Next Gen drugs are designed to meet the challenges of the most complex and intractable diseases, such as cancer and aging. To do this, next gen drugs will be administered systemically, navigating the body for targeted cells. While the concept isn't new (heck, viruses do it) the ability to manufacture this new class of drugs certainly is.
Next gen drugs are virus-like particles built from multiple molecular parts. Together, those discrete components confer structural integrity, immune system stealth, and cellular targeting. Some drug particles may be directed to genetic modification; others on delivering chemotherapy. In every instance, a next-gen drugs are engineered devices designed to maximize therapeutic effect.
Despite being directed at quite different diseases, next-gen drugs, as a class, share much in common. Most notably, there are common aspects in structural integrity, stealth, targeting, and nuclear location. But while next gen drugs will share most of their building blocks across a spectrum of medical conditions, specialist labs will be required to fine tune all elements of the design, and in particular, the specific mode of action. Large, inward-looking research programs will be replaced with a more distributive model. Drug design is a team sport, led by smaller, independent labs all over the world.
Most next gen drugs will be aimed at genetic modification and transcription interference. Several such platform technologies, including CRISPR, PNAs, zinc-finger, mRNA, and RNAi, are entering the clinic. These platforms are suitable for different types of disease conditions; all of them share the need for a delivery system.
How many particles are in a dose? The answer depends on the nature of the disease and its cure as much as particle targeting efficiency. For example, a disease like sickle cell might require only 10% transfection to achieve a functional cure; cancer, on the other hand, likely needs virtually all cancer cells neutralized. In short, a dose might range from millions to billions of particles.
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