Could personalized therapies be developed in a few days?
A copy of our genome is present in every cell of our body, with more than 20 thousand genes and billions of DNA nucleotide pairs. DNA is two chains of nucleotides paired together by a simple rule: Adenine pairs with Thymidine, and Cytosine pairs with Guanine.
Our genes shape what we are as a species, and as individuals, they profoundly influence our health and well-being. Most of our genes will not have mutations, but some will and these mutations may increase our risk or susceptibility to disease or, in some cases, make us ill. Today it is possible to obtain a sequence of an entire individual genome and discover any genomic mutations that are present which may make us prone to disease.
But this is only part of the journey toward a cure. Biotechnology must advance side by side with bioproduction to enable personalized medicines or drugs to be available in days. In vivo gene editing, the ability to edit genes in live cells, is a step away via CRISPR/CAS technology. We already have gene therapies on the market with examples mediated by viral and non-viral vectors to transport and transfer genetic information, to silence, replace, or bring in function to defective genes. For the time being, these solutions are being developed for the community in general and focused on market needs.
By 2030 this will have changed. A sample of your breath, cells from your mouth, or a blood sample will be sequenced in hours. In a couple of days, an overview of your genetic background, weakness, and strengths will be built based on your genetic sequencing. You can then evaluate your risk of various pathologies and order personalized therapies to reduce the likelihood of a specific pathology based on your genetic fingerprint.
For this to happen, the bioproduction of personalized medicines must develop to a stage where standardized manufacturing approaches can be applied, and personalized bio-drugs can be produced with short lead times and cost-effectiveness. AI and data analysis are being implemented today and are boosting the knowledge and control of biology, biotechnology, and bioproduction.
Alongside scientific biotechnology and bioproduction challenges, work with health authorities and regulators will need to advance to streamline the regulatory requirements for personalized medicine. Will the path for personalized medicine approval be equivalent to the existing path to global medicines? Will the individual be able to assume the risk around their personalized medicine, and therefore will health authorities simplify the authorization process or not even need to authorize it?
These are questions with no current answer, but it is extremely important to address them to allow the biotechnological boom in personalized medicine to deliver on its potential.