RNA Therapeutics March Onward

RNA Therapeutics March Onward

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...
New Hope For Spinal Muscular Atrophy

New Hope For Spinal Muscular Atrophy

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...
Exon-Skipping Therapy For DMD

Exon-Skipping Therapy For DMD

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...
Inching Across The Blood-Brain Barrier

Inching Across The Blood-Brain Barrier

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...