Four trends that will transform healthcare in Europe in 2016

With 2015 giving some hints of what is to come, the Kaiser Associates European Healthcare team are predicting another year of big disruptions, transformations and innovations as the healthcare industry continues to overhaul outmoded business models.

Gene therapy: The next generation of targeted drug delivery

The emergence of precision medicine has at its core the ability to target diseases with specialised therapies specific to certain types of people with specific genetic genotypes. To increase specificity and efficacy, and reduce adverse effects resulting from a medicine going where it is not needed and harming healthy cells, targeted therapies have been a focus of drug development for small molecules, biologics and, more recently, CRISPR/Cas9 genome editing and gene therapy.

The rapid advancement of genome-editing techniques holds much promise for the field of human gene therapy. From bacteria to model organisms and human cells, tools such as CRISPR/Cas9 have been successfully used to manipulate the respective genomes with unparalleled precision. In 2015, a number of companies invested in the technology, starting with pharma giant Novartis, which signed two separate deals with gene-editing start-ups Intellia Therapeutics and Caribou Biosciences. Just a month after the Novartis deals, AstraZeneca sealed four deals with the Innovative Genomics Initiative, the Broad and Whitehead Institutes, the Wellcome Trust Sanger Institute, and Thermo Fisher Scientific.

In the past few years there has been growing commercial interest and activity within the field of gene therapy with large investments and an array of licensing deals and funding rounds reported. Gene therapy aims to treat diseases by replacing a non-functioning gene with a functioning one. Unlike usual drug therapies that must be taken for months or years, gene therapy offers the alluring possibility of a one-time treatment, so biotech startups are racing to get such therapies on the market. 

2015 saw the European Medicines Agency approval of UniQure’s Glybera (alipogene tiparvovec), a gene therapy that mitigates lipoprotein lipase deficiency, a rare disease affecting only 150-200 people in Europe1. This was the first gene therapy accepted by regulators; this watershed augurs future approvals for other such therapies in development.

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