Simon has come from an academic research and charity background.
We are both employed by a company called Ryboquin, which owns a gene called ITCH that, when silenced, can enhance chemotherapy by up to 400 percent. So this product, ECP-102, is quite interesting, but we had the same challenges with delivery that we described earlier. So we licensed LipTide from a company spun out of UCL. And we thought, why don’t we acquire the technology, because it had been developed for a while but hadn’t turned itself into a big biotech company. We’re actually just closing the acquisition and Ryboquin is becoming the new company, Nanogenics.
Our strategy is twofold – we are going to continue to develop our own products using the LipTide platform, like the chemo-sensitiser, ECP-102, and in order to fund that development, especially as we get into the clinic, we will make the LipTide platform widely available to enable these therapies to work for many companies. So we are now marketing very actively for the first time in the history of this platform, which beforehand was quietly being developed at UCL.
We see it as a tremendous opportunity because it’s opening the door for new therapies and there’s a tremendous inbuilt demand for such a targeted product, and the need is clear. It’s hard work because there are only two executives, Simon and myself, in the company at the moment. But we have some significant backers who see this potential and want us to continue to develop the technology, not just for commercial reasons, but also because they see that solving these diseases is a tremendously humane thing to do.
Which diseases areas could the technology potentially be useful in?
Newman: One of the most promising is cystic fibrosis. We know which gene is mutated and what the mutations are. We can see quick progress in that area because so much of the biology is already in place and it’s now all about getting that delivery right.
The great thing about Liptide is that some of the applications are being done through IV administration, but some are equally effective by airway delivery: nebulisation. That’s an approach we’re taking to cystic fibrosis.
Walker: CAR-T technology in cancer treatment is another therapy area we are interested in. We’re in discussions with several companies who can see that Liptide might offer quite a major advantage there. CAR-T technology is extremely expensive, and it’s not without its own risks. If you can make the changes to the T-cells much more targeted and specific – outside the body to start with, but maybe even long term inside the body – both the cost and in theory the dangers could be avoided.
What could the wider implications on the industry be?
Newman: It could be quite dramatic. Something we’re just coming across is that, because of the current limitations of delivery, there are a lot of companies that are beginning to only focus on diseases where viruses can go, so the limitations of viral delivery is beginning to risk driving companies’ therapeutic programmes.
With LipTide we might be able to open it up again and say gene therapy isn’t just for rare diseases – the optimism that was there 20 years ago can be realised, gene therapy can be used for more mainstream diseases as well. That’s the longer term goal, and hopefully LipTide can play a part in delivering that.
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http://www.pharmatimes.com/magazine/2017/november_2017/delivering_the_goods
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