What Might Stop the Cell and Gene Therapy Industry from Reaching its Full Potential?

Cell and gene therapies (CGT) are an innovative form of medical treatment that uses cells and genetic material to fight disease. Cancer, cycstic fibrosis, and diabetes (to name just a few) can all be treated by CGT, and as it stands, outcomes are promising. The potential is driving scientists to look for innovations in the field to make medical breakthroughs. The demand for CGT is ripe, and the space is primed for significant growth: BCC Research expects the market to grow at an eye watering CAGR of 33.6% to reach $17.4 billion by 2026. But if the industry wants to fully realize this potential, it must first overcome some huge hurdles.

High manufacturing and treatment costs

While it's not the only medical field that incurs some seriously high costs, CGT demands a lot of monetary investment that could stop the treatment becoming widespread. CGTs are personalized for each patient and have an unusually complex manufacturing process. Covering these costs isn’t affordable for most patients – nor companies that are required to fork out huge upfront investments.

Currently, most cell therapies are produced one dose at a time, unlike the traditional ready-to-stock approach. This makes it difficult to estimate the patient pool and increases the complexity in demand and capacity forecasting. Not only this, but the manufacturing requires quality testing of each individual product instead of a whole batch. Subsequently, this is highly labor-intensive, prone to failure and difficult to scale with manual methods.

These overheads hinder the access and affordability of CGT therapies in both developed and developing countries. Let’s give a concrete example: The first two approved CAR-T-cell therapies, Kymriah and Yescarta, are priced at $475,000 and $373,000 respectively per treatment in the US. Unsurprisingly, the inaccessibility is escalated in low- and middle-income countries. According to WHO, comprehensive treatment options for cancer are available in over 90% of high-income countries while it is available to less than 15% of patients in low-income countries. Even in high-income countries such as those in the European Union, there is constant pressure for cost-containment by the government and payors for these therapies. For instance, bluebird, which manufactures Zynteglo, a one-time gene therapy product for beta-thalassemia, costs $1.8 million. The company pulled the drug off the market in Germany due to pricing disagreements with the health authorities.

As a result of these sky-high prices, there’s a growing focus on cost-effective and scalable manufacturing for CGTs, which should hopefully address this issue in the future.

Lack of skilled workforce

Lack of qualified labor is another major bottleneck that could stop CGT from taking off. Cell processing, washing and feeding are complex and often repetitive processes involved in CGT therapies that require highly qualified workers. For example, a form of CGT called autologous therapy uses a patient’s own cells. It often requires weeks of work by multiple workers in a dedicated infrastructure. Plus, the operators, once recruited, may need additional training for a long period to attain the required expertise.

In 2018, the CGT Catapult launched The Advanced Therapies Apprenticeship Community with the support of the UK government. It is the first apprenticeship program designed to specifically train people in developing, manufacturing and delivering CGTs.

Growing advancements in CGT technologies

Despite the various factors that could limit the market, the outlook is still incredibly optimistic. Growing advancements in CGT technologies like the development of off-the-shelf products and application of advanced gene-editing techniques such as CRISPR will lower the complexities involved in manufacturing CGTs. This should help bring costs down and also address the skills gap issue.

CRISPR (clustered regularly interspaced short palindromic repeats) technology: CRISPR/Cas9 is a gene-editing tool technology with significant application in the CGT area. It can disrupt a disease-causing gene or correct and insert new genetic material. It has applications in drug discoveries and can help develop therapeutics for cancer, rare genetic diseases, infectious diseases and other conditions. The current approved CAR-T therapies are limited to only liquid tumors. However, companies are working towards developing CAR-T therapies for solid tumors using CRISPR technology.

Off-the-shelf solutions: Immunotherapies such as CAR-T therapies are personalized therapies that involve the collection of T-cells from patients (autologous) that makes the process complex. Most of the time, patients may have undergone multiple therapies such as chemotherapy previously and may have compromised immunity. To minimize the risks associated with autologous therapies, researchers are currently focusing on developing therapies using health donors that can be delivered as off-the-shelf products.

Companies such as Allogene Therapeutics are working on off-the-shelf CAR T-cell therapies using T-cells from healthy donors. This approach mainly helps companies minimize pricing issues with CGTs. It also makes these therapies readily available with improved potency and improves patient access and affordability.

Discover more about the cell and gene therapies market

The CGT market is growing rapidly. Clinical trials are increasing in numbers, stakeholders are upping investments, and personalized medicine for rare and chronic conditions is receiving a sharper focus. Having access to the full picture is vital for anybody operating within the industry.

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