Biopharma Industry

 

Immune Checkpoint Inhibitors

A Killer(-T) Nobel Prize In Medicine On October 1, James Allison, now at the M.D. Anderson Cancer Center in Houston, and Tasuku Honjo, now at Kyoto University, won the 2018 Nobel Prize in Physiology or Medicine. The two scientists discovered the...

Articles

Amazing Antibodies Part 2: Enlightened & Nano

What Can't These Little Dudes Do? When last we met, we discussed the fundamentals of monoclonal (mAb) therapies and looked at two recent advances: antibody-drug conjugates and bispecific antibodies. This week continues our adventure in antibody innovation by...

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CRISPR to the People

CRISPR: SNIPPING AWAY AT DISEASE CRISPR genome editing is one of the most exciting developments in biotechnology since its discovery a few years ago. Bacteria use this mechanism to destroy the DNA of invading viruses. Scientists subsequently discovered CRISPR’s...

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Immunotherapy Goes Viral

VIRUSES BLASTING CANCER Last week, Johnson & Johnson (New Brunswick, NJ ) announced that their biotech arm Janssen Labs (Rariton, NJ) is investing up to $1 billion dollars to acquire BeneVir, a small biotech based in Rockville, Maryland, which focuses on oncolytic...

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Gene Therapy Cures

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...

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CAR-NK: Natural Born Cancer-Killers

Further Down the Cancer Treatment Road with CARs This past August, to much fanfare, the FDA approved the first chimeric antigen receptor (CAR) T-cell therapy for blood cancer. Called Kymriah (Novartis), it promises to revolutionize treatment by genetically altering a...

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From Fantasy To Reality: Xenotransplantation

TRANSPLANTING ORGANS FROM ANIMALS INTO HUMANS Every ten minutes, a new person is added to the national transplant waiting list. A little more than 75,000 people are active waiting list candidates — meaning they are medically eligible for transplantation according to...

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Eye Of The Cytokine Storm

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’...

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Putting The CAR-T Before The Horse

THE STORY BEHIND CAR-T The hottest cancer therapy in the pipeline — chimeric antigen receptor therapy (CAR-T) — got a big boost last month when an FDA advisory panel unanimously recommended approval of the treatment for children and young adults with a severe form of...

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Breast Cancer Subtypes

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...

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A Skin Cell With Stem Cell Diversity?

INDUCED PLURIPOTENT STEM CELLS SHOW PROMISE Imagine being able to reprogram one of your own skin cells to produce a functioning nerve cell or section of cardiac tissue. This may sound like science fiction — but the groundwork for this to become a reality is already in...

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Nanobodies: These Are Not Your Mother’s mAbs

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...

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RNA Therapeutics March Onward

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...

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The ABCs of DNA & RNA

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...

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Stem Cell Snapshot

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...

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Eye Of The Cytokine Storm

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...

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Neoantigen Cancer Vaccines

The Elusive Cancer Vaccine The promise of cancer vaccines have proven to be elusive. A new crop of biotechs are hoping to change that by taking advantage of the latest advances in genomics. Scientists are working overtime trying to develop cancer vaccines that train...

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Exon-Skipping Therapy For DMD

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...

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A Drop Of Gene Therapy

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...

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Next-Generation CAR-T

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...

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RNAi Crashes The PCSK9 Party

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...

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Antisense, RNAi And MicroRNA Explained

Make Way For RNA Based Therapies The up hill battle of RNA therapeutics to the clinic continues despite extensive use in research. Recall from high school biology that RNA translates DNA code into a language ribosomes can understand in order to make proteins required...

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The State Of Cystic Fibrosis And Precision Medicine

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...

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Designer Genes: An Introduction To Genome-Editing

Five years ago, altering an individual's genome would have been labeled unimaginable. Fast forward to today and one of the hottest new developments in biotech is genome-editing—the ability to selectively disable or edit the sequence of specific genes. In this WEEKLY...

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Biotech’s Battlefront: Monoclonal Antibodies

Since their premier on the scene, monoclonal antibodies (mAb) have demanded top billing on the biotech marquee, creating a cast of therapeutics used to treat diseases like autoimmune disorders and cancer. The first player debuted in 1986 when Janssen-Cilag's OKT3...

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Pursuing The Promise Of Unlimited Platelets

Universal platelet cells—generated from human induced pluripotent stem cells—made their debut last week, courtesy of Advanced Cell Technology (Marlborough, MA). The possibility of producing platelets—the component of blood that stops bleeding on-demand and...

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Squeezing The Juice Out Of Drug Metabolism

When popping a pill, we seldom think about what happens next—to the pill, or even to our bodies. We assume the body welcomes any extra help to fix the problem, but the reaction is quite contrary. A swallowed pill (small molecule drug) is instantly labeled by our body...

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Tailoring Stem Cells To Fashion Replacement Organs

If stem cells have their way, replacement organs may find their place as a plentiful standard of treatment. This pairing prompted us to wonder: what is it about stem cells that make them attractive to organ replacement therapies? The therapeutic potential of stem...

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Therascreen: New Companion Diagnostic

NEW COMPANION DIAGNOSTIC APPROVAL Colorectal cancer is the third most common cancer found in both men and women in the U.S., and is the second leading cause of cancer deaths. Approximately 1.2 million cases of colorectal cancer are expected to occur globally. Just...

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Genome Editing: Curing HIV?

CURING HIV? HIV destroys its victim’s immune system by infecting T-cells, a type of white blood cell critical for immunity. HIV binds to two different proteins on the T-cell’s surface: CD4 and CCR5. In the late 1990s, scientists identified a population of individuals...

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RNAi Advantage Goes To Dicerna

THE SCIENCE BEHIND THE IPO CRAZE: OUR FOCUS FOR THE NEXT FEW WEEKS I want to thank you for subscribing to Biotech Primer WEEKLY. Now, imagine yourself on the set of Jeopardy! facing this clue: "We saw 45 in 2013, and there's been 17 so far this year." The correct...

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