The pharmaceutical industry relies heavily on small-molecule drugs to treat a variety of diseases and medical conditions [1]. Their ability to cross biological barriers, modulate multiple biological targets, and be chemically synthesized makes small-molecule drugs highly desirable for medical use [1].

Small-molecule drugs are organic substances that interact with key proteins to influence biological pathways [1]. These compounds have a low molecular weight, making it easier to penetrate cells.

Boulder Colorado pharmaceutical companies, such as Crestone, Inc., and companies across the world, use structural biology to gain a greater insight into the activity of small-molecule drugs. Through this approach, researchers can analyze how molecules derived from plants, roots, herbs, vines, and fungi interact with orthosteric and allosteric inhibitors. Such studies can reveal the structural basis of the drugs’ action, providing a better understanding of their chemical synthesis.

Through biophysical and biochemical methods, molecular interactions between small molecules and their targets can be identified, providing insight into the function of a target, and helping to pinpoint druggable sites for purposeful drug design [2].

Evolution of Small Molecule Drugs

For centuries, humans have been utilizing small-molecule drugs to address various ailments. Initially, natural sources such as plants, roots, herbs, vines, and fungi were used for medical treatments. However, with the industrialization of the 19th century, the production of organic chemicals increased and enabled the invention of the first artificial drug, chloral hydrate, in 1869 [3]. Aniline and p-nitrophenol, by-products of coal-tar production, were the first to be used as analgesics and antipyretics.

In 1899, Bayer pioneered a major milestone in small-molecule drug development with the invention of acetylsalicylic acid, more commonly known as Aspirin [4]. Since then, small molecules have become the primary focus of the pharmaceutical industry due to their advantageous properties. These molecules have the capacity to traverse biological boundaries, modulate a variety of biological targets, and their structure can be quickly modified, facilitating systematic progress in their effectiveness.

Impact on Healthcare Innovation

Small-molecule drugs have had a tremendous impact on healthcare innovation. They have enabled the development of more effective and safer treatments for various diseases and conditions. They are easier to manufacture, transport, and store than other medications, making them more cost-effective [5].

Small-molecule drugs offer greater precision and control when targeting specific parts of the body and avoiding harmful side effects. Treating rare diseases has fully utilized the use of small-molecule drugs. They can target specific molecular pathways involved in the condition, leading to more effective treatments and improved quality of life for patients [5].

Small molecule drugs ensure the development of personalized medicines is possible. Personalized medicine uses small-molecule drugs to target individual genetic features to provide tailored treatments for patients. By considering an individual’s genetic makeup, doctors can modify treatments to be more effective for specific people. This can help to improve outcomes and reduce side effects associated with traditional treatments. For example, a patient with a specific gene mutation may benefit from a particular drug, while another patient with a different gene mutation may not.


Small-molecule drugs continue to be the primary source of treatments for many diseases and medical conditions. Their ability to cross biological barriers, modulate multiple biological targets, and be chemically synthesized makes them highly desirable for medical use.

At Crestone, Inc., one of the leading pharmaceutical companies in Boulder, our team is continually researching small-molecule drugs in the pursuit of treating a variety of medical issues, such as C. difficile, NTM, and other ailments.


  1. Next Generation Therapeutics – Astrazeneca.
  2. Qingxin, Li, and Kang Congbao. “Mechanisms of Action for Small Molecules Revealed by Structural Biology in Drug Discovery.” International Journal of Molecular Sciences, U.S. National Library of Medicine,
  3. Alan, Jones Wayne. “Early Drug Discovery and the Rise of Pharmaceutical Chemistry.” Drug Testing and Analysis, U.S. National Library of Medicine,
  4. Bayer Patents Aspirin – History. A&E Television Networks,
  5. “What Are Small-Molecule Drugs?” Smolecule,