Cell and gene therapy (CGT) is an experimental space that has grown exponentially in recent years; the sector received nearly $20bn in funding last year, a 50% increase on the record $13.5bn it saw in 2018.
The clinical potential of these advanced therapies is clear, and has already been demonstrated in several cases. Removing cells, proteins or DNA from patients or donors allows this material to be re-engineered and reinfused into patients, providing highly personalised, often single-dose treatments that have long-lasting therapeutic effects, offering the prospect of disease-modifying or even curative impacts in tricky indications.
In the last few years the first crop of CGTs has begun to emerge, often with eye-watering price tags attached. Novartis’s gene therapy Zolgensma – the most expensive drug in the world – targets the genetic basis of spinal muscular atrophy, while Luxturna, which Novartis licensed from Spark Therapeutics in January 2018 for markets outside the US, delivers the functional copy of the RPE65 gene to improve vision and block progressive sight loss, and has been FDA-approved since December 2017.
CAR-T cell therapies such as Novartis’s Kymriah and Gilead’s Yescarta, meanwhile, have emerged as premium one-dose options for the precision treatment of aggressive forms of leukaemia and lymphomas.
Help wanted: the CGT skills gap
While the impressive investments of the last few years have allowed the CGT space to evolve, its rapid growth has led to a skills gap that’s hindering the delivery of much-needed therapies to patients.
Less than a decade ago, the number of workers recorded in the advanced therapy field in the UK was a mere 500. According to a report by the country’s Cell and Gene Therapy Catapult (CGT Catapult), the UK’s workforce headcount in the field is now predicted to double from over 3,000 in 2019 to more than 6,000 in 2024. Despite this, the increased demand for skilled personnel in such a quickly evolving industry has become a significant obstacle for cell and gene therapy companies, with manufacturing reported as the skill area of greatest concern.
Project Farma is a consulting firm that provides biomanufacturing strategy and execution for the life sciences industry. The company’s director Phil Massey says “demand is outstripping supply in every sort of functional area” in the CGT field, which has seen a “paradigm shift” in recent years.
“In other disciplines like biologics or small molecules, the manufacturing is now a known commodity,” Massey explains. “We have facilities utilising established technologies such as large stainless-steel bioreactors and there is a great deal of automation already established.
“Switch to cell and gene therapy, where it’s personalised, or there are a lot of manual manipulations. The field just started to become industrialised about five years ago, and the manufacturing process is slower and less automated than the aforementioned other disciplines.”
The manufacture of cell and gene therapies – which can be highly personalised to patients and often target rare, hard-to-treat diseases – is a largely manual process that requires numerous skilled workers. The complexity of CGT manufacturing operations means that if companies are to scale up production and deliver therapies on a commercial level, a sizeable labour pool and extensive facilities are a must.
“Many of these therapies are personalised in nature,” Project Farma’s senior manager Daniel Foody says. “The batch sizes are so small that sometimes it doesn’t make sense to build a facility and do your own manufacturing.
“The manufacturing process is also more manual and labour-intensive. Therefore, different aspects of the manufacturing process become more challenging and require an enhanced level of monitoring and control.”
Scaling up UK skills capacity in CGT
In addition to the industry’s growing need for skilled professionals and capable facilities, the CGT Catapult’s report also revealed that advanced therapy medicinal products (ATMP) clinical trials across the UK rose by 20% last year, constituting an impressive 12% of all cell and gene therapy trials worldwide. The US has seen similar growth in the space; of the more than 1,000 cell and gene therapy developers worldwide, half are located in the US.
With the UK and US currently leading global CGT innova...
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