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

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

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 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

Unmasking Multiple Sclerosis

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

 Many Channels Available To Impede MS Progression Continuing our series on central nervous system (CNS) disorders—previously covering Alzheimer’s and Huntington’s—we pivot to unmask Multiple Sclerosis this week. Famous faces suffering from Multiple Sclerosis (MS) include former talk show host Montel Williams and Sopranos star Jamie-Lynn Sigler. MS typically occurs in susceptible individuals between the ages of 20 and 50, and there are … Read More

Attacking All Angles Of Alzheimer’s

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

Alzheimer’s Pipeline Roundup Alzheimer’s disease ranks as one of the toughest nuts to crack within drug discovery and development. Current treatments merely manage symptoms, so finding a better solution becomes more and more urgent as the aging population grows. The pathology most commonly associated with Alzheimer’s disease (AD) is the buildup of amyloid-beta (Aβ) plaques in the brain. Recent research from … Read More

A Drop Of Biotech

Emily BurkeCancer, Clinical Trials, Cocktail Fodder, Diagnostics, Drug Development, Genomics, HIV, Orphan Disease, Orphan Drugs, The WEEKLY

Basics of Blood Disorders Blood is the carrier of a multitude of fundamental body processes—responsible for delivering vital nutrients/oxygen and for removing wastes. Like the highway exchange feeding a city of life, blood is simply essential. The branch of medicine concerned with the study, diagnosis, treatment, and prevention of blood related diseases is known as hematology. This single therapeutic area covers a broad range … Read More

Inching Across The Blood-Brain Barrier

Emily BurkeBiologics, Biotech Basics, Cocktail Fodder, Drug Development, Drug Targets, Monoclonal Antibodies, Term of the Week

A Constant Hurdle in Drug Delivery When it comes to achieving success in drug development, picking the right drug target and developing an effective inhibitor (or activator) is only half the battle. A drug candidate may appear promising in cell-based testing—and even in preclinical testing—but still fail to work in humans. It simply does not get to where it needs to be … Read More