DNA Vaccines Explained

Emily BurkeBiotech Basics, Clinical Trials, Cocktail Fodder, The WEEKLY, Vaccine

MORE ON THE POWERFUL, ELEGANT SIMPLICITY OF VACCINES Last week, we overviewed vaccine development and manufacture, focusing on those that use whole pathogens to protect us from a disease. This week, we examine subunit and polysaccharide vaccines, which use different strategies to fight infection. We also take a brief look at the US Food and Drug Administration (FDA)’s vaccine approval … Read More

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

The Top Tech Of 2016

Emily BurkeBiotech Basics, Cancer, Clinical Trials, Drug Targets, FDA, Mechanism of Action

CAR-T Primer The hottest cancer therapy in the pipeline — chimeric antigen receptor therapy (CAR-T) — continued to mature in 2016 with its first FDA approval for blood cancer patients set to arrive this year. What’s next in the world of CAR-T? A whole lot: Additional cancer and autoimmune disease indications in preclinical development. Added safety features. More affordable “off … Read More

The First Three-Parent Baby

Emily BurkeCocktail Fodder, Deoxyribonucleic Acid (DNA), FDA

BREAKING DOWN THE SCIENCE & SCRUTINY “World’s First Three-Parent Baby” made headlines in publications ranging from Nature to CNN last week. This human interest story is of a baby boy born in Mexico with genetic material from three different parents, achieved by a technique known as three-parent in vitro fertilization (TPIVF). In this issue, we will explain why TPIVF is used … Read More

Pushing The Self-Destruct Button

Emily BurkeCancer, Drug Approvals, FDA, Genetics, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs, The WEEKLY

 Pushing the Self-Destruct Button Breakthrough drug Venclexta charged onto the marketplace three months early to battle chronic lymphocytic leukemia. AbbVie’s (North Chicago, IL) and Roche’s (Basel, Switzerland) new therapy gained a quick approval after 80% of patients in the 106-person clinical trial responded to the small molecule inhibitor. Chronic lymphocytic leukemia (CLL) is the most common type of leukemia in adults, with approximately … Read More

Orphan Drugs Lead 2015 Approvals

Emily BurkeBiologics, Cardiovascular Disease, Drug Approvals, FDA, Mechanism of Action, Monoclonal Antibodies, Orphan Disease, Orphan Drugs, Small Molecule Drugs, The WEEKLY

Treatment Options Broaden Driven by 19 new orphan approvals, a whopping 45 novel drugs were cleared for the marketplace in 2015. Additionally, new approvals opened up treatment options for Americans with diseases ranging from breast and lung cancers to irritable bowel syndrome. According to FDA numbers: 19 were for orphan diseases that affect fewer than 200,000 patients per year in the US. … Read More

Cancer Immunotherapy Goes Viral

Emily BurkeBiologics, Cancer, Drug Approvals, Drug Targets, FDA, Mechanism of Action, The WEEKLY

Oncolytic Viruses Make Their Debut Does a virus engineered to harness the immune system to fight cancer sound like a clever idea? Amgen (Thousand Oaks, CA) certainly thinks so, because their talimogene laherparepvec (T-Vec) recently earned an FDA approval to fight inoperable melanoma recurrent after initial surgery. Oncolytic viruses—like T-Vec—have the attention of both industry media and mainstream news programs. This new class of therapy is an elegant “hack” of the immune … Read More

Deciphering DMD

Emily BurkeAntisense, Clinical Trials, Drug Development, Drug Targets, FDA, Genetics, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), Small Molecule Drugs

THE ROOT OF DUCHENNE MUSCULAR DYSTROPHY World Duchenne Awareness Day called attention to Duchenne Muscular Dystrophy (DMD) this past Monday. Affecting one in 3,500 newborn baby boys worldwide, this fatal disease is caused by a mutation in the gene that codes for the dystrophin protein. Easily damaged muscle cells, progressive muscular weakness, and serious medical problems—including significant impairment of the heart and lungs—are the direct results of this … Read More

Biotech’s Battlefront: Monoclonal Antibodies

Emily BurkeBiologics, Biotech Basics, Cancer, Drug Targets, FDA, Mechanism of Action, Monoclonal Antibodies, Orphan Drugs, The WEEKLY

Since their premier on the scene, monoclonal antibodies (mAb) have demanded top billing on the biotech marquee, creating a cast of therapeutics used to treat diseases like autoimmune disorders and cancer. The first player debuted in 1986 when Janssen-Cilag’s OKT3 gained FDA approval to treat transplant rejection patients. Fast forward to 2013, where half of the top ten best selling … Read More

Mutations And Disease: The Spice Of Life

Emily BurkeBiologics, Biotech Basics, Cancer, Cardiovascular Disease, Cocktail Fodder, Drug Targets, FDA, Genetics, Mechanism of Action, The WEEKLY

This week’s issue features excerpts from the “Genetic Variation” chapter of The Biotech Primer, our 200-page book that provides an in-depth look at the biotech industry and the science that drives it. In this chapter, we explain the different types and causes of genetic mutations and then explore their relationship to disease and therapeutics. Today, we begin with a discussion … Read More

MRSA vs. Dalvance: Let’s Get Ready To Rumble

Emily BurkeThe WEEKLY

MRSA’S MATCH Last week, the FDA approved Dalvance, Chicago-based Durata Therapeutic’s new antibacterial drug for the treatment of methicillin-resistant staphylococcus aureus (MRSA) infections. Why is Dalvance newsworthy? MRSA is an infectionthatis difficult to treat because it is resistant to all the usual medications: the cephalosporins and penicillins (methicillin, dicloxacillin, oxacillin, etc.) What is MRSA? MRSA skin infections may begin as … Read More

The FDA’s Decidedly Average Year

Emily BurkeThe WEEKLY

2013 DECIDEDLY AVERAGE FOR THE FDA The FDA approved 27 new drugs in 2013, down from the 15-year high of 2012 with its 39 approvals. This should not be cause for alarm since 2013 numbers fall in line with the average of 28 over the last five years (FDA.gov). The FDA attributes the drop to fewer applications received: 32 for … Read More