From Drug Development to Approval: A Recap

Emily BurkeClinical Trials, Drug Approvals, Drug Development, Drug Discovery, Drug Targets, FDA

From The Lab To The Patient In this issue of the Biotech Primer WEEKLY we will recap the past seven issues that highlight the journey a molecule takes from the lab to the patient. Beginning in the 1980’s, scientists took a new tack in developing drugs. They adopted an approach known as rational drug discovery. Using this methodology, researchers first … Read More

From Drug Development To Approval: Phase IV

Emily BurkeClinical Trials, Cocktail Fodder, Drug Approvals, Drug Development, FDA, Orphan Drugs

Pharma Finish Line: FDA Approval Last week, we focused on the final stage of clinical testing, Phase III trials, where drug developers assess the safety and efficacy of their drug in large patient groups. At the end of Phase III, drug developers face the moment of truth: does the study data support claims that the new drug is both safe … Read More

From Drug Development to Approval: Phase I/II

Emily BurkeClinical Trials, Cocktail Fodder, FDA, The WEEKLY

Phase I and II Clinical Trials Every drug in clinical use today, from the latest CAR-T treatment to older cholesterol-lowering statins, share one thing in common: they have all successfully navigated the rigorous clinical trials process. This is no small feat, as only ~10% of the drugs that enter Phase I testing successfully emerge as marketed products. Those few drugs … Read More

DNA Vaccines Explained

Emily BurkeBiotech Basics, Clinical Trials, Cocktail Fodder, The WEEKLY, Vaccine

MORE ON THE POWERFUL, ELEGANT SIMPLICITY OF VACCINES Last week, we overviewed vaccine development and manufacture, focusing on those that use whole pathogens to protect us from a disease. This week, we examine subunit and polysaccharide vaccines, which use different strategies to fight infection. We also take a brief look at the US Food and Drug Administration (FDA)’s vaccine approval … Read More

Picturing Disease

Emily BurkeBiotech Basics, Cancer, Clinical Trials, Diagnostics, Term of the Week, The WEEKLY

USING MEDICAL IMAGING TO INVESTIGATE DISEASE Medical imaging — using various modalities to take a snapshot of the body’s interior structure — has been around since 1895, with the discovery of X-rays by Wilhelm Roentgen. X-rays are a type of electromagnetic radiation (more on that later!) that are able to pass through soft tissues such as skin, fat, and muscle … Read More

The Multiple Myeloma Landscape

Emily BurkeBiologics, Cancer, Clinical Trials, Drug Development, Drug Targets, Easily Confused, Genetics, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs

BLOOD CANCER: MULTIPLE MYELOMA Plasma cells are the antibody-producing cells of our immune system which happen to play a critical role in our defense against infections. In multiple myeloma, plasma cells begin to grow and divide in an uncontrolled manner, forming a cancerous mass known as a plasmacytoma. Marrow — which produces plasma — no longer functions in our defense, it simply takes … Read More

Eye Of The Cytokine Storm

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

THE FLASH OF THE FIRST CAR-T Last week’s much anticipated FDA approval of the first chimeric antigen receptor T-cell (CAR-T) therapy for acute lymphocytic leukemia hails as the first gene therapy on the US  market. Classified as a “cell-based gene therapy,” Novartis’ (Basel, Switzerland) Kymriah works by removing patients’ T-cells, using a viral vector to introduce a gene that will allow the … Read More

Vaccines: Schooling The Herd

Emily BurkeBiologics, Biotech Basics, Clinical Trials, Drug Development, Mechanism of Action, The WEEKLY

VACCINATION NATION Back to school means shopping for new school supplies, adjusting to a new schedule, and making sure all required vaccinations are up to date. Every state requires school-age children to be vaccinated against certain infectious diseases including tetanus, hepatitis B, measles, mumps, rubella, polio, pertussis (whooping cough), and chicken pox. Vaccination policies are highly effective at eliminating many … Read More

The Science Of CRISPR/Cas9

Emily BurkeBiologics, Business of Biotech, Cancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genetics, Genomics, Mechanism of Action, Ribonucleic Acid (RNA)

CRISPR/CAS9 UPDATE As CRISPR/Cas9 adds new indications to its resume, legal battles over its IP continue to be waged in the US and Europe. On the clinical front, CRISPR/Cas9 entered its first human trial at Sichuan University (Chengdu, China) last fall for metastatic lung cancer, and is widely expected to do so in the U.S. by the end of the year. This … Read More

A Skin Cell With Stem Cell Diversity?

Emily BurkeBiologics, Clinical Trials, Drug Development, Drug Targets, Term of the Week, The WEEKLY

INDUCED PLURIPOTENT STEM CELLS SHOW PROMISE Imagine being able to reprogram one of your own skin cells to produce a functioning nerve cell or section of cardiac tissue. This may sound like science fiction — but the groundwork for this to become a reality is already in the works as researchers expand their ability to create and manipulate induced pluripotent stem … Read More

Epigenome: Writing, Reading & Erasing

Emily BurkeCancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genomics, Mechanism of Action, Small Molecule Drugs

FOUNDATIONS OF EPIGENETICS Genetic mutations — changes in the order of the A, C, G, and T nucleotide bases that make up a gene — have been the primary focus of cancer researchers over the last several decades. By sussing out mutations involved in regulating cell growth and division, scientists better understand the molecular range of different cancers and consequently develop … Read More

Nanobodies: These Are Not Your Mother’s mAbs

Emily BurkeBiologics, Biotech Basics, Cancer, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Orphan Disease, Orphan Drugs

The Drug Kingpins Monoclonal antibodies (mAbs) are the undisputed drug kingpins. In 2013, the mAb market raked in $75 billion in combined sales, covering a whole range of indications from cancer and infectious disease, to autoimmune disorders, and even high cholesterol. Despite the success, mAbs have one chink in their armor: they cannot enter cells due to their large size, hampering their range … 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

The Science Of CRISPR/CAS9

Emily BurkeBiologics, Business of Biotech, Cancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genomics, Mechanism of Action, Ribonucleic Acid (RNA), The WEEKLY

 CRISPR/CAS9 UPDATE CRISPR/Cas9 can’t seem to stay out of the news — from first in human to patent disputes, we here at the WEEKLY want to update you on this hot technology. A group of scientists from the State Key Laboratory of Proteomics (Beijing, China) and the National Center for Protein Sciences (Beijing, China) recently reported the first ever edit using CRISPR/Cas9 in healthy human … Read More

The PARP Race Is On

Emily BurkeCancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Approvals, Drug Development, Drug Targets, Easily Confused, FDA, Mechanism of Action, The WEEKLY

PARP1 INHIBITOR LINEUP PARP1 inhibitors are making a strong statement! Tesaro’s (Waltham, MA) just-approved Zejula has garnered predictions of blockbuster status. AstraZeneca’s (Cambridge, UK) Lynparza was the first PARP1 inhibitor to make it to market back in 2014, and their recent clinical trial results showed significant survival benefit in ovarian cancer. Clovis Oncology (Boulder, CO) achieved the second FDA approval of a PARP1 inhibitor with Rubraca in … Read More