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

Post-translational Modifications In Biotech

Emily BurkeBiologics, Biomanufacturing, Biotech Basics, Cancer, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies

Basic Science Meets Application “Post-translational modification” may not be a term you hear every day, but it is critically important to the biotech industry—from understanding how cancer develops to producing biologics. Breaking the term down, we know that: “post” means “after.” “translation” is “the cellular process of making proteins.” “modification” means “change.” So, a post-translational modification (PTM) simply means that … 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