Brian O'Connor of Orchard Therapeutics. Image: Paul Sherwood
Brian O’Connor, who leads quality organisation at London-based Orchard Therapeutics, tells us how recent trends are reshaping the pharma sector.
While research and development is a crucial aspect of developing advanced therapies in medicine, it is only half the work done. The other half, commercialisation, is what helps bring these innovative treatments to millions for better health outcomes.
And according to Brian O’Connor, the advanced therapy medicinal market, known within the industry as ATMP, has undergone “a transformational change” over the past few years, transitioning from a focus on research and development to commercialisation.
“The commercialisation of ATMPs has offered revolutionary treatment approaches to patients suffering from rare and intractable diseases, previously thought to be uncurable. Investment in ATMPs has significantly increased over the past few years, leading to over 2,000 clinical trials currently in progress,” he tells SiliconRepublic.com.
O’Connor is an executive at Orchard Therapeutics, a biotechnology company based in London that is building ATMPs to improve the lives of patients with genetic diseases through innovative gene therapy.
At Orchard, O’Connor leads quality organisation to ensure that all products and processes within the company meet regulatory standards, quality requirements and industry best practices.
He and his team maintain compliance to standards and expectations using risk-management principles and a quality-management system, ensuring the safety and efficacy of pre-clinical, clinical and commercial stage hematopoietic stem cell (HSC) gene therapies.
“HSCs are responsible for the life-long, sustained production of all blood cells and are ideal for personalised gene therapy because they have the capacity to self-renew, providing long-lasting therapeutic effects,” he explains.
“They also have the capability to differentiate into multiple cell types in the blood and they can migrate into various tissues and organs, enabling delivery of therapeutic genes to tissues and organs that are inaccessible to other therapeutic modalities.”
The impact of AI and challenges
As a veteran of the pharma and biotech industry at sites across the world from Ireland to Switzerland and Singapore, O’Connor is no stranger to change. And one such change that is having a palpable impact on his industry right now is artificial intelligence.
“AI is having a major impact on the discovery and development of novel drug entities. For example, AI can analyse vast chemical libraries of pharmaceutical companies to predict potential drug candidates that are likely to interact with specific disease targets,” he says.
“AI can also speed up drug design by generating novel chemical structures or optimising existing molecules. There are numerous other applications in use across the industry from the optimisation of clinical trials to quality oversight of manufacturing.”
Despite this, there are significant challenges the ATMP market faces in terms of manufacturing and quality assurance, O’Connor notes, including capacity, cost, scalability and compliance.
“With so many clinical trials in progress, the global capacity available at contract manufacturing organisations is low, also impacting the cost of manufacturing, along with the price of starting materials such as plasmid DNA,” he explains.
“Scalability is a challenge as the industry moves from bench-top like manufacturing, for ultra rare diseases, to larger scale bioreactors for bigger disease indications. Compliance to regulations can be challenging as regulatory guidelines related to ATMPs are continually under development or revision by global health authorities.”
While O’Connor and those in similar positions continue to find news ways to overcome these challenges, the ATMP sector in Ireland has been growing rapidly with increased investment across manufacturing and training facilities.
“Several established multinational companies have invested in constructing advanced therapy manufacturing facilities on their sites, including the recent approval of the first commercial advanced therapy product manufactured in Ireland,” he says. “In June 2023, the National Institute for Bioprocessing Research and Training (NIBRT) on the University College Dublin campus completed an expansion of their training and research facility, specifically for advanced therapies.”
Orchard Therapeutics, which was founded in 2015, established its first Irish presence last December after hiring O’Connor in his current role.
“Since then, Orchard Therapeutics has actively participated in the ATMP Forum of Ireland and lead discussions, as related to ATMPs, with the Parenteral Drug Association of Ireland events,” he went on.
“We will continue to promote and educate on ATMPs in Ireland and look forward to further establishing our presence here.”
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