Cell & Gene Therapy- Making a difference


28 Mar 2019

Cell & Gene Therapy- Making a difference

Cell and Gene Therapy technology is evolving rapidly and we are now closer than ever to gene and cell therapies for many genetic mutations and diseases, although many of these still remain as experimental medicines. Nevertheless, ongoing research will hopefully provide long term effective treatment more freely available to patients globally.

The exciting breakthrough in the treatment of HIV with two patients becoming free of HIV after stem-cell therapy through a bone marrow transplant has once again thrown the spotlight on the potential impact of cell and gene therapies. In both cases the patients’ blood cells were destroyed and replenished with stem cells from a healthy donor, more specifically, someone who had two copies of a mutation in the CCR gene which gives resistance to the HIV infection. Although it remains to be seen if the patients will be fully cured this is very promising and highlights the potential benefit that further research may deliver in helping to find a truly effective treatment for millions of people suffering from HIV worldwide.

These breakthroughs demonstrate the need for continued cell and gene therapy research. There are currently nearly 300 cell and gene therapies in development targeted at over 100 diseases, making 2019 potentially a very big year for further breakthroughs. Indeed, the FDA are expecting to see 200 cell and gene therapy investigational new drug applications per year by 2020. To put this into context there are only 3 cell and gene therapies that are approved for sale, so far, so this future projection is looking incredibly promising.

As in all areas of research there are however questions over future funding and in the UK, this has been compounded by fears over the impact of Brexit. However, in the commercial sector last year we have seen a large mergers and acquisition with a focus around cell and gene therapy. Novartis acquired the gene therapy company AveXis, Celgene bought the CAR-T Cell company Juno Therapeutics and have also completed a large merger with Bristol-Myers Squib on their cell and gene therapy research. We have also seen Biogen take over the Ophthalmology gene therapy focused company Nightstar. It is hoped these acquisitions and mergers may provide new opportunities for both translational and discovery research.

As a company Penna will be continuing to support our clients find the research, scientific and commercial talent they need to maximise the social and economic impact that this exciting area of research can bring.