Drug Discovery 301

Emily BurkeBiologics, Biomanufacturing, Cocktail Fodder

DRUG DISCOVERY 301 Biotech Primer Weekly wrapped up last year by exploring the first two stages of drug discovery. We looked at how pharmaceutical companies identify drug targets, or the molecules (usually proteins) involved in an illness that an ‘as yet undeveloped drug’ will hopefully act on. Next, we examined how researchers develop those pharmaceutical candidates. Now we turn to … Read More

Gene Therapy Cures

Emily BurkeBiologics, Gene Therapy, Genetics, Term of the Week

The Promise of Gene Therapy Unfolds In many ways, 2017 was the year of gene therapy in the United States. Patients and pharmaceutical companies celebrated the approval of not one, but three treatments for otherwise untreatable health conditions. Researchers have been working on developing safe, effective gene therapies for three decades. Early trials were plagued with safety issues. Consequently, the … Read More

Vaccines: Powerful Simplicity

Emily BurkeBiologics, Biomanufacturing, Cocktail Fodder, The WEEKLY, Vaccine

Vaccines: Elegant, Powerful Simplicity Anyone who’s suffered the aches and fever of influenza has good reason to value the simple flu shot. In fact, millions roll up their sleeves and literally take their medicine. The US Centers for Disease Control (CDC) (Atlanta, GA) estimates that about 146 million doses of influenza vaccine went to doctors’ offices, health departments, and the … Read More

Circadian Rhythm & Disease

Emily BurkeBiologics, Cancer, Diabetes, Mechanism of Action, The WEEKLY

AND THE BEAT GOES ON Earlier this week, the 2017 Nobel Prize in Physiology or Medicine was awarded to three American scientists (Jeffrey Hall and Michael Rosbash, of Brandeis University, and Michael Young, of Rockefeller University) for their work in deciphering the molecular basis of circadian rhythm – the 24-hour cycle that governs the inner workings of all life on … 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 Microbiome Magnified

Emily BurkeBiologics, Biotech Basics, Drug Development, Drug Targets

DECODING THE GUT-BRAIN AXIS There is no shortage of microbiome-focused startups in biotech right now. The link between the gut microbiome — the entire collection of microbes living in the gut — and diseases such as inflammatory bowel disease are well-established. New research has made it clear, however, that the gut microbiome also impacts neurological health, leading to the phrase “the gut-brain … 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

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

Phage — More Than Just A Phase

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

Old Way Of Fighting Bacteria Renewed One of the greatest public health challenges of the 21st century is antibiotic resistance, which occurs when a few bacteria in a given population develop a genetic mutation that enables them to survive — even in the presence of antibiotics. How do bacteria become drug resistant? Suppose a particular antibiotic inhibits an enzyme required for bacterial replication. … 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 Mechanics Of Melanoma

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

Spectrum Of Therapies Melanoma accounts for less than 1% of skin cancer cases yet causes the vast majority of skin cancer deaths. If detected early enough, melanoma is almost always curable. If not, its ability to metastasize makes it difficult to treat. Melanoma is more common in young adults than many other types of cancer, with 25% of new cases occurring in people under age … Read More

Therapeutic Antibody Primer

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

Basics & Innovations Monoclonal antibody (mAb) therapeutics burst onto the healthcare scene 20 years ago, and today they remain one of the most versatile and effective therapeutics available. The tried and true mAbs are still in high demand, and we suspect this first wave of derivative products clamoring their way through the pipeline will be equally as successful. In this WEEKLY, we’ll … Read More