RNA Therapeutics Are Coming of Age


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RNA therapeutics are new, innovative high-impact medicines. The combined efforts of pioneering biotechnology companies, pharmaceutical and academic institutions have helped these treatments, which act on ribonucleic acids in the cell, to evolve and come of age.

For those unfamiliar, these medicines are designed to target RNA and can ultimately affect many cellular components. They can provide numerous distinct advantages when compared to conventional methods of targeting proteins with small-molecule chemical compounds, or large-molecule protein drugs like antibodies. By targeting RNA, scientists now have the opportunity to develop drugs for a plethora of diseases which can’t be treated today with conventional methods. Moreover, RNA therapies have the potential to synergize with existing therapies and can address severe unmet medical needs such as cancer, cardiovascular disorders and autoimmune diseases.

The world of RNA therapeutics consists of several different kinds of technologies, including antisense therapies, RNA interference (RNAi) therapeutics, nucleic acid or peptide aptamers and microRNA therapeutics. RNA is one of three major macromolecules in a cell and is made up of a long chain of nucleotides. It is a single-stranded molecule that can adopt very complex three-dimensional structures and can affect many cellular functions. RNA is a critical molecule involved in many cell activities. RNA may be the first molecule that allowed life to pass genetic information from one generation to the next. There is also evidence that RNA may have been the primordial source of life.

However, a key question has always been: can targeting RNA translate into first-in-class, powerful therapeutic drugs? For more than two decades, academic institutions, biotech and large pharmaceutical companies have been investigating the answer to this question.

Deregulation of protein expression may be a primary cause of chronic illnesses such as cancer, cardiovascular and autoimmune diseases. Therefore, therapies that may directly inhibit the overexpression of the disease-causing proteins may address these chronic illnesses. RNA therapies aim to prevent the translation of these proteins and ultimately block their production.

RNA therapeutics represent an exciting and potentially transformative approach to developing new treatment regimens. Part of the appeal with RNA therapies is that while they can be effective on their own, they can work in combination with existing treatments. That means RNA therapeutics do not have to necessarily displace existing therapies to provide a benefit to patients.

In the world of drug discovery and development, full of small molecules and larger protein drugs, typically there is one established target and numerous drug candidates competing to hit the target with the best combination of safety and effectiveness. However, there is still a very large space of unmet medical need. Oral drugs cover a significant portion of drug targets, followed by non-oral drugs, such as monoclonal antibodies. But collectively, these types of therapies can address only a small portion of all potential targets. Targeting RNA has created the ability to venture into a whole new space, essentially covering the entire genome, with new mechanisms of action that can be combined with current therapies.

Antisense Therapeutics

There are multiple ways for these nucleic acid therapies to affect a particular target. With antisense therapies, such as those pioneered by Carlsbad, CA-based Isis Pharmaceuticals (NASDAQ: ISIS), the goal is … Next Page »

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Kleanthis G. Xanthopoulos, Ph.D. is the former president and CEO of Regulus Therapeutics, a company developing drugs based on microRNA technology. Follow @

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3 responses to “RNA Therapeutics Are Coming of Age”

  1. Kyle Smith says:

    why NO mention of delivery RNAI stocks TKMR or ARWR

    • Kevin CC says:

      Because author has ties with Regulus which is tight with ALNY which is not tight with TKMR to say the least.

  2. miRNAblog says:

    “To date, more than 500 microRNAs have been identified in the human genome.”

    This number is rather outdated. Since 2007 there are more than 500 human miRNAs known. Currently, there >2000 human microRNAs validated and listed in the public data base miRBase.org .