Technology

There are no magic bullets in drug discovery. Technology on its own can rarely change the game, but it does help creative people make better decisions. Vertex was founded around the idea that integrating information from biology, chemistry and biophysics will increase the success rate of drug discovery. We measure success in terms of breakthrough first-in-class therapies. Our drug development pipeline is comprised entirely of novel molecules from our research labs around the world—novel molecules that we believe could fundamentally change the treatment of serious diseases.

Glossary

Here are some helpful descriptions of scientific processes, techniques and disciplines, including their relevance to our day-to-day activities at Vertex:

In drug discovery, a variety of assays may be performed to measure the biological or pharmacological potency of candidate compounds in order to select the best molecules for more advanced testing. We use modern cell biology, enzymology, and screening techniques to develop high-throughput assays that provide high-quality information to support drug discovery. Many of these assays are built upon a number of gene reporter technologies such as green fluorescent protein and beta lactamase. We also utilize our assay capabilities to develop novel proprietary in vitro assays to rapidly establish ADME/toxicology profiles for compounds in our screening library.

We have invested in instrumentation in order to provide automation solutions that address many of the more repetitive and labor intensive aspects and processes of drug discovery. We have a dedicated research and application development group that has invented numerous proprietary hardware and systems. For example, most of our ion channel research is conducted using E-VIPR , our proprietary screening technology that uses fluorescent probes and waves of electrical stimulation to study ion channels. E-VIPR provides an automated, high-throughput platform that enables us to collect high-quality data at speeds up to a thousand times faster than patch clamping.

Biophysics is an interdisciplinary science that applies the theories and methods of physics to questions of biology. At Vertex, our structural biologists generate atomic structural information on molecular targets using X-ray crystallography and nuclear magnetic resonance (“NMR”) spectroscopy to guide design and optimization of lead classes of drugs.

Computer based modeling, as applied in drug discovery, allows scientists to quickly evaluate many different possibilities in silico before making the most promising prototype compounds for testing in the lab. We apply advanced proprietary computational modeling tools to guide the characterization and selection of compounds for synthesis. We use proprietary algorithms to sort and filter compounds for specific properties in order to seek compounds that are more likely to become development candidates.

Functional genomics is a field of molecular biology that attempts to make use of the vast wealth of data produced by genomic projects (such as genome sequencing projects) to describe genes and their interactions. Unlike genomics and proteomics, functional genomics focuses on the dynamic aspects such as gene transcription, translation, and protein-protein interactions, as opposed to the static aspects of the genomic information such as DNA sequence or structures. At Vertex, we use functional genomics techniques, such as gene knock-out mice, to help guide target selection and test the potential of chemical compounds in disease models. We also use antisense, siRNA, dominant negative cell lines, transcriptional profiling, proteomics and other biological approaches to better characterize the role played by specific targets in cellular processes.

High-throughput screening (HTS) is a method for scientific experimentation especially used in drug discovery and relevant to the fields of biology and chemistry. Through a combination of modern robotics, data processing and control software, liquid handling devices, and sensitive detectors, HTS allows a researcher to effectively conduct millions of biochemical, genetic or pharmacological tests in a short period of time. We conduct assays for most enzyme and receptor targets using very high-throughput screening approaches, many of which are proprietary. These assays enable us to rapidly generate large numbers of lead compounds and drug candidates across targets from many different gene families. These approaches integrate compound management, plate replication with miniaturized screening, hit (potential lead) identification and follow-up.

Pharmacokinetics is the study of the action of drugs in the body over a period of time, including the processes of absorption, distribution, localization in tissues, biotransformation and excretion. Pharmacology is the study of how substances interact with living organisms to produce a change in function. We employ a number of approaches to obtain predictive information on the bioavailability, pharmacokinetic profile and efficacy of potential drug candidates. These approaches include in vitro metabolism and toxicological studies and in vivo assessment of leads in predictive animal models.

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