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

Zinc Finger Nucleases

Emily BurkeGene Therapy, Genetics

CATCHING THE RIGHT BREAK How are ZFNs made? To start, zinc finger proteins (ZFP) are sequence-specific, DNA-binding proteins that activate gene expression. They are engineered to recognize unique sites within the genome. While ZFPs do not have the ability to cut DNA on their own, scientists can fuse a ZFP together with a DNA-cutting enzyme called nuclease—the “N” in ZFN. The marriage of ZFP … Read More

Off-Color: The Science Behind Color Vision Deficiency

Emily BurkeGenetics, Medical Device, Pharmacogenomics, The WEEKLY

You’re at the supermarket, puzzling over whether those peaches for the pie are ripe. Maybe you’re watching your child’s soccer team, and struggling to separate the Green Hornets from the Scarlet Knights. As if determining offsides isn’t hard enough! Or more seriously, you’re approaching a stoplight on a busy street and can’t tell if the signal is red or green. … 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

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

Breast Cancer Subtypes

Emily BurkeBiotech Basics, Cancer, Genetics, Mechanism of Action

FOUR MOLECULAR VARIANTS EXPLAINED Hearing your doctor utter the words HER2-positive, HR-positive, triple-negative, or BRCA mutation can be devastating — even for the most resilient person. Simply put, breast cancer is a complex disease. A diagnosis can be derived from any combination of the factors listed above — or, none at all. The National Cancer Institute (Bethesda, MD) has outlined four molecular subtypes of the disease. … Read More

Decoding Your Genes

Emily BurkeBiotech Basics, Deoxyribonucleic Acid (DNA), Diagnostics, Drug Approvals, Easily Confused, FDA, Genetics, Genomics, Orphan Disease, Term of the Week, The WEEKLY

The Skinny On DNA Testing 23andMe (Mountain View, CA) recently found itself back in the limelight after the disease risk section of its mail-in DNA kit received an OK from the FDA. The Silicon Valley biotech had to halt sales of its direct-to-consumer genetics testing back in 2013 after regulatory officials grew concerned over marketing claims and the possibility of consumers misinterpreting the test results. 23andMe rebooted a limited part of … 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

GMO Apples Hit Marketplace

Emily BurkeGenetics, GMO, Ribonucleic Acid (RNA), The WEEKLY

Coming Soon To A Supermarket Near You Coming soon to a supermarket near you: apples that have been genetically modified to resist browning.  Dubbed Arctic Apples, the plants were approved two years ago by the USDA and will begin appearing on select retailers’ shelves in Midwestern U.S. states this month. Developed by Okanagan Specialty Fruits (Summerland, B.C., Canada), the Arctic Apple also … 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 Long And Short Of AMD

Emily BurkeBiologics, Clinical Trials, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Genetics, Mechanism of Action, Monoclonal Antibodies, Term of the Week, The WEEKLY

A Spotlight On One Of The Most Prevalent Eye Diseases It’s easy to take our ability to see for granted. On a day-to-day basis, we rely on vision to process information and to navigate the world without giving it a second thought. For the millions of people affected by eye disease, vision loss, and blindness, impaired vision is a daily reality … 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

Pushing The Self-Destruct Button

Emily BurkeCancer, Drug Approvals, FDA, Genetics, Mechanism of Action, Monoclonal Antibodies, Small Molecule Drugs, The WEEKLY

 Pushing the Self-Destruct Button Breakthrough drug Venclexta charged onto the marketplace three months early to battle chronic lymphocytic leukemia. AbbVie’s (North Chicago, IL) and Roche’s (Basel, Switzerland) new therapy gained a quick approval after 80% of patients in the 106-person clinical trial responded to the small molecule inhibitor. Chronic lymphocytic leukemia (CLL) is the most common type of leukemia in adults, with approximately … Read More

On The Hunt

Emily BurkeBiotech Basics, Drug Development, Drug Targets, Genetics, Genomics, Mechanism of Action, Orphan Disease, Orphan Drugs, Term of the Week, The WEEKLY

The Hurdles Of Huntington’s The nervous system is an incredibly complex piece of human machinery, stretching to the far reaches of the body while controlling and receiving the nuances of life from a central command station. Just like any part of the human body, the central nervous system (CNS) is affected by various diseases that are sometimes not entirely understood. After … Read More

Biotech Battles Zika

Emily BurkeDeoxyribonucleic Acid (DNA), Genetics, GMO, Term of the Week, The WEEKLY

Latest Mosquito-Borne Disease Outbreak The buzz around the Zika virus isn’t going to die down anytime soon. The global health threat is dominating the news while the Brazilian outbreak threatens to spread to the rest of the Americas. As the ramifications of the virus emerge every passing day, we here at Biotech Primer WEEKLY wonder: what is biotech doing to fight Zika? In this issue, … Read More