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

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

Stem Cell Snapshot

Emily BurkeBiologics, Cardiovascular Disease, Clinical Trials, Drug Development, Drug Targets, Genetics, Genomics, Mechanism of Action

Inducing Stem Cells To Heal Headlines touting stem cells often claim the therapies heal everything from hair loss to hearing loss. While many of these treatments are not FDA approved, there are some promising innovations winding through preclinical and clinical development. Here at WEEKLY headquarters, we like to tease out the science behind the scene, so let’s review regenerative medicine basics and survey … Read More

Eye Of The Cytokine Storm

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

Understanding CAR-T Safety Chimeric antigen receptor therapy (CAR-T) is our top technology pick of 2016, blazing trails in drug development with its innovative approach of using engineered immune cells to fight cancer. Despite its success in clinical trials, safety concerns remain. Serious adverse events, including death, have occurred as a result of “cytokine storms” during CAR-T treatments. We reviewed the basics of CAR-T in last week’s … Read More

Unwiring The Biology Of Fibrosis

Emily BurkeAntisense, Biologics, Clinical Trials, Drug Development, Drug Targets, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs, Term of the Week, The WEEKLY

Fibrotic Disease Innovations Fibrotic diseases — organ and tissue disorders that occur as a result of the buildup of excessive scar tissue — are sounding the alarm in the biotech sector. A silent epidemic creeping up on the Western world involves non-alcoholic steatohepatitis (NASH), or liver fibrosis associated with obesity and type 2 diabetes. Left untreated, NASH can lead to liver failure. Currently, … Read More

The Science Behind The Opioid Epidemic

Emily BurkeBiotech Basics, Clinical Trials, Cocktail Fodder, Drug Development, Drug Targets, Mechanism of Action, Small Molecule Drugs

A Big Pain The opioid addiction crisis gained attention at the highest levels of U.S. policy circles this past year. Presidential candidates that disagreed on nearly everything else vowed to make fighting the epidemic a priority if elected. In July, the U.S. Senate overwhelmingly approved a bill to strengthen prevention, treatment, and recovery efforts. And no wonder – according to … Read More

The Molecular Cause Of Obesity

Emily BurkeClinical Trials, Diabetes, Drug Development, Drug Targets, Mechanism of Action, Term of the Week

Targeting Fat Obesity is considered one of the most pressing public health issues of the day. According to the Center for Disease Control, 37% of adults and 17% of children in the U.S. are obese. The latest drug interventions work by attempting to suppress food intake, which has proven beneficial for some. However, there is still a large unmet need … 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

The Next Generation Fight Against CF

Emily BurkeClinical Trials, Drug Approvals, Drug Development, Drug Targets, Easily Confused, Genetics, Mechanism of Action, Orphan Disease, Orphan Drugs, The WEEKLY

TARGETING THE ROOT OF CYSTIC FIBROSIS Innovative therapies targeting the root cause of cystic fibrosis (CF) hit the market several years ago, but those treatments were only for a subset of CF patients.  Now, companies like AbbVie and Vertex may have the potential to treat a large majority of the population—up to 90%—according to clinical trial data. In this issue, we’ll explain … Read More

A Drop Of Gene Therapy

Emily BurkeBiologics, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genetics, Mechanism of Action, Orphan Disease, Orphan Drugs, The WEEKLY

A Walk In The Park For Hemophilia? Imagine tripping over your feet during a leisurely stroll down the sidewalk. Ouch! Your knees are scraped below your shorts and blood starts to drip. A quick wipe from a conveniently pocketed napkin and soon enough, you are the proud owner of some new scabs. Life goes on. If you are one of … Read More

Next-Generation CAR-T

Emily BurkeBiologics, Business of Biotech, Cancer, Clinical Trials, Drug Development, Drug Targets, Genomics, Mechanism of Action, Monoclonal Antibodies, The WEEKLY

The Race Is On Our last WEEKLY—Hacking the Immune Response—unveiled the science behind CAR-T and TCR, two immunotherapies under the microscope of the mainstream press. The well-deserved media attention highlights the ability of these “living drugs” to recognize and obliterate cancers. With all of the early phase clinical success, a few challenges have popped up: • Safety: CAR-T and TCR can … Read More

Attacking All Angles Of Alzheimer’s

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

Alzheimer’s Pipeline Roundup Alzheimer’s disease ranks as one of the toughest nuts to crack within drug discovery and development. Current treatments merely manage symptoms, so finding a better solution becomes more and more urgent as the aging population grows. The pathology most commonly associated with Alzheimer’s disease (AD) is the buildup of amyloid-beta (Aβ) plaques in the brain. Recent research from … Read More

Interrupting HIV

Emily BurkeBiologics, Clinical Trials, Drug Development, Drug Targets, Genomics, HIV, Mechanism of Action, Monoclonal Antibodies, Ribonucleic Acid (RNA), Small Molecule Drugs, The WEEKLY

Therapies In HIV Pipeline Medicine has made gigantic strides in understanding and treating the human immunodeficiency virus (HIV) since its emergence in the early 1980s. Thanks to robust drug discovery efforts, today HIV is managed as a chronic disease instead of the death sentence of years past. The secret to HIV’s infectious success lies in its high mutation rate. Developing drug resistance over … Read More

Feeding Cancer

Emily BurkeBiotech Basics, Cancer, Clinical Trials, Deoxyribonucleic Acid (DNA), Diabetes, Drug Development, Drug Targets, Mechanism of Action, Term of the Week, The WEEKLY

The Metabolics Of Cancer Tackling cancer requires a multi-pronged strategy. Currently, many of the cancer drugs on the market work by inhibiting the signaling pathways that activate cell division. These pathways are often the root cause of cancer—a mutated gene leads to a dysfunctional signaling protein which tells the cell to divide inappropriately. In these cases, the cancer may be treated by inhibiting the … 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