How do you design biotechnology products to treat specific diseases?

Often, the disease burden is not well understood in a specific population and is not documented in the literature, creating the biggest challenge when recommending and working towards a possible product. For example, the burden of Celiac disease or Gluten intolerance isn't well understood and reported in Pakistan, which diminishes the reported rate of the disease. Consequently, the industry may not identify the potential production rate and may refrain from investing a large amount. This is the primary challenge, explaining why most products don't reach to the production stage.

Early quantification of the potential economic impact of a problem or need is a key step in evaluating and prioritizing needs to address. Questions to be asked and answered include: What exactly is the problem? What are the parameters of the problem? (who, what, when, how) What are the potential benefits of fixing the problem? Who would receive the direct benefit? In what setting(s)? In what scenario(s)? Who cares? What economic incentives are associated with fixing the problem? Are there reverse incentives? Who can benefit most?

While this article makes the story flow by first identifying a problem, the reality is that often times biotech does not follow a specific path: a solution/product might come before the problem! An example is Moderna, which had a mRNA platform product, was quickly adopted to make COVID-19 vaccines. I bet that before 2019/2020, no one in the world knew that making a vaccine quickly was a problem they were going to have!

Begin by identifying the specific need or problem your product addresses. This could be a gap in current medical treatments, a need for more efficient agricultural practices, or an environmental issue. Clearly conceptualize what the product is. If it's a therapeutic drug, what disease does it target? If it's an agricultural biotech product, what crop does it benefit and how? For medical devices or diagnostic tools, what is the intended use? Functional Specifications: Define the specific functions of the product. Highlight the benefits of your product. This includes its advantages over existing solutions, its efficiency, cost-effectiveness, and any additional benefits like environmental friendliness for bio-agricultural products.

Designing biotech products for targeted disease treatment involves a meticulous understanding of disease biology and patient needs. Employing advanced genomic and proteomic tools for personalized medicine allows tailoring therapies to individual variations. Integration of cutting-edge technologies like CRISPR for precise genetic modifications and continuous refinement based on real-world evidence can further optimize product design, ensuring adaptability and efficacy in the ever-evolving landscape of disease biology.

Improving the API (active pharmaceutical ingredient) in FDA approved therapeutics is not an option. However, there are other levers that can be varied. Some examples are: - Dosage - Patient selection - Companion diagnostics - Indication expansion Though getting more granular on the variables above won't improve the API, it will improve its overall efficacy by better tailoring to the patient.