Take a moment to reflect upon our progress with genetic medicine
Hello friends,
The era of genetic medicine is upon us.
We now have our first wave of in vivo, gene therapies available for patients. Five of these drugs are commercially available in US and Europe. Many more drugs will see approval in the next decade, soon these medicines will distribute globally.
The road has been long…it tracks back to Charles Darwin and Gregor Mendel in the 1860s. Species evolve as they try to adapt to new environments…and they do so via inheritance of traits…and these traits pass forward into future generations in a predictable, discreet and quantitative manner.
Fast forward to 2003, when we decoded the human genome and first learned that a human body can be built with just 20,000 genes.
Healthy people are out in the world, walking and talking and enjoying a body built from 20,000 good genes.
In contrast, when a child inherits a gene with an improper DNA sequence, the symptoms of a genetic disorder may begin to arise. Therein lies the medical necessity for genetic medicine.
The image above illustrates the large volume of gene therapies that are moving into late stage clinical development. The bullseye in the center contains the commercially approved drugs. Drugs on the periphery are still in the pre-clinical stage.
AAV has become a widely used technology platform for gene therapies. We rely on this modified virus to deliver a functional DNA sequence into the target cells of the patient with a genetic disorder.
Scientists have been chipping away at AAV for 50 years. Nothing happens overnight.
AAV is a relatively safe vesicle for the delivery of genes into human cells. The size of this vesicle is surprisingly small; AAV is only 25% the size of a coronavirus.
AAV is about 20 nanometers in diameter.
The AAV particles in the image above are in blue. As you would imagine, 20 nanometers is pretty small, so the image gets a bit grainy. Adjacent to the AAV lies a larger virus in red, an adenovirus.
By manipulating the genes and life-cycles of these two types of virus, the biotech community has learned to design and manufacture a clinical grade, AAV virus, which can deliver genetic medicine.
If this topic is of interest to you, please join me for a free, hour long discussion of AAV gene therapies this Friday.
The zoom class is titled ‘Focus on AAV gene therapy’ on 4/7/23 at 12pm eastern time (ET).
Follow this link to register and sign up for the class.
Or hit the button below….
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All the best,
Kevin Curran PhD