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

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

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

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

Zika On The Brain

Emily BurkeClinical Trials, Diagnostics, Drug Development, Drug Targets, Mechanism of Action, Ribonucleic Acid (RNA), The WEEKLY

The Latest On The Rise Of Zika The spread of the Zika virus is raising alarm bells in the United States, and Congress has yet to pass any legislation to combat the fight. Zika infections reported in Miami confirm the virus’ stateside arrival, pushing residents to take measures to protect their pregnancies from potentially devastating effects. In our previous issue, we explored the underpinnings … 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

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

Catching The First Drop Of Cancer

Emily BurkeCancer, Deoxyribonucleic Acid (DNA), Diagnostics, FDA, Monoclonal Antibodies, Ribonucleic Acid (RNA), Term of the Week, The WEEKLY

The Latest In Cancer Diagnostics Hearing the words “it might be cancer” paired with your doctor’s perplexed look is enough to send shockwaves through your body. Getting to the heart of a diagnosis usually requires a surgical biopsy—removal and examination of the suspected tissue for visible signs of cancer. Less invasive diagnostic tests—called liquid biopsies—might just bring more choices to … Read More

The Uber Of The Human Body?

Emily BurkeBiologics, Business of Biotech, Cancer, Cardiovascular Disease, Deoxyribonucleic Acid (DNA), Drug Development, Drug Targets, Mechanism of Action, Ribonucleic Acid (RNA), Term of the Week, The WEEKLY

A Tiny Vesicle With Big Potential Cambridge-based startup Codiak BioSciences made headlines last month with $40M launch funding and another $40M if their technology shows promise. So, what’s the big deal? A tiny little particle—once described as a cellular trash truck—called the exosome. First observed in the early 1980s, exosomes were originally thought to be a way for cells to get rid of … Read More

The ABCs Of Hepatitis

Emily BurkeDrug Targets, FDA, Mechanism of Action, Ribonucleic Acid (RNA), Small Molecule Drugs, The WEEKLY

Hepatitis Viruses Explained Curing disease is serious business. Lou Reed, the infamous leader of the rock band Velvet Underground, died in 2013 of complications from hepatitis C and liver disease. Death is the most profound fear for people living with hepatitis C, but with today’s treatments patients can beat this disease. Thank you Gilead’s (Foster City, CA) Harvoni or AbbVie’s (North … Read More

Presenting The New Class Of Transcriptional Therapeutics

Emily BurkeBiotech Basics, Cancer, Deoxyribonucleic Acid (DNA), Drug Development, Easily Confused, Genetics, Genomics, Mechanism of Action, Ribonucleic Acid (RNA), Small Molecule Drugs, Term of the Week, The WEEKLY

DNA: Coding Vs. NON-Coding The completion of the Human Genome Project in 2003 revealed a big surprise: up to 98% of the DNA making up the human genome does not code for proteins! The notion that parts of the genome were non-coding had been circulating for several decades, but when the actual percentage was confirmed it blew the industry’s mind. This … Read More

Deciphering DMD

Emily BurkeAntisense, Clinical Trials, Drug Development, Drug Targets, FDA, Genetics, Orphan Disease, Orphan Drugs, Ribonucleic Acid (RNA), Small Molecule Drugs

THE ROOT OF DUCHENNE MUSCULAR DYSTROPHY World Duchenne Awareness Day called attention to Duchenne Muscular Dystrophy (DMD) this past Monday. Affecting one in 3,500 newborn baby boys worldwide, this fatal disease is caused by a mutation in the gene that codes for the dystrophin protein. Easily damaged muscle cells, progressive muscular weakness, and serious medical problems—including significant impairment of the heart and lungs—are the direct results of this … Read More