Genome engineering

Week 07: Imagine that you are a scientist told by your advisor or boss to come up with a plan to make a difference. Possible ways to go about this include therapeutic drugs (small molecules like nucleoside analogs or biologicals like enzymes, antibodies, or nucleic acids), new vaccines, or new or better diagnostics.

Describe your new product. Do this using 6 sentences or less. I used to work in the pharmaceutical industry on developing new therapeutics to treat infectious diseases. One thing that was drilled into us was that it takes 3000 days to get a new drug to market once it has been discovered. That’s about 8 years. During that time things like toxicology studies using animals, pharmacokinetics, and plans for large scale synthesis of the drugs are the first things determined. Then there are Phase !, II, and III clinical trials to do. Under ordinary circumstances, every day is precious because a new drug or vaccine patent life diminishes while all this research is going on. Now haste is even more important because we are in a pandemic, and people are dying. If you roam around the internet, you will see that there are ways to accelerate some of these processes. President Trump talks about having a vaccine in a few months. That seems farfetched, but as I mentioned in the lecture, Moderna is already testing a mRNA-based vaccine against the corona virus. Many companies are developing new diagnostic approaches. Provide a bullet point outline of the steps needed to get your product approved by the US FDA and into clinical use. Be brief, my goal here is that you gain some understanding of the regulatory processes for medical solutions to a problem like the current pandemic. How would your approval process be different if you were developing a product for a new rabies vaccine (i.e. something that was not urgent)?