What Are GTTs? These Technologies Can Impact How a Gene Functions

Genetically targeted technologies (GTTs) are scientifically advanced technologies that can impact how a gene functions.¹ One category of GTTs, known as small interfering RNA (siRNA), can act like a light switch to “turn off” genes that lead to certain diseases.^2,3

The technology is complex and still emerging. However, with further research and development, we could potentially target the root causes of many diseases that are not responsive to treatment or are cumbersome to manage, including cancer, Alzheimer’s, cardiovascular disease, and rare diseases.^4,5 

1. siRNA therapies are complex, sometimes requiring more than a decade to develop

In 2006, the Nobel Prize in Physiology or Medicine was awarded to researchers for their "discovery of RNA interference - gene silencing by double-stranded RNA," a natural mechanism that controls the flow of genetic information, and that led to new opportunities in regulating gene expression.^6,7  

siRNAs utilize short sequences of genetic material to address an underlying cause of the disease rather than just treating the symptoms.² This technology requires a highly controlled, multi-step process, which means it can take years to develop a therapeutic for specific disease states. siRNAs may allow a level of precision that can be tailored for individual patients or patient groups, offering targeted treatments.³

2. Some siRNA therapies can address genetic issues by influencing gene messaging

Although both gene therapies and siRNAs work at the genetic level, their approaches are different. Most siRNAs are expected to be approved by the FDA as small molecules and these therapies may turn off genes to prevent production of unwanted proteins, including those that cause chronic diseases.^2,8 For example, siRNA, works to selectively silence unfavorable genes through RNA interference.^2,3  

FDA-approved gene therapies are typically biologics and can change how genes express themselves (via gene replacement, addition, inhibition or editing) by delivering DNA into the body.^8,9,10

3. siRNA therapies may help revolutionize the patient experience

It is estimated that about half of people prescribed medications for chronic diseases do not take their medication as directed.¹¹ siRNAs can be long-acting, which may enable patients to go from daily pills to less frequent injections, some only requiring administration several times a year.³ This may lead to higher adherence rates through simplified care routines, reduced daily impact, and improved overall quality of life. 

4. siRNA therapies may help transform the treatment of a broad range of serious illnesses 

Although only a limited number of siRNA therapies have been FDA-approved to date, including some that target the liver, the research is ongoing.¹² Researchers are testing novel ways to target different organs, such as the brain and lungs, which could bring the benefits of gene silencing therapies to people with a much broader range of serious illnesses, including neurological diseases.^5,12 Other treatments have the potential to transform the standard of care for more common illnesses, including cancer, Alzheimer’s, and cardiovascular diseases.^4,5 And there are even more still in development. 

It's important to understand how these powerful genetically-targeted technologies work, and their complexities relative to traditional small molecules they are grouped with, in order to protect further development and research.