cell and gene therapy

Is cell and gene therapy still one of the 8 great technologies

5 years ago, regenerative medicine was lauded as one of the “8 Great Technologies” which would propel the UK to future growth. Has it stood the test of time?

Regenerative medicine, or cell and gene therapy as it also known, uses cells or genes in the treatment or cure of diseases. They can even use the patient’s own cells to fight diseases, so-called immunotherapy. Perhaps the best-known technologies are CAR-T (chimeric antigen receptor T) cell therapy and CRISPR (clustered regularly interspaced short palindromic repeats) gene editing. 

Alongside the 8 Great Technologies announcement, the UK government established a Cell and Gene Therapy Catapult to catalyse the discovery and development of new therapies. Since then the field has flourished. 

From gene therapy for Parkinson’s disease to growing new blood vessels to preventing limb amputations in diabetic patients, cell and gene therapies have unsurprisingly provided hope to a lot of patients. A few therapies have even made it to market.

Curing cancer with a single dose

In August 2017, Novartis received the first ever FDA approval for a CAR-T cell therapy. Kymriah, a single treatment immunotherapy, was approved for acute lymphoblastic leukaemia in children and young adults. Without the treatment, less than 10% of patients survive for five years.

Bursting the bubble

Earlier this year, NICE approved GlaxoSmithKline’s Strimvelis, a gene therapy treatment for the rare immune deficiency condition ADA-SCID (severe combined immunodeficiency due to adenosine deaminase deficiency). Children who have the disease are so susceptible to infection that they have to be isolated, giving the condition the nickname “bubble baby syndrome”. The only alternative treatment is a stem cell transplant, but finding a compatible donor is very difficult.

Strimvelis will give children with ADA-SCID the chance to live outside their bubble, go to school and play with other children without the fear of infection.

Can the pace of innovation be sustained?

Growth in UK cell and gene therapy industry has increased for five successive years, rendering it second only to the United States. 

Last year, the Cell and Gene Therapy Catapult added a state-of-the-art manufacturing centre to its pipeline. This £55M large-scale GMP manufacturing centre will help bring cell and gene therapies to market in the UK and internationally. The recent announcement of a further £12M investment to double the capacity of the manufacturing centre will ensure that growth in the sector is not limited.

Among the first inhabitants of the manufacturing centre are Autolous and Cell Medica who will manufacture their respective CAR-T cell therapies as immuno-oncology treatments.

What does this mean for recruitment?

The roles which are currently in high demand include regulatory affairs; chemistry, manufacturing and control (CMC); and scientists at all levels in viral vector, cell line and process development, as well as manufacturing. The large number of deals in the area means there is an increasing need for business development, licensing and particularly intellectual property specialists.

With discovery, development and clinical trials at a 5 year high, as well as pharma giants GlaxoSmithKline and Novartis growing their cell and gene therapy divisions, we envisage the recruitment needs increasing. This will ensure that cell and gene therapy fulfils its potential and truly can propel the UK to future growth.

For more fascinating insights into the ever-changing world of the life sciences sector, stay tuned to all SRG Blogs.

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