No sign of stopping: The rise of cell and gene therapy


Ryan Leahy, a science communication expert in cell and gene therapy, at Phacilitate, says there is a lot to celebrate but also much to assess, question and challenge in this sector.

While the global economic landscape faces a major crisis and biotech is not recession-proof, cell and gene therapy (CGT) has continued to progress with acquisitions, breaking ground on new facilities and approvals across the year – not limited to the breakout of the world’s most expensive drug. 

There’s an element of stability that CGTs must achieve to encourage developers, investors, and manufacturers to take the jump to back them. Large pharma involvement in advanced therapies has seen ebbs and flows, with AstraZeneca taking a larger stake in the market and Takeda developing its automation and digital capabilities, but also with GSK letting go of its CGT projects, along with most of the staff involved in its R&D.

The cost

2022 has not been shy of approvals – Uniqure and CSL’s Haemophilia B drug even nabbed the title of “Most Expensive Drug” at $3.5 million per dose. But are these price tags a barrier to access? Simply, yes, as we saw when the European Commission rejected the price point for bluebird bio’s Zynteglo. Its subsequent success with approval in the US shows us that some drugs will be unavailable to some markets and patients for now. The fact remains that the science behind the drugs is sound, there is direct patient need, and the medical benefits are pertinent. In the case of Hemgenix, the assurance behind the price point is that the drug would reduce long-term healthcare costs as patients would have fewer healthcare incidents and need fewer clotting treatments. For the first gene therapies, prices are high but these reflect costs and are in line with the benefits – and when the three most common haemophilia B treatments have a lifetime cost of upwards of $20 million per patient, it’s easy to see this price/benefit relationship.

Biotech and pharma are actively working with payers and insurers to deliver on equitable pricing models that ensure access. It is imperative that these payment processes are supported to expand access. From private plans to government programmes, there are workable systems that support CGTs, proven by MediCare’s support of CAR-T and the UK’s National Health Service coverage.

Patient access

A study on the socioeconomic backgrounds of CAR-T patients showed that children from across the spectrum of treatment for acute lymphoblastic leukaemia received similar results. You could thus infer that in some cancers, CAR-T treatment may be the great equaliser, which is why access should be more widespread and costs of goods, and eventually cost of drugs, must be brought down. 

CGTs are classified as ‘living drugs’ and are much more complex entities than other biologics. The reliance on variable and unstable patient materials is a lynchpin of cell therapy drugs, so maintenance of critical quality attributes becomes paramount, and the variability of a product must be vetted throughout the entire lifecycle. Closed and automated systems are an oft-cited solution to the problems of process variability and contamination, and even under the particularly delicate therapeutic modalities of CGT, technical innovation has come along leaps and bounds over the past 10 years. 

Looking forward 

Automation might be the solution to the lack of skilled personnel. Certain CDMOs that are giants outside of the CGT space regard it tepidly, but creative solutions are never far behind in our industry. For instance, apprenticeships are on the rise as technical areas such as bioprocessing become more reliant on unconventional candidates, including training of personnel without a biologics or life science background. 

There are questions regarding whether enough doctors are able to recommend advanced therapies, as well as where to send patients to receive treatment. This tracks with a similar lack of public, medical and government knowledge about the availability, progress, and effectiveness of CGTs. This is something the network of CGT developers will have to consider going forward. 

Advanced Therapies Week highlights the dynamic landscape of CGTs. The scope of advanced therapies for the next five to 10 years is a bright but relatively unforgiving road.Public awareness of these therapies will accompany a surge of new approvals, while manufacturers and developers look to bring down costs – to a contrary extent – to the level of advancement and innovation for new medicinal products. 

DDW Volume 24 – Issue 1, Winter 2022/23 – Cell and Gene Therapy Guide

Ryan LeahyAbout the author:

Ryan Leahy is a science communication professional and Vice President of Research and Head of Content for Phacilitate. He is engaged in the increase of effective scientific communication for a variety of stakeholders and populations to advance the development of and access to cell and gene therapies.

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