Chaperoning The Rare Disease Dance

Emily BurkeThe WEEKLY

PROPERLY FOLDING MISFOLDED DISEASE PROTEINS Amicus Therapeutics (Cranbury, NJ) found itself in the news earlier this month when the FDA agreed to review the company’s new drug application for their investigational therapy to treat Fabry’s disease. The drug under consideration, migalastat, has already been approved by the European Medicines Agency. It belongs to a small, but growing class of therapeutics known … 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

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

Orphan Drugs Lead 2015 Approvals

Emily BurkeBiologics, Cardiovascular Disease, Drug Approvals, FDA, Mechanism of Action, Monoclonal Antibodies, Orphan Disease, Orphan Drugs, Small Molecule Drugs, The WEEKLY

Treatment Options Broaden Driven by 19 new orphan approvals, a whopping 45 novel drugs were cleared for the marketplace in 2015. Additionally, new approvals opened up treatment options for Americans with diseases ranging from breast and lung cancers to irritable bowel syndrome. According to FDA numbers: 19 were for orphan diseases that affect fewer than 200,000 patients per year in the US. … Read More

Is Genetic Variety The Spice Of Life?

Emily BurkeBiotech Basics, Cancer, Cardiovascular Disease, Cocktail Fodder, Deoxyribonucleic Acid (DNA), Genetics, The WEEKLY

Understanding Basis Of Evolution Take a look at the person closest to you. Compare the color of their eyes, the texture of their hair, even the complexion of their skin to your own. Do you notice a lot of differences? Genetic variation accounts for the dissimilarities we observe between individuals—seemingly trivial ones such as the differences in eye, hair, and … Read More

Targeting The Cause Of Cystic Fibrosis

Emily BurkeBiologics, Clinical Trials, Drug Approvals, Drug Development, Drug Targets, FDA, Orphan Disease, Orphan Drugs, Small Molecule Drugs, The WEEKLY

Biotech Changes The Game The FDA approval of Vertex Pharmaceutical’s (Boston, MA) Orkambi last week further shifted the paradigm of treating cystic fibrosis (CF) for up to half of its sufferers—moving from a management of symptoms approach to targeting the underlying cause. Until very recently, the only strategy against CF involved reducing the risk of lung infections by taking mucus thinning medications and antibiotics—and rarely, late stage bilateral lung transplants. CF … Read More

Ready. Set. CRISPR.

Emily BurkeBiotech Basics, Business of Biotech, Cancer, Deoxyribonucleic Acid (DNA), Easily Confused, Genetics, Genomics, HIV, Ribonucleic Acid (RNA), The WEEKLY

CRISPR/CAS9 TECHNOLOGY BUILDS UP STEAM The world definitely weighed in after Chinese scientists published a paper detailing the use of CRISPR/Cas9 to edit nonviable human embryo genomes. While the embryos used were never intended to become a living human being, this controversial milestone created a firestorm of opinion. Tweets, blogs, and mainstream news stories were abuzz about the opening of Pandora’s box. … Read More

New Brain Cells From A Pill

Emily BurkeBusiness of Biotech, Clinical Trials, Drug Development, Genetics, Small Molecule Drugs, Term of the Week

Imagine being able to regenerate your brain cells by swallowing a pill. The potential treatment relies on a process known as neurogenesis—”neuro” meaning nerves and “genesis” meaning creation. Adults suffering from neurological diseases or brain disorders may one day benefit from small molecule activators to promote the birth of neurons in the brain. Neurogenesis naturally occurs during fetal brain development and declines with … Read More

The State Of Cystic Fibrosis And Precision Medicine

Emily BurkeCancer, Clinical Trials, Drug Approvals, Drug Development, Drug Targets, Easily Confused, FDA, Genetics, Genomics, Mechanism of Action, Orphan Disease, Orphan Drugs, Small Molecule Drugs, The WEEKLY

During President Obama’s State of the Union address last month, a cystic fibrosis patient named Bill Elder sat beside First Lady Michelle Obama. Diagnosed with the disease at 8 years old, Mr. Elder is “healthier now than ever before” at age 27, thanks to Vertex’s (Boston, MA) Kalydeco. As a third-year medical student, he is not only surviving, but thriving. Receiving an invitation to … Read More

Mutations And Disease: The Spice Of Life

Emily BurkeBiologics, Biotech Basics, Cancer, Cardiovascular Disease, Cocktail Fodder, Drug Targets, FDA, Genetics, Mechanism of Action, The WEEKLY

This week’s issue features excerpts from the “Genetic Variation” chapter of The Biotech Primer, our 200-page book that provides an in-depth look at the biotech industry and the science that drives it. In this chapter, we explain the different types and causes of genetic mutations and then explore their relationship to disease and therapeutics. Today, we begin with a discussion … Read More