Alongside the topic of gene editing technologies that keeps making headlines these days, there is also a wave of breakthroughs in the field of RNA-targeting medicines, primarily falling into the following two categories: antisense oligonucleotides (ASO), and RNA interference (RNAi) technologies. (another promising approach, dealing with editing RNA itself by ADAR enzymes, is not covered in this post).
Pharmaceutical manufacturers find themselves at a critical juncture. In the past 30 years, pharma has seen some significant shifts. Consider the expanding range of drug therapies, once confined almost exclusively to small molecule drugs. Biologics now comprise a substantial share of the market, and novel treatments such as cell and gene therapies are rapidly gaining traction. Another major trend is the rise of outsourced manufacturing, primarily for small molecule drugs.
A transformative technology for gene editing - clustered regularly interspaced short palindromic repeats (CRISPR) -- has become ubiquitous, at least in research laboratories, where scientists efficiently adopted CRISPR technology to manipulate genes of interest. Still, applying it for humans is associated with greater risks and faces ethical issues. However, alongside a too reckless experiment of a Chinese scientist who edited babies’ genomes, plenty of companies have made well-thought progress toward transferring gene editing technologies into the clinic. Here we make a brief but pithy review on up-to-date gene editing approaches and start-up companies active in this area.
As we enter a new decade, our belief in the the impact of Artificial intelligence (AI) is only getting stronger. Supporting the industry to drive the right drug to the right patient at speed is a huge responsibility that we take very seriously. Towards the end of the last decade we have seen great progress made within life sciences and the use of AI, but moving into 2020 the spotlight on commercial teams and gaining competitive advantage with AI will intensify.
Pharmaceutical companies are increasingly outsourcing their R&D activities, including early-stage research programs, to third party organizations -- academic institutions, biotech startups, and private contract research organizations (CROs) -- as a means to stay competitive, flexible, and profitable against all odds.
Economically, there are factors such as increasing downward pressure on drug pricing by governments, an impending “patent cliff” threatening $198 billion worth of sales during 2019-2024), and downturns in income due to the increasing competition from generics and biosimilars.
From the innovation's point of view, there is a boom in life sciences, stimulating the emergence of novel biological targets, therapeutic modalities, and even whole new areas of drug discovery -- adding opportunities, but also complexity and uncertainty to research programs. In fact, according to Deloitte’s report, return on late-stage pipelines dropped for the top 12 pharma companies from 10.1% in 2010 down to 3.7% in 2016.
Technologically, there is an unfolding “digital revolution”, bringing even further complexity and investment cost to the table -- in a form of artificial intelligence (AI), data mining and big data technologies, data-driven diagnostics, and digital health.
Finally, the rise of the personalized medicine paradigm forces companies to rethink their research pipelines and “one-size-fits-all” product development programs, as well as reconsider their market strategies.