A new strategic partnership between APC, the National Institute for Bioprocessing Research and Training (NIBRT) and UCD is aimed at improving the availability of revolutionary new gene therapies to patients in Ireland and across the world.
Co-funded by Research Ireland and APC, an Irish biopharmaceutical process development and manufacturing company based in Cherrywood in Dublin, the €2 million Trans-Am project (Transformation of Advanced Medicines Manufacture) aims to identify more efficient and effective ways to produce and analyse gene therapies to deliver cost efficiencies for patients.
Gene therapy has made extraordinary advances in recent years and frequently provides life-changing benefits to patients. Such therapies are designed to replace faulty genes, and in some cases the patient is effectively cured by a single treatment or with just one or two repeat doses.
Ironically, that efficacy can create problems. As Pro Niall Barron, the lead principal investigator for the project, explains, “a single dose of gene therapy can often be a cure, but it is still a challenge to figure out how you compensate producers”.
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In other words, the massive costs involved in developing and manufacturing such a medicine would translate into enormously expensive prices per dose. That’s where the Trans-Am project comes in.
“APC is an Irish company that works with global pharma companies to help them figure out how to make medicines safely and reliably, to a high quality, and at lower costs,” says APC chief technology officer and co-founder Prof Brian Glennon. “Gene therapy can almost seem like science fiction it’s so extraordinary. It harnesses incredibly complex science, and the medicines take a very long time to develop and get to market.”
The company is in the business of lowering manufacturing costs. “We work with medicines that when they come to us can cost millions of euro for a single dose,” Glennon adds. “We bring it down to thousands. That’s still a lot of money but the cost reduction is very important in making those medicines available to patients.”
Gene therapy manufacturing is extremely complex and costly. The therapies are delivered most efficiently by viruses which are made using living cells. “Chemists can’t synthesise gene therapies; we need living biological systems to manufacture them,” says Barron.
The complexity arises because the manufacturing process involves getting cells to do something that is fundamentally against their nature. “We are asking them to make viruses that contain a piece of genetic material that will cure cystic fibrosis or haemophilia or other conditions,” says Barron. “But cells and viruses don’t get on with each other. Cells don’t like making viruses.”
On the other hand, viruses are very good at injecting genetic material into cells. “They have spent millions of years figuring out how to do it,” he notes.
Glennon explains that until now APC has been able to hire people with the skills it required, but that is not really possible with advanced therapeutics. “Through Trans-Am we will harness the expertise of NIBRT and UCD to support our international expansion,” he says.
Trans-Am’s research aims to identify more efficient and effective ways to produce and analyse gene therapies while at the same time training a new cohort of scientists and researchers to support Ireland’s biopharma industry in the future.
“Trans-Am is focused on manufacturing, not developing the therapies,” says Barron. “We will look for the most efficient ways to make them. When we ask a cell to make a virus, we look at what defence mechanisms it comes up with and how we can counter that. We have PhD students looking at changes happening in cells and then potentially coming up a with a new cell line without the defences. We are also developing technologies to measure the quality of what’s made to give us confidence that it will work and be safe as a product.”
Consistency is absolutely critical to the safety of the products. “You need to make sure that every time you make it, it’s the same,” says Glennon. “You have to be able to detect impurities right down to the level of a few parts per billion. The patients receiving the medicines are very sick and need extraordinarily high levels of purity. We are pushing the boundaries of analytical science.”
Trans-Am will also support Ireland’s broader biopharmaceutical industry. “We want to build on the success of the industry and ensure that advanced therapeutics are part of the story,” says Glennon. “This project is important to APC, but it will also build Ireland’s reputation as a place where you can do this kind of work. We want to help to ensure that Ireland has a seat at the top table when it comes to attracting FDI and supporting indigenous industry in the manufacture of gene therapies.”
