What if you could edit your own genome? Fix a genetic error or decide to alter or optimize one of your traits? Would you?
Each of us is running a genomic “software-code” in each of our 40 trillion cells. The code is 3.2 billion letters long and defines your hair color, height, personality, and propensity to disease.
Since 2001, the cost reading that code, of sequencing a whole human genome, has plummeted exponentially—outpacing Moore's Law threefold.
From an initial cost of $2.7 billion (Human Genome Project), it dropped to $100 million (Craig Venter, PhD) in 2001, and today will cost you less than $500, rapidly approaching $100 per read.
We’re not far from the point where every single human will be sequenced at birth or at admission to the hospital.
And while sequencing your genome has been amazing, editing your genome is our future—a future where we can correct, and perhaps eventually optimize the software being run by the 40 trillion cells in your body.
Let's dive in…
NOTE: Want to get first-hand experience learning from the leading CRISPR/gene-editing companies in the field? Consider joining me on my upcoming Longevity Platinum Trip.
Editing Your Genome: Curing Your Disease
2023 turned out to be a breakthrough year for CRISPR, the 2020 Nobel Prize-winning work of Jennifer Doudna, PhD and Emmanuelle Charpentier, PhD.
For all of human history—for those with a genetic disease—all we could ever do was treat the symptoms, hope to make you feel better.
Now we have the potential for CURES... not treatments, not temporary relief, but cures, a chance to re-write your software and fundamentally fix the problem, the software bug.
In November 2023, we saw the first-ever approval of CRISPR-based medicine: Casgevy, a cure for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in the United Kingdom.
The Innovative Genomics Institute (IGI), which was founded by Jennifer Doudna, noted that even though this milestone has been anticipated, the speed of the approval marks a significant step.
As Doudna mentioned following the approval:
“Going from the lab to an approved CRISPR therapy in just 11 years is a truly remarkable achievement … I am especially pleased that the first CRISPR therapy helps patients with sickle cell disease, a disease that has long been neglected by the medical establishment. This is a win for medicine and for health equity.”
A few weeks later, in early December 2023, the Food and Drug Administration in the US greenlighted the therapy, and in February 2024, The European Union approved it as well.
And more CRISPR-based cures are on the way…
As IGI Director of Technology & Translation Fyodor Urnov points out:
“CRISPR is curative! Two diseases down, 5,000 to go.”
As you can see from the chart above, two other diseases are currently in phase 3 trials:
Chronic Bacterial Infection: Urinary tract infections (UTIs) are a common infection that disproportionately affects women. The treatment currently in clinical trials is a cocktail of three bacteriophages combined with CRISPR-Cas3, designed to attack the genome of the three strains of E. coli responsible for about 95% of UTIs. Locus Biosciences completed their Phase 1b trial in 2021 and began enrolling participants for a phase 2/3 trial in 2022.
Protein Folding Disease (hATTR): Hereditary transthyretin amyloidosis (hATTR) is a fatal disease caused by mutations in the TTR gene, leading to a faulty version of the TTR protein. The treatment uses CRISPR-Cas9 to reduce the amount of faulty TTR protein and is delivered in a single IV dose. The trial, sponsored by Intellia Therapeutics, has reported efficacy data on 62 participants, showing deep reduction in the toxic protein levels. Intellia has gotten FDA approval to move forward with a phase 3 study.
Notably, CRISPR-based treatments for both inflammatory disease and cancers are currently in Phase 2 trials, and those for cardiovascular disease, HIV/AIDS, Diabetes, and Lupus aren't far behind.
This year at my fall Platinum Longevity Trip, I’ll be featuring in-depth conversations with the leadership of a number of leading CRISPR/gene-editing companies that I’m super excited about. These include:
Gordian Biotechnology, co-founded by Martin Borch Jensen and Francisco LePort, PhD, is transforming genetic research with their high-throughput screening method, unraveling the secrets of age-related diseases.
Rejuvenate Bio, founded by Harvard’s Dr. George Church and led by Daniel Oliver, MBA, is harnessing CRISPR to introduce longevity gene combinations, aiming to reverse age-related decline.
NewLimit, co-founded by Blake Byers, PhD, and Coinbase CEO Brian Armstrong is boldly editing aging genes, manipulating epigenetic markers to restore cells to a more youthful state.
Rubedo Life Sciences, co-founded by Marco Quarta, PhD, is coupling CRISPR with single-cell RNA sequencing to study gene editing effects at an individual cell level, providing unprecedented insights.
Finally (although not fully confirmed yet for the Platinum Trip), this week I’m visiting Science Corp, founded by Max Hodak (who previously ran Neuralink with Elon Musk), which is using CRISPR to develop optogenetic visual prostheses—potentially restoring vision in patients with retinitis pigmentosa and dry age-related macular degeneration.
Why This Matters
The biotech revolution is here and moving fast.
Instead of fearing the continued advancement of biotechnologies, we should see them as an exceptional opportunity to unburden our medical systems and eventually IMPROVE them.
Medicine has a long history of once-frightening interventions—like heart transplants and IVF—that now help save, create, and improve billions of lives.
CRISPR and gene therapies are no different.
If you have a genetic disease, what are you doing about it? Hopefully you’re finding others with a similar genotype, self-organizing, raising funds and searching for a lab and research team who can take a shot at your CRISPR cure.
The good news is that AI will also play a role in accelerating this work.
For me, the extraordinary benefits of CRISPR edits and gene therapies, border on “biblical”... curing the blind, and permanently transforming the lives of those who previously had no hope.
While these treatments are currently expensive—and yes, they can run from hundreds of thousands of dollars, to millions of dollars—like all exponential technologies they are on a rapid demonetization and democratization curve.
And, of course, this opens up Pandora’s box. While in the past, we’ve discussed the idea of editing the genome of a “future human” at the fertilized zygote stage, now we can begin to seriously discuss how each of us might want to alter and optimize the code being run in the 40 trillion human cells we call “me.”