Putting The CAR-T Before The Horse

Emily BurkeThe WEEKLY

THE STORY BEHIND CAR-T The hottest cancer therapy in the pipeline — chimeric antigen receptor therapy (CAR-T) — got a big boost last month when an FDA advisory panel unanimously recommended approval of the treatment for children and young adults with a severe form of leukemia who have run out of other options. Developed by Novartis (Basel, Switzerland), this elegant … Read More

Chaperoning The Rare Disease Dance

Emily BurkeThe WEEKLY

PROPERLY FOLDING MISFOLDED DISEASE PROTEINS Amicus Therapeutics (Cranbury, NJ) found itself in the news earlier this month when the FDA agreed to review the company’s new drug application for their investigational therapy to treat Fabry’s disease. The drug under consideration, migalastat, has already been approved by the European Medicines Agency. It belongs to a small, but growing class of therapeutics known … Read More

Unpacking Digital Medicine

Emily BurkeCardiovascular Disease, Diabetes, FDA, Medical Device

CAN APPS PROGRAM BETTER HEALTH? Digital medicine is defined by the field’s pioneer Dr. Eric Tool of the Scripps Translational Science Institute (La Jolla, CA) as “the ability to digitize human beings, by a variety of means (sequencing, sensors, imaging, etc.), fully exploiting our digital infrastructure of ever-increasing bandwidth, connectivity, social networking, the Internet of all things, and health information systems.” This new field is changing the way … Read More

Stopping A Big Problem: Blood Clots

Emily BurkeCocktail Fodder, Drug Approvals, Drug Targets, Mechanism of Action, Small Molecule Drugs

MEDS FOR THINNER BLOOD CAN EQUAL FEWER CLOTS, BUT HOW? The FDA’s recent approval of Portola Pharmaceuticals’ (South San Francisco, CA) new blood thinner drug Bevyxxa paved the way for the prevention of blood clots in patients hospitalized for conditions such as heart failure, stroke, and pulmonary disease. The medical term for blood clot is venous thromboembolism (VTE), but if we take it apart: “venous” means relating … Read More

Breast Cancer Subtypes

Emily BurkeBiotech Basics, Cancer, Genetics, Mechanism of Action

FOUR MOLECULAR VARIANTS EXPLAINED Hearing your doctor utter the words HER2-positive, HR-positive, triple-negative, or BRCA mutation can be devastating — even for the most resilient person. Simply put, breast cancer is a complex disease. A diagnosis can be derived from any combination of the factors listed above — or, none at all. The National Cancer Institute (Bethesda, MD) has outlined four molecular subtypes of the disease. … Read More

Cell Signaling Explained

Emily BurkeBiotech Basics, Cancer, Deoxyribonucleic Acid (DNA), The WEEKLY

UNDERSTANDING THE DISEASE PROCESS Greetings from BIO 2017! It’s been a busy week here at the BIO International Convention here in San Diego, CA. At our convention booth, this year’s giveaway was our book written especially for non-scientists: The Biotech Primer: An insider’s guide to the biotech and pharma industry. If you weren’t at the convention to stop by and … Read More

The Biotech Primer BIO 2017 Preview

Emily BurkeBiotech Basics, Business of Biotech, The WEEKLY

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! Learn the fundamentals of biotechnology in our all-day class BioBriefing: Biotech for the Non-Scientist on Monday, June 19th. SOLD OUT What are the most popular topics in the WEEKLY? Attend Readers’ … Read More

A Skin Cell With Stem Cell Diversity?

Emily BurkeBiologics, Clinical Trials, Drug Development, Drug Targets, Term of the Week, The WEEKLY

INDUCED PLURIPOTENT STEM CELLS SHOW PROMISE Imagine being able to reprogram one of your own skin cells to produce a functioning nerve cell or section of cardiac tissue. This may sound like science fiction — but the groundwork for this to become a reality is already in the works as researchers expand their ability to create and manipulate induced pluripotent stem … Read More

Epigenome: Writing, Reading & Erasing

Emily BurkeCancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genomics, Mechanism of Action, Small Molecule Drugs

FOUNDATIONS OF EPIGENETICS Genetic mutations — changes in the order of the A, C, G, and T nucleotide bases that make up a gene — have been the primary focus of cancer researchers over the last several decades. By sussing out mutations involved in regulating cell growth and division, scientists better understand the molecular range of different cancers and consequently develop … Read More

Nanobodies: These Are Not Your Mother’s mAbs

Emily BurkeBiologics, Biotech Basics, Cancer, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Orphan Disease, Orphan Drugs

The Drug Kingpins Monoclonal antibodies (mAbs) are the undisputed drug kingpins. In 2013, the mAb market raked in $75 billion in combined sales, covering a whole range of indications from cancer and infectious disease, to autoimmune disorders, and even high cholesterol. Despite the success, mAbs have one chink in their armor: they cannot enter cells due to their large size, hampering their range … Read More

Viruses Blasting Cancer

Emily BurkeBiologics, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Mechanism of Action, Ribonucleic Acid (RNA)

Engineering Viruses To Attack Getting bacteria-eating viruses to combat antibiotic resistance isn’t the only way viruses are being hacked to defend team homo sapien. This week, we’ll turn our attention to another benevolent use of viruses: cancer-fighters known as oncolytic viruses. Oncolytic Virus Primer Oncolytic viruses are an immunotherapy — a type of therapy that harnesses the power of a patient’s immune system to combat a disease. … Read More

Phage — More Than Just A Phase

Emily BurkeBiologics, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Term of the Week, The WEEKLY

Old Way Of Fighting Bacteria Renewed One of the greatest public health challenges of the 21st century is antibiotic resistance, which occurs when a few bacteria in a given population develop a genetic mutation that enables them to survive — even in the presence of antibiotics. How do bacteria become drug resistant? Suppose a particular antibiotic inhibits an enzyme required for bacterial replication. … Read More

Decoding Your Genes

Emily BurkeBiotech Basics, Deoxyribonucleic Acid (DNA), Diagnostics, Drug Approvals, Easily Confused, FDA, Genetics, Genomics, Orphan Disease, Term of the Week, The WEEKLY

The Skinny On DNA Testing 23andMe (Mountain View, CA) recently found itself back in the limelight after the disease risk section of its mail-in DNA kit received an OK from the FDA. The Silicon Valley biotech had to halt sales of its direct-to-consumer genetics testing back in 2013 after regulatory officials grew concerned over marketing claims and the possibility of consumers misinterpreting the test results. 23andMe rebooted a limited part of … Read More

RNA Therapeutics March Onward

Emily BurkeAntisense, Biotech Basics, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Mechanism of Action, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), The WEEKLY

Taking Steps With Antisense With their high specificity and relative low manufacturing cost, RNA therapeutics may be tomorrow’s biotech sweetheart. In fact, chances are good that previously “undruggable” targets that cannot be accessed by small or large molecule drugs, are now within reach. However, the main roadblock continues to be delivery—getting the RNA drug where it needs to be, in high enough concentrations, to … Read More

Putting The NA in DNA

Emily BurkeBiologics, Biotech Basics, Cancer, Cardiovascular Disease, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Ribonucleic Acid (RNA), Small Molecule Drugs, The WEEKLY

Nucleic Acid Therapeutics Small molecule, peptide, and biologic drugs aren’t the only players in the game of drug development. A fourth class of therapeutics differs from all three of these: nucleic acid-based drugs. These drugs are rising in prominence due to their potential to specifically target a wide range of diseases, including various types of cancer, autoimmune, and infectious diseases. Companies … Read More

Pills, Proteins & Peptides

Emily BurkeThe WEEKLY

Promising Peptide Therapies The front runners in the game of drug delivery include small molecule and large molecule drugs, but there is another class that lands right in between: peptides. Several companies, including Rhythm Pharmaceuticals (Boston, MA), Kalos Therapeutics (San Diego, CA), Aileron Therapeutics (Cambridge, MA), and Bicycle Therapeutics (Cambridge, MA) have emerged as prominent players in the peptide arena. Let’s … Read More