THE SCIENCE DRIVING BIOTECH
Next week, BioTech Primer will be participating the BIO 2014 Annual International Convention in San Diego, CA. The event draws thousands and, as in years past, BioTech Primer will be headlining a few events. Join us and learn!
On Monday, June 23, I will be teaching an all-day class called “The Science Driving Biotech.” What can you expect to learn?
Starting with the basics: what is DNA? How do cells use DNA to make proteins? How has the biotech industry adapted this knowledge to make biologic drugs? I will then quickly move into more complex, industry-relevant applications and products.
Some of my favorite topics to teach include the genetic variation, genomics, and stratified medicine sections of the course. Building on the concept of the gene as the basic unit of human inheritance, I love to surprise my students with information on just how highly similar we are at the level of DNA sequence—but how in some cases, very small differences can lead to a disease state. We then move on to a discussion of stratified medicine—how disease-related genetic information is being used to develop therapeutics that are targeted at specific subsets of patients and the companion diagnostics being developed to identify the patients most likely to respond favorably. Finally, I give an overview of the next-generation sequencing technologies that are being used to generate this genetic data.
This is a BIO2014 affiliate event and registration is open to everyone. The class will take place at Manchester Grand Hyatt San Diego. If interested click here for more information and registration. Due to limited space, preregistration and payment are necessary. I hope to see you there!
2 OF THE TOP 10
Next week, I will be joined by my fellow BioTech Primer instructor Dr. Collins Jones to deliver a two-hour talk on the Top 10 Current Science Trends & Advances at BIO2014 in San Diego. The hardest part about putting this talk together was limiting it to just ten topics! Two of the topics that I chose are summarized below:
Genome Editing: we often refer to a person’s genome—his complete set of genes—as a blueprint for life. Sometimes, there are errors in this blueprint. Wouldn’t it be great if we could edit the blueprint and correct these errors? We might soon be able to. Researchers have developed tools that make it possible to cut an individual’s DNA in a specific location. Cellular enzymes then attempt to repair this break, but typically make mistakes in this repair, rendering the gene ineffective. This approach can be used to “knockout” a defective gene. This may sound like science fiction, but Sangamo BioSciences is currently conducting Phase II human clinical trials testing this technology’s ability to render human T-cells resistant to HIV. Sangamo also has positive preclinical data supporting the idea of using this technology to actually correct a gene sequence by co-delivering a “repair template”—a short piece of DNA containing the correct gene sequence which the cellular repair enzymes can then use to correct the defective gene sequence.
Immunotherapy: the term “immunotherapy” covers a wide range of topics, including vaccinations, monoclonal antibody therapeutics, stimulating a patient’s own immune system to fight cancer or other diseases, and using antibodies to deliver toxic compounds directly to cancer cells. During Tuesday’s talk, we will give an overview of what vaccines are, how they work, and describe promising work that may lead to an HIV vaccine. We will also discuss a personalized cancer vaccine developed using a patients’ own cancer cells, and describe a therapeutic vaccine designed to activate the body’s own immune response to cancer. Finally, we will talk about some of the new and exciting applications of monoclonal antibodies from targeting Alzheimer’s disease to use as a vehicle for drug delivery in antibody-drug conjugates.
Please join me and Collins on Tuesday morning, June 24th, from 9:00-11:00 at the San Diego Convention Center in room 23ABC. This talk is only open to BIO2014 registrants. FYI: Last year the room was filled to capacity and many were turned away at the door. Get there early and get your seat!
NO SCIENCE SINCE HS? WE CAN HELP
BioTech Primer has published a book titled The Biotech Primer: An insider’s guide to the biotech and pharma industry—though some refer to it as “Biotech for Dummies.” The 200-page book explains the science behind the biotech industry, and includes a glossary of commonly used terms. Similar to our classes, The Biotech Primer starts out with the fundamentals of biology used by researchers and progresses to how those fundamentals are employed to create therapeutics. The text is written to be understood by all—even those who have not taken a science class since high school. The illustrations and cocktail fodders (so you can impress your friends at your next party) keep things interesting. The Biotech Primer will be available all next week at the BIO Bookstore. If you are not going to San Diego, but would like a copy, click here to order online.
Excerpted below are a few paragraphs from Chapter 8: The Science of Discovery
Validating The Target
Once a potential drug target has been identified, researchers will try to validate the target by determining whether the target plays a key role in the disease process and whether targeting it is likely to be both safe and effective. Target validation is a very important step in the drug discovery process, since research and development gets progressively more expensive—if a drug is unlikely to be successful, millions of dollars can be saved if this is realized early on.
Target validation will most often include cell-based assays (in vitro testing) and animal models (in vivo testing). Since the goal of many therapeutic interventions is to inhibit the activity of the selected target, many validation assays attempt to measure the effects of inhibition. In some cases, a selected target may play a role in disease progression – but if it is inhibited, another cellular protein will simply take its place, nullifying the potential therapeutic effect of an inhibitor. In other cases, inhibiting a selected target may have the desired therapeutic effect—halting cancer cell growth, for example—but may also result in unexpected side effects, such as the death of healthy cells.
One of the most popular ways of testing the effects of inhibition in cell-based assays is through the use of RNAi, described in detail in the chapter “From Gene to Protein.” RNAi is an effective way to quickly determine the results of blocking the production of a particular protein, thus mimicking the effects of a strong inhibitor.
If the cell models show promise, the researchers will move on to animal models, most likely designing experiments using so-called “knockout” mice—mice in which a particular gene has been disrupted. Researchers can ask similar questions to those asked in the cell model, but on the scale of the whole animal: do the experimental mice still get cancer, Parkinson’s disease, diabetes, or heart disease when the target gene is silenced or absent? The animal model also provides valuable information about targeting safety that might not be addressed in cell models because it is possible to examine the effects of gene targeting on the whole organism.
The introduction of genetic information from spiders into goats allows these mammals to produce spider silk in their milk, which is collected and purified to make products such as soft body armor (89 The Biotech Primer).