Chemical medicine. Small molecules in medicines that can be produced in a lab.
The vast majority of medicines
Protein medicines. Enzymes
Cut out the middle-man – we don’t need a chemical that alters the shape and function of proteins. Just give the protein as a drug itself.
Enzyme treatments, antibody therapies, Humira (for arthritis – the most successful drug of all time)
Living medicines.
Use viruses, bacteria, cells, etc. to impart changes in your body
Gene and cell therapies
Why? Other types of medicine go through your entire body and we don’t always want that
Chemo for example
What if you could target cells specifically?
With this we can engineer in logic circuits (e.g. if statements, loops, etc.) to get really precise delivery of drugs
Really common misconception – all viruses are bad
Very few viruses make you sick
But viruses are really good at getting inside of you
These viruses can be used to deliver different kinds of medicines
Where are we now?
A bunch of companies are working in gene therapy for various diseases and on their way to FDA approval
Typically, the therapies so far have only been rare, single-gene disorders
Can we use this on broader problem spaces
Could you use a virus to cure cancer?
Conventional gene therapy takes 10-15 years, costs a few billion, and treats rare diseases
Immunotherapy - retraining your immune system to fight cancer
You get large tumors because an individual tumor cell may randomly generate a mutation that will give it essentially an invisibility cloak so your immune system can’t detect it
Could you make a universal gene therapy?
Nicole set off to try this
Essentially they try to deliver a type of cancer announcing payload that can alert the immune system that there is cancer so it can fight it off
This therapy was able to cure brain cancer in mice
Types of gene therapy
Gene transfer – copying a section of DNA into a place that is missing it
Gene therapy – editing an existing mutation in place
Every virus on the planet has a size that they can carry
AAV viruses (what Nicole works on) can only package about 4.75 KB of dna
One KB is a kilobase which means 1000 base pairs of DNA
About 80% of gene transfer therapies fit within 4.75 KB
Gene editing isn’t size constrained because you don’t need to send the whole DNA
These viruses can carry the arbitrary strands of DNA that you want to put into the genes
The tech is there today to go in and change one A to a C for an example
