Future cures are in our genes

Imagine a world where rare or inherited genetic disorders could be cured or prevented by precisely targeting the root causes. Rather than just treating blood disorders, retinal diseases, neuromuscular and other diseases, we would eliminate them.

This is the present – and promise – of gene therapy. But gene-based therapy opens even more possibilities, and almost unlimited potential, beyond rare genetic disorders.

In many ways, the limitations are only what scientists can think of, and that’s really where the value of collaboration across Health N Medic and with our current and future partners comes in.”

“Imagine a world where no life is limited by genetic disease and the ambition of gene therapy to treat or possibly prevent disease is realized for the millions of people living with an inherited condition. “By addressing some diseases at a genetic level, entire lives, families and our global healthcare system could be transformed by unlocking the full potential of gene therapy.”

The next generation of pioneering medicines, with gene therapy front and centre, is blazing a path towards truly personalised global healthcare. It’s a proactive approach, strengthened by partnering and collaboration, that seeks better healthcare at a lower cost to society.

First, a bit about genes

To understand gene therapy, we should start with genes. Cells are the basic units of life, and each cell contains a full set of instructions, or genes. Each gene encodes for a different protein, or the “business ends” of the cell and the regulatory instructions of when to use this gene. Cells in our body are different, and each cell uses only some of the genes based on its needs – immune cells will need genes for proteins like antibodies to fight off infections, brain cells will need the genes that encode neurotransmitters, and so on.

But sometimes our genes have a glitch in the code, either one that we inherit from our parents at birth, or one that arises within our own bodies over time, and that can cause disease, because the cells cannot work properly. In some inherited cases, the error is major, and will cause a severe disease like haemophilia or Huntington’s disease. In other cases, the differences we inherit are part of the natural variation between humans, but can increase our risk of common diseases, like heart disease or type 2 diabetes. But genes can also mutate during our lifetime, especially due to errors accrued over the lifespan, resulting in illnesses, especially cancer.

In the future, instead of a series of traditional medical treatments that only manage some diseases – or a lifetime of injections, infusions, monitoring, adjustments and ongoing doctor appointments – patients might instead receive gene therapy treatment to fix that glitch.

A tough trail ahead

While the prospects of gene therapy are awe-inspiring, it won’t be easy or instantaneous. Before we ride off into a sunset with great therapies for genetic disease, there are challenges to overcome. Some aspects of the science behind gene therapy are uncharted; there will be stumbles, disappointments and setbacks.

Even when solutions are found in a laboratory, in the form of novel vectors or “messengers” that carry new genetic instructions to block, take down or take over for malfunctioning ones, those breakthroughs must then be meticulously translated to humans and into large-scale production processes that can repeatedly and reliably produce the same medicines in manufacturing facilities.

Making our vision a reality

“We believe that within 20 or 30 years from now, gene therapy will represent the most important class of pioneering medicines in our industry, “The reason for that is clear: if you can go in and do genetic surgery – by removing malfunctioning or abnormally regulated genes and replacing them with genes that are normally regulated – then you can get to the basis of what causes disease and fix it. It’s version 1.0 right now for gene therapy, but we’re very excited about the future, and our partnership with trailblazing companies.”

Already today, there is initial evidence that gene therapy could change the lives of patients with certain diseases by introducing a working gene to step in and do the job of a malfunctioning one. But in versions 2.0 or 3.0, gene therapy would involve taking out one gene and replacing it with another, like surgery at a genetic level, or by adding a new gene to produce a protein that is therapeutic, like a vectorised antibody medicine against a target that causes disease.

Ultimately, the future of medicine will be a combination of complementary approaches, including small molecules, large molecules, and programmable therapies that include gene therapy, cell therapy, bacterial therapies and RNA-based therapies.