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

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

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

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

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

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

Drugging The Undruggable

Emily BurkeBiotech Basics, Business of Biotech, Drug Development, Drug Targets, Mechanism of Action, Ribonucleic Acid (RNA), Small Molecule Drugs, Term of the Week, The WEEKLY

Antagonists Fight A Good Fight Small molecule inhibitors, also known as antagonists in the industry, fight a good fight. In fact, many drugs on the market today work by inhibiting overactive, disease-associated proteins. Novartis’ (Basel, Switzerland) top selling leukemia drug Gleevec, for example, is a small molecule inhibitor of a protein called Bcr-Abl, whose overactivity promotes excessive cell division. The downside? It is … Read More

RNAi Crashes The PCSK9 Party

Emily BurkeBiologics, Business of Biotech, Cardiovascular Disease, Clinical Trials, Drug Approvals, Drug Development, Drug Targets, FDA, Mechanism of Action, Monoclonal Antibodies, Ribonucleic Acid (RNA), The WEEKLY

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. Enter Alnylam (Cambridge, MA), with its RNAi-based experimental drug ALN-PSCsc, which just completed Phase I with positive results. What makes PCSK9 inhibitors so hot, and why are the new cholesterol drugs on the market … Read More

What The Heck Is qRT-PCR Diagnostics?

Emily BurkeBiotech Basics, Cancer, Cocktail Fodder, Deoxyribonucleic Acid (DNA), Diagnostics, Easily Confused, Genetics, Genomics, HIV, Ribonucleic Acid (RNA), The WEEKLY

From Impossible to possible The genetics of cancer has progressed from the impossible to a multifaceted mountain of possible. Comprehensive projects in whole genome sequencing and tumor genome sequencing are providing data to unravel the genetic predispositions of cancer. The other half of possible lies in quantitative PCR. This platform technology can identify types of cancer, effective therapeutics, and aggressiveness of the … Read More

Antisense, RNAi And MicroRNA Explained

Emily BurkeAntisense, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Mechanism of Action, Ribonucleic Acid (RNA), The WEEKLY

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 by the cell. Fighting the good fight are antisense, RNAi, and microRNA. With their high specificity and relative … Read More