Thinking dynamically across biological levels

Biotech is moving very fast… and somebody has to try to make sense of it all.

Ideally this person is an extrovert who enjoys explaining complex biology to a broad audience….

Wait that’s me!

Okay, I accept the offer.

I’ll be using this Substack channel to communicate new developments in the biomedical space and share insights on emerging trends.

There’s a lot happening in this sector….

  • AI looks very exciting for drug development

  • Ex vivo gene therapy (CAR-Ts) are hitting stride

  • In vivo gene therapy (AAVs) carries the potential to hit large patient populations

  • Genome editing (CRISPR/Cas-9) is halfway through clinical trials and next-gen Genome editing tools appear more precise

  • RNA drugs coupled with Lipid Nanoparticles are now a proven drug platform

For my first article on Substack, I’m posting a series of images I use when speaking to a broad, biotech audience.

I encourage everyone to think dynamically across the Biological Levels of human physiology.

Ideally, your mind can move in a stepwise manner from gene to RNA to protein to cellular behavior to tissue…. and ultimately to the clinical condition a physician will observe when a patient walks into their office.

This fluid movement of thought provides a functional understanding of:

  • a healthy human body

  • a disease state body

  • the function of genetic medicine

Mentally shifting up and down these biological levels allows you to think clearly about disease pathology and also helps you understand the mechanics of new genetic medicines.

Most people are unclear about the nature of genetic medicine and how it can resolve disease at the root, genetic level.

Below, I refer to a new medicine, called Zolgensma, to explain how a gene therapy can treat a rare genetic disorder, Spinal Muscular Atrophy.

Zolgensma is an AAV9 based gene therapy that delivers the missing gene, SMN, into the target cells, motor neurons.

Advances happening in the genetic medicine realm will affect society on multiple levels. One point of concern…. is the expensive cost of new, branded genetic medicine. The list price for new gene therapies can range from 300K to 3M for a single injection.

We need to focus on broad access to these medicines and make sure they distribute globally beyond just the wealthiest nations.

I’ll be touching on this issue in upcoming posts.

It starts with a gene, but it ends with a shift in societal dynamics.

Hopefully, these images are helpful when contemplating the current state of gene therapies.

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I’ll be posting regularly here.

Kevin Curran, PhD