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 studies for the treatment of spinal muscular atrophy (SMA). In this Weekly, we’ll take a look at the science behind these headlines by explaining exactly … Read More
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 truth: does the study data support claims that the new drug is both safe … Read More
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 cholesterol. Despite the success, mAbs have one chink in their armor: they cannot enter cells due to their large size, hampering their range … Read More
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 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
New Hope For Spinal Muscular Atrophy
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
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
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 Next Generation Fight Against CF
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
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
An Inborn Error Of Metabolism
Diet Soda’s Ominous Warning “Phenylketonurics—contains phenylalanine” is listed on many diet sodas, including Diet Coke and Diet Pepsi. There is no elaboration, just an ominous warning in bold typeface. Since a few of us at BioTech Primer are big fans of diet soda, we wondered: Should we be worried? And what exactly is a phenylketonuric? A quick Google search reveals … Read More
On The Hunt
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
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 syndrome. According to FDA numbers: 19 were for orphan diseases that affect fewer than 200,000 patients per year in the US. … Read More
Deciphering DMD
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
A Drop Of Biotech
Basics of Blood Disorders Blood is the carrier of a multitude of fundamental body processes—responsible for delivering vital nutrients/oxygen and for removing wastes. Like the highway exchange feeding a city of life, blood is simply essential. The branch of medicine concerned with the study, diagnosis, treatment, and prevention of blood related diseases is known as hematology. This single therapeutic area covers a broad range … Read More
Targeting The Cause Of Cystic Fibrosis
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
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 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
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