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...
The ABCs of DNA & RNA

The ABCs of DNA & RNA

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...
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...
RNAi Crashes The PCSK9 Party

RNAi Crashes The PCSK9 Party

Newest Hopeful In Cholesterol Lowering Landscape Just weeks after the biotech world celebrated the approvals of two new cholesterol-lowering PCSK9 inhibitors, Regeneron/Sanofi’s Praulent and Amgen’s Repatha, a potential future rival arrived in style....
Antisense, RNAi And MicroRNA Explained

Antisense, RNAi And MicroRNA Explained

Make Way For RNA Based Therapies The up hill battle of RNA therapeutics to the clinic continues despite extensive use in research. Recall from high school biology that RNA translates DNA code into a language ribosomes can understand in order to make proteins required...

RNAi Advantage Goes To Dicerna

THE SCIENCE BEHIND THE IPO CRAZE: OUR FOCUS FOR THE NEXT FEW WEEKS I want to thank you for subscribing to Biotech Primer WEEKLY. Now, imagine yourself on the set of Jeopardy! facing this clue: “We saw 45 in 2013, and there’s been 17 so far this...