It is evident from a recent market survey on gene editing in drug discovery that CRISPR/Cas9 is now recognised as the superlative method when attempting a gene knockout or when introducing defined mutations, insertions or modifications to the genome.
The Pistoia Alliance (whose membership includes most of the world’s major pharmaceutical companies, technology vendors and publishers active in the life science R&D sector), is keenly interested in discovering the ways in which innovation through pre-competitive collaboration can be used to explore and exploit the constant stream of new technologies that become available.
Modern drug discovery approaches take too long, are too expensive, have too many clinical failures and uncertain outcomes. There are many reasons for this unsustainable business model, but primarily, the approaches are not comprehensively holistic.
If we have learned anything from scientific research in the last 20 years, it is that finding cures to complex diseases is difficult. Despite the promise of the genomic revolution, disease progression and patient outcomes are still not easily predicted by genetic factors alone.
Epigenetics is a term that refers to the durable and context-specific regulation of transcription processes. The majority of epigenetics involves a complex system of enzymes, binding proteins and accessory proteins that control access to the genome by transcription factors and other parts of the DNA replication machinery.
Christopher Voigt is a professor of biological engineering at the Massachusetts Institute of Technology, where his lab focuses on synthetic biology. Two major areas of interest for him are developing a genetic programming language for cells and applying synthetic biology to biotechnology challenges.
Recent advances of single cell technologies are facilitating the opportunity to discern biological insights within individual cells and providing a means to reveal previously hidden relationships between individual cells within a population.
From the pioneering days of Watson and Crick in 1953, to completion of the human genome project in 2003, advances in our understanding of DNA have raised hopes that its direct manipulation at the level of the genome could revolutionise the drug discovery process.