The Biotech Primer BIO 2017 Preview

In Biotech Basics, Business of Biotech, The WEEKLY by Emily Burke

Biotech Primer At BIO 2017

Biotech Primer will be headlining a few events at the BIO 2017 Annual International Convention in San Diego, CA next week. Will you be there? If so, please join us!

Biotech Primer’s Biotechnology For Non-Scientists Courses

What can you expect to learn when you take a class with us? Starting with the basics, we build upon this base to deliver more complex, industry-relevant knowledge about disease, diagnostics and drugs.

Some of my favorite topics to teach include the genetic variation, genomics, and immunology sections of the course. Starting with 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 all are at the level of DNA—but how in some cases, very small differences can lead to a fatal disease state. We then discuss how advances in next generation whole genome sequencing technologies are enabling us to better identify disease specific mutations, and how this information is already being used to develop better diagnostics and drugs. In the immunology section, I give an overview of how our immune system works and explain how biotech companies have leveraged this knowledge to develop cutting-edge therapies.

This is a BIO2017 affiliate event and registration is now closed because we have sold out. However, we deliver this type of course in various locations and can customize it and bring it in-house to your team. For a list of upcoming classes click here. To learn more about our in-house classes, contact Stacey at Franklin@BiotechPrimer.com.

Reader’s Choice: Top Topics From Biotech Primer Weekly

Dr. Emily Burke

On Tuesday, June 20th, I will be giving a talk, Readers’ Choice: Biotech Primer WEEKLY Top Topics, that highlights the most popular Biotech Primer WEEKLY topics over the past year. Two of the topics chosen by our readers are summarized below:

  • Genome editing: we often refer to a person’s genome—the complete set of genes—as a blueprint for life. Sometimes, there are errors in this blueprint. Wouldn’t it be life changing if we could edit and correct these errors? We might soon be able to! Researchers have developed tools that make it possible to cut an individual’s DNA at a specific site, and either disrupt or correct the gene sequence at that location. The first of these therapies are now entering clinical trials. Come learn more about how this technology works!
  • Immunotherapy: the term “immunotherapy” covers a wide range of topics, including vaccinations, monoclonal antibody therapeutics, checkpoint inhibitor therapies, CAR-T, and antibodies that deliver toxic compounds directly to cancer cells. During Tuesday’s talk, I’ll give a detailed overview of how each of these different types of immunotherapies work.

Please join me on June 20th, from 10:45am-12:15pm at the San Diego Convention Center in room 10. This talk is only open to BIO 2017 full 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 High School? 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 basics 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 fodder (so you can impress your friends at your next party) keep things interesting. Stop by booth #5225 to pick up your free copy!

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.

Cocktail Fodder

Proteins are easily broken down by the body’s digestive system, therefore patients receiving biologics (large molecule drugs) do not take them orally, but rather as injections.