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

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

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

Therapeutic Antibody Primer

Emily BurkeBiologics, Biotech Basics, Cancer, Clinical Trials, Drug Development, Drug Targets, Easily Confused, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs

Basics & Innovations Monoclonal antibody (mAb) therapeutics burst onto the healthcare scene 20 years ago, and today they remain one of the most versatile and effective therapeutics available. The tried and true mAbs are still in high demand, and we suspect this first wave of derivative products clamoring their way through the pipeline will be equally as successful. In this WEEKLY, we’ll … Read More

New Hope For Spinal Muscular Atrophy

Emily BurkeAntisense, Biologics, Clinical Trials, Drug Development, Drug Targets, FDA, Genomics, Mechanism of Action, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), The WEEKLY

FIRST THERAPY APPROVED FOR SMA Squeaking by on December 23rd as the last new drug approval of 2016, Biogen’s (Cambridge, MA) Spinraza now provides hope for the thousands of families affected by a debilitating neuromuscular disorder known as spinal muscular atrophy (SMA). SMA robs people of their ability to walk, eat, and ultimately, breathe. In addition to Spinraza, there are 13 … Read More

Taking A Swing At Allergies

Emily BurkeClinical Trials, Mechanism of Action, Small Molecule Drugs

HOW DO ALLERGIES DEVELOP? Watching a game at the ballpark and digging into a bag of peanuts is a source of entertainment for many Americans. For the 15 million who suffer from peanut allergies, the idea of being taken out to the ballgame elicits concern — or even anxiety. Food allergies — think tree nuts, milk, eggs, wheat, soy, fish, and … Read More

Unwiring The Biology Of Fibrosis

Emily BurkeAntisense, Biologics, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs, Term of the Week, The WEEKLY

Fibrotic Disease Innovations Fibrotic diseases — organ and tissue disorders that occur as a result of the buildup of excessive scar tissue — are sounding the alarm in the biotech sector. A silent epidemic creeping up on the Western world involves non-alcoholic steatohepatitis (NASH), or liver fibrosis associated with obesity and type 2 diabetes. Left untreated, NASH can lead to liver failure. Currently, … Read More

The Science Behind The Opioid Epidemic

Emily BurkeBiotech Basics, Clinical Trials, Cocktail Fodder, Drug Development, Drug Targets, Mechanism of Action, Small Molecule Drugs

A Big Pain The opioid addiction crisis gained attention at the highest levels of U.S. policy circles this past year. Presidential candidates that disagreed on nearly everything else vowed to make fighting the epidemic a priority if elected. In July, the U.S. Senate overwhelmingly approved a bill to strengthen prevention, treatment, and recovery efforts. And no wonder – according to … Read More

The Molecular Cause Of Obesity

Emily BurkeClinical Trials, Diabetes, Drug Development, Drug Targets, Mechanism of Action, Term of the Week

Targeting Fat Obesity is considered one of the most pressing public health issues of the day. According to the Center for Disease Control, 37% of adults and 17% of children in the U.S. are obese. The latest drug interventions work by attempting to suppress food intake, which has proven beneficial for some. However, there is still a large unmet need … Read More

Cancer Vaccines & Game Changers

Emily BurkeBiologics, Cancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genomics, Mechanism of Action, Term of the Week

The Elusive Cancer Vaccine The promise of cancer vaccines have proven to be elusive. A new crop of biotechs are hoping to change that by taking advantage of the latest advances in genomics. Scientists are working overtime trying to develop cancer vaccines that train the immune system to recognize and fight an established tumor. In this WEEKLY, we’ll break down the science … Read More

The Race To Beat SMA

Emily BurkeAntisense, Biologics, Clinical Trials, Drug Development, Drug Targets, FDA, Genomics, Mechanism of Action, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), The WEEKLY

Zeroing In On The SMA Pipeline A decade ago, there was only one drug in development for a debilitating neuromuscular disorder known as spinal muscular atrophy (SMA) — robbing people of their ability to walk, eat, and ultimately, breathe. Today, there are 14 therapies making their way through the clinic according to the patient advocacy group Cure SMA. The increase is … Read More

Killing Cancer At Its Stem Cell Origin

Emily BurkeBiologics, Cancer, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs

Killing Cancer When it comes to killing cancer, eradicating every single cell is the goal. But did you know there are different kinds of cancer cells? One specific type, called cancer stem cells (CSC), is catching the eye of drug developers. Discovered more than a decade ago, CSCs are hypothesized to be the drivers of cancer growth and metastasis. Let’s … Read More

DMD Makes The Cut

Emily BurkeAntisense, Biologics, Clinical Trials, Cocktail Fodder, Deoxyribonucleic Acid (DNA), Drug Approvals, Drug Development, Drug Targets, FDA, Genetics, Mechanism of Action, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), The WEEKLY

The Science Behind Sarepta’s Hotly Debated Antisense Drug Sarepta’s (Cambridge, MA) Duchenne muscular dystrophy drug Exondys 51 crossed the finish line earlier this week, with a conditional stamp of approval by the FDA. This hotly debated regulatory result offers new hope for patients and families whose previous treatment options only managed the descent of the disease. Affecting one in approximately 3,500 … Read More

The Tight Junction’s Function

Emily BurkeBiologics, Biotech Basics, Cancer, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, The WEEKLY

A Pathway Lies In The Bridge Between Cells Most of us remember from high school biology that cells are the basic building blocks of all living things. Cells join together to form tissues and organs such as hearts, lungs, and livers, which work together to create a functioning human. An adult human body contains trillions of cells, and these cells must … Read More

The Long And Short Of AMD

Emily BurkeBiologics, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genetics, Mechanism of Action, Monoclonal Antibodies, Term of the Week, The WEEKLY

A Spotlight On One Of The Most Prevalent Eye Diseases It’s easy to take our ability to see for granted. On a day-to-day basis, we rely on vision to process information and to navigate the world without giving it a second thought. For the millions of people affected by eye disease, vision loss, and blindness, impaired vision is a daily reality … Read More

The Next Generation Fight Against CF

Emily BurkeClinical Trials, Drug Approvals, Drug Development, Drug Targets, Easily Confused, Genetics, Mechanism of Action, Orphan Disease, Orphan Drugs, The WEEKLY

TARGETING THE ROOT OF CYSTIC FIBROSIS Innovative therapies targeting the root cause of cystic fibrosis (CF) hit the market several years ago, but those treatments were only for a subset of CF patients.  Now, companies like AbbVie and Vertex may have the potential to treat a large majority of the population—up to 90%—according to clinical trial data. In this issue, we’ll explain … Read More