Drug Development

 

Drug Discovery 201

WE WANNA NEW DRUG “One that won’t make me sick/ One that won’t make me crash my car/ and make me feel three feet thick…” Huey Lewis is singing about love, but he voices very human concerns when it comes to the medicines that heal bodies and minds....

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Drug Discovery 101

Drug Discovery 101

On the Road to New Medicines For most of the 20th century, we discovered new drugs by trial and error. Scientists investigated countless unrelated compounds in animals to see which improved disease symptoms. For instance, in the 1950s and 60s, British scientists at...

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The Tight Junction’s Function

The Tight Junction’s Function

A Pathway Lies In The Bridge Between Cells As any high school biology teacher will happily remind us, cells are the basic building blocks of all living things: you, the office ficus tree, the yeast that bubbles your beer. In animals, cells join to form tissues and...

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Viruses To The Rescue?

Viruses To The Rescue?

Viruses to the Rescue? Last week, a paper in Nature Medicine described a British teenager whom doctors pulled back from the brink of death. The young woman had developed a deadly, antibiotic-resistant infection following lung surgery. The treatment? Phage therapy, in...

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Circadian Rhythm & Disease

Circadian Rhythm & Disease

AND THE BEAT GOES ON Earlier this week, most areas of North America and Europe observed the annual ritual of “springing forward.” People started setting their clocks ahead one hour in the U.S. in 1918. The federal government aimed to save electricity and increase...

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The Gut-Brain Axis

The Gut-Brain Axis

Decoding The Gut-Brain Axis Earlier this week, a newly published study made headlines for showing a direct link between the gut microbiome and depression. For the past several years, scientists have suspected a link between bugs in the gut and neurological health....

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MICROBIOME ADVENTURES, PART 2

MICROBIOME ADVENTURES, PART 2

The most recent Biotech Primer WEEKLY looked at a particularly productive new area of medicine courtesy of our own tiny personal zoo: the microbiome. We considered how resetting a person’s microbiome with a fecal matter transplant (FMT) aka bacteriotherapy can heal such troubling conditions as antibiotic-resistant C. difficile infections and inflammatory bowel disease (IBD). Thankfully for the millions suffering from these health conditions, acceptance of FMTs is growing. Now the research community is turning its attention to the specific components behind the microbiome’s healing potential. Several biotech companies are already developing more precisely-defined therapeutics. This work will foster better quality control and more comfortable formulations. Who wouldn’t prefer swallowing a capsule to undergoing an enema or colonoscopy?
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The Microbiome Magnified

The Microbiome Magnified

You'll Never Walk Alone Ready for a fascinating, “earthy” truth about ourselves? Our every surface and crevice teems with microbes—inside and out. In fact, bacterial cells in and on our body outnumber human cells by about ten to one. Most people walk around completely...

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Autophagy: The Incredible, Edible Cell?

Autophagy: The Incredible, Edible Cell?

Autophagy: The Incredible, Edible Cell? True, weird fact: our cells are cannibals. Right now, these itty-bitty sacs of vital fluids inside your body are eating themselves. This phenomenon is called autophagy, or “self-eating.” Autophagy Is Awesome This seemingly odd...

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Hacking The Fountain Of Youth

Hacking The Fountain Of Youth

Hacking The Fountain Of Youth Last week, we began to delve into longevity research — the area of biotech which seeks to understand the biomolecular changes that occur with aging and possibly underlie many of the dubious gifts of old age: heart disease, Alzheimer’s and...

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Fountains of Healthy Old Age?

Fountains of Healthy Old Age?

Fountains Of Healthy Old Age? Plenty of famous people have uttered (or supposedly uttered) lots of pithy comments about aging. A couple gems: “Age is mind over matter; if you don’t mind, it don’t matter,” said Satchel Paige.  “We are always the same age inside,”...

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Epigenetics: Writing, Reading, & Erasing

Epigenetics: Writing, Reading, & Erasing

Foundations Of Epigenetics Genetic mutations — changes in the order of the A, C, G, and T nucleotide bases that make up a gene — have been the primary focus of cancer researchers over the last several decades. By sussing out mutations involved in regulating cell...

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Swallowing A Biologic Drug?

Swallowing A Biologic Drug?

Swallowing A Biologic Drug? Over the past two decades, biologic drugs — drugs composed of proteins produced by living cells — have become the safest, most effective top sellers within the pharmaceutical industry. Approved to treat a variety of diseases including...

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Biotech In Space!

Biotech In Space!

Experimenting In Lower Earth's Orbit Drug discovery in space? If this sounds like a page from science fiction, think again. Leading pharmaceutical companies are now collaborating with NASA to run experiments on the International Space Station (ISS). The focus on bone...

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Red Blood Cells: Ready For Double-Duty?

Red Blood Cells: Ready For Double-Duty?

RED BLOOD CELLS: READY FOR DOUBLE-DUTY?   Biotech Primer WEEKLY talks a lot about white blood cells, with good reason. These powerful immune cells defend us against pathogens and have recently been adapted to fight cancer as CAR-T cells. What about the body’s...

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$8.7 Billion SMA Drug Explained

$8.7 Billion SMA Drug Explained

The Science Behind the Deal   Earlier this week, news of the $8.7 billion acquisition of gene therapy company AveXis (Bannockburn, IL) by Novartis (Basel, Switzerland) made big biotech headlines. AveXis’ lead candidate, AVXS-101, is now in Phase III clinical...

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Turning On Cellular Garbage Disposals

Turning On Cellular Garbage Disposals

Proteasomes to the Rescue   Many drugs work by stopping overactive proteins that cause disease. The leukemia drug Gleevec, for example, is a small-molecule inhibitor (antagonist) of the protein Bcr-Abl, whose over-activity promotes excessive cell division. Humira...

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From Drug Development to Approval: A Recap

From The Lab To The Patient In this issue of the Biotech Primer WEEKLY we will recap the past seven issues that highlight the journey a molecule takes from the lab to the patient. Beginning in the 1980's, scientists took a new tack in developing drugs. They adopted an...

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Market Access

Market Access

Market Access Primer For the last few weeks we here at Biotech Primer have tracked the progression of a drug candidate from the lab to the marketplace, where only the fittest survive. Winning at clinical trials means earning an official regulatory approval....

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From Drug Development To Approval: Phase IV

From Drug Development To Approval: Phase IV

Pharma Finish Line: FDA Approval Last week, we focused on the final stage of clinical testing, Phase III trials, where drug developers assess the safety and efficacy of their drug in large patient groups. At the end of Phase III, drug developers face the moment of...

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From Drug Development To Approval: Phase III

From Drug Development To Approval: Phase III

Phase III Is No Guarantee Our last Biotech Primer WEEKLY explored the riskiest part of the human clinical trials pathway: Phase II. About 70% of drugs that enter Phase II never make it out. Most often, it’s because they fail to demonstrate effectiveness. Even making...

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Plants That Heal

Plants That Heal

Nature’s Medicine Cabinet Where does medicine come from? Before it gets to your medicine chest? Before you purchase it from your neighborhood drugstore? Next time you’re hiking through a forest or gazing at your pretty screensaver of the Olympic Peninsula, think of...

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Chaperoning The Rare Disease Dance

Chaperoning The Rare Disease Dance

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...

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Red Blood Cells: Ready For Double-Duty?

Hypoxia-Inducible Factor (HIF) Compounds & Drug Discovery

The Versatility Of HIFs Quite a few headlines touting a term called "HIF compound" or "hypoxia-inducible factor compound” have intrigued us here at WEEKLY headquarters. GlaxoSmithKline (London, England), Akebia Therapeutics (Cambridge, MA) and more have...

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Biotech In Space!

Biotech In Space!

Experimenting In Lower Earth's Orbit Drug discovery in space? If this sounds like a page from science fiction, think again. Leading pharmaceutical companies are now collaborating with NASA to run experiments on the International Space Station (ISS). The focus on bone...

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100 Drug Development Acronyms

100 Drug Development Acronyms

ADME: Absorption, Distribution, Metabolism, Excretion AE: Adverse Event ANDA: Abbreviated New Drug Application API: Active Pharmaceutical Ingredient ATMPs: Advanced Therapy Medicinal Products BLA: Biologic License Application BsUFA: Biosimilar User Fee Act CAT:...

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100 Drug Development Acronyms

The Medicine Machine

Modern Technology & Mother Nature For thousands of years, nature has been the best medicine cabinet around. Natural products are drugs derived from nature, typically plants or microbes, and have been especially useful in fighting cancer and infectious disease....

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Orphan Drugs Lead 2015 Approvals

Orphan Drugs Lead 2015 Approvals

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...

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Exosomes: Tiny Vesicles, Big Potential

Exosomes: Tiny Vesicles, Big Potential

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...

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Natural Products Drug Discovery: A Nobel-Winning Journey

Natural Products Drug Discovery: A Nobel-Winning Journey

Drug Discovery Via Natural Products Half of the 2015 Nobel Prize in Medicine went to Satoshi Omura and William C. Campbell for their work leading to the discovery of the drug avermectin, used in the treatment of parasitic diseases. The other half went to Youyou Tu for...

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Inching Across The Blood-Brain Barrier

Inching Across The Blood-Brain Barrier

A Constant Hurdle in Drug Delivery When it comes to achieving success in drug development, picking the right drug target and developing an effective inhibitor (or activator) is only half the battle. A drug candidate may appear promising in cell-based testing—and even...

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Inching Across The Blood-Brain Barrier

The Science Behind The Blockbuster Drugs Of Tomorrow

Billion Dollar Mechanisms Of Action The Most Promising Drugs of 2015—a Thomson Reuters Cortellis Competitive Intelligence report—includes several new drugs with predicted sales of $1 billion plus by 2019. Those on the list are well on their way to winning or have...

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Orphan Drugs Lead 2015 Approvals

Interchangability Denied To First US Approved Biosimilar

G-CSF: The original Innovator Sandoz's (Princeton, NJ) Zarzio rode the first wave into the U.S. biosimilars market after it received FDA approval last week. Zarzio is similar to Amgen's (Thousand Oaks, CA) Neupogen and both of these medications are recombinant...

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Exosomes: Tiny Vesicles, Big Potential

Biosimilars: Ready or Not, Here They Come

Last week, the FDA's Oncologic Drugs Advisory Committee unanimously recommended Novartis'  biosimilar version of Amgen’s megahit Neupogen (filgrastim), effectively paving the way for the first US biosimilar approval in the near future. Filgrastim is a recombinant...

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First-in-Class: 2014 Drug Approvals

The FDA ushered in six new drugs just before the close of 2014, bringing the final tally to 41– the highest rate since 1996, when the agency approved 53 drugs. 2014 drug approvals were notable not only for their quantity, but also their quality. A number of new drugs...

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Inhaling Insulin Is A Breath Of Fresh Air

INSULIN INHALED Injections are a part of life for most diabetics. Type 1 patients inject insulin into themselves multiple times per day, and as many as 40% of type 2 diabetics require at least one daily injection. Thanks to the FDA's approval of MannKind Corporation’s...

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Mitochondrial Medicine Going Mainstream?

MORE THAN "JUST" A POWERHOUSE When we hear the word mitochondria, most of us remember it described in high school biology class as the “powerhouse” of a cell. It is an apt moniker, and not just because mitochondria are the subcellular compartments that convert glucose...

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Alphabet Soup: Drug Development Acronyms

ALPHABET SOUP: NEW DRUG APPROVALS Acronyms abound in the world of new drug approvals. Check out our cheat sheet below for help with some of the most common: BLA: Biologic Licensing Application Planning on developing a biologic drug? Would you like to bring it to...

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The FDA’s Decidedly Average Year

2013 DECIDEDLY AVERAGE FOR THE FDA The FDA approved 27 new drugs in 2013, down from the 15-year high of 2012 with its 39 approvals. This should not be cause for alarm since 2013 numbers fall in line with the average of 28 over the last five years (FDA.gov). The FDA...

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