Earlier this month, the FDA approved an entirely new family of drugs, one so powerful that it could put CRISPR-based gene therapy to shame. Backed by two decades of research and a Nobel Prize, these drugs have the ability to cure inherited diseases—and do so without actually needing to edit the delicate genome.
The green-lit therapy is patisiran, a drug ten years in the making by the Cambridge-based Alnylam for genetic nerve damage. But in this rare case, the nature of the drug is perhaps more significant: patisiran is based on a technology called RNA interference, or RNAi, which allows doctors to silence genes that aren’t properly functioning.
Patisiran is just the tip of the iceberg. In theory, RNAi has the potential to cure any disease caused by “bad” proteins, such as stroke, Alzheimer’s, high cholesterol, or other neurodegenerative disorders. The potential is so grand that scientists have long dubbed the technology a way to “drug the undruggable.”
“This approval is part of a broader wave of advances that allow us to treat disease by actually targeting the root cause, enabling us to arrest or reverse a condition, rather than only being able to slow its progression or treat its symptoms,” said FDA commissioner Scott Gottlieb in a press release. “New technologies like RNA inhibitors, that alter the genetic drivers of a disease, have the potential to transform medicine, so we can better confront and even cure debilitating illnesses.”
With other RNA-based therapies hurtling down the drug development pipeline, it’s likely we’ll be seeing more of these drugs soon. Here’s what you need to know.
Image Credit: Shutterstock
News This Week
Immune Aspects of Biopharmaceuticals and Nanomedicines 1st Edition Raj Bawa, Janos Szebeni, Thomas J Webster, Gerald F. Audette The enormous advances in the immunology of biotherapeutics and nanomedicines in the past two decades have [...]
LAS CRUCES, N.M. — Members of an independent NASA safety panel said they were worried that the Oct. 11 Soyuz launch failure could make safety concerns with the agency's commercial crew program even worse. [...]
Canadian astronaut Col. Chris Hadfield (Ret.) on Friday said that human travel to space will happen sooner than we think, adding that the first destination will likely be a return trip to the moon. [...]
Antibody-based imaging of a particularly aggressive form of breast cancer is undergoing clinical trials worldwide, but the path from trial to application is being hampered by a major obstacle: safety. Concerns stem from inefficient [...]
Following the discovery of graphene in 2003, there has been considerable interest in other types of 2D materials. However, splitting a bulk crystal material into 2D flakes for use in electronics has proven hard [...]
The digital economy is set to unlock tremendous economic value for countries over time. But a common setback for the use of various new technologies is their vulnerability to hackers. That's because companies and [...]
Nanomedical Device and Systems Design: Challenges, Possibilities, Visions now available to rent on Kindle
To accommodate students who wish to read the book at an affordable cost, Nanomedical Device and Systems Design: Challenges, Possibilities, Visions by Frank Boehm (CEO NanoApps Medical Inc.) is available to rent on Kindle. This book benefits [...]
The last five years has seen a surge of attacks on the healthcare industry, with the largest breaches impacting as many as 80 million people. In July this year, it was revealed that 150,000 [...]
The Nanoaugmented Materials Industry Summit (NAUM) 2018 in Shanghai on October 31 will gather the world’s leading companies who use and apply graphene nanotubes to share exciting results. This year the event is opening [...]
Any truck operator knows that hydraulics do the heavy lifting. Water does the work because it's nearly incompressible at normal scales. But things behave strangely in nanotechnology, the control of materials at the scale [...]
Myeloperoxidase - an enzyme naturally found in our lungs - can biodegrade pristine graphene, according to the latest discovery of Graphene Flagship partners in CNRS, University of Strasbourg (France), Karolinska Institute (Sweden) and University [...]
In today’s hospitals and healthcare clinics, a new doctor’s new assistant is now often on the job — in the form of artificial intelligence. Whether it’s analyzing medical images or guiding robots that assist [...]
Healthcare cybersecurity will be one of the top 10 challenges, issues, and/or opportunities facing healthcare executives next year, according to a new poll by the Healthcare Executive Group (HCEG). Healthcare cybersecurity covers such issues [...]
A research team has developed a nano-platform technology that works in combination with existing chemotherapeutic drugs that may reverse drug-resistance in renal cell carcinoma. Drug resistance to chemotherapy is a significant clinical and financial [...]