Scientific revolutions tend to happen gradually—until, suddenly, they don’t. According to Rahul Bhushan (pictured), Managing Director at ARK Invest Europe, genomics is currently facing a similar trajectory. In his fascinating insight below, Rahul explains why advisers and investors should be taking note of developments and how and why these are accelerating the investment opportunities in the sector.
Since DNA was first isolated in 1869, to the first draft of the human genome sequence being released in 2001, genomics has come a long way. Fast forward to the present day and we’re now at a major inflection point that investors should be aware of.
The study of Genomics – the analysis of DNA and how genes interact with each other and the environment – has become a game-changer in the medical world. From predicting susceptibility to disease and developing better drug treatments, to treating cancer and identifying genetic disorders.
In February alone, three major breakthroughs in genomics, gene therapy and AI-driven biology signalled that we’re entering a phase where innovation is no longer incremental.
1. Restoring Sight: The Power of Precision Gene Therapy
A functionally blind man regains sight with Luxturna gene therapy.
A 31-year-old man in Ireland who was functionally blind is now able to see again, thanks to a revolutionary ocular gene therapy called Luxturna. The treatment works by injecting a functional copy of a faulty gene directly into the retina, allowing cells to produce the missing enzyme needed for vision. Within weeks, his eyesight returned.
This marks a major milestone in precision medicine, where a single, targeted genetic intervention can reverse what was once considered an irreversible condition. It also underscores the growing accessibility of gene therapy—what was once experimental is now becoming standard medical practice.
2. Treating Genetic Disorders Before Birth
A genetic disorder was successfully treated in the womb for the first time.
In another first, a baby girl born two years ago never developed spinal muscular atrophy (SMA) – a rare genetic disorder that usually leads to severe motor dysfunction and infant mortality. The reason? She received gene therapy while still in the womb.
Her mother took Risdiplam, a gene-modifying drug, during the final weeks of pregnancy. Today, at two and a half years old, the child shows no symptoms of a disease that otherwise would have been devastating.
This success story opens the door to a future where genetic diseases can be intercepted before birth, transforming the way we think about medicine. Rather than treating diseases after symptoms emerge, we are entering an era of preventative genomics, where early interventions can completely alter health trajectories.
3. Designing Life with AI: Evo 2 and the Next Frontier of Genomics
An AI model that understands and writes DNA.
If curing blindness and preventing genetic disorders in the womb weren’t shocking enough, last week also saw the unveiling of Evo 2, a new AI model that can read, predict and even design DNA sequences.
Developed by researchers at the Arc Institute, Evo 2 was trained on 9.3 trillion DNA base pairs and has autonomously learned the rules of life’s genetic code. It can predict whether mutations are harmful, design new functional genetic sequences and even control how genes are expressed.
In one test, Evo 2 successfully encoded Morse Code into DNA, demonstrating the ability to engineer genetic systems with precision. The implications are staggering:
- AI-assisted drug discovery could accelerate by an order of magnitude.
- Synthetic biology applications could expand into areas like biofuels, carbon capture and advanced therapeutics.
- Gene editing could become as programmable as writing software.
Evo 2 is open-source, which means it won’t just be a tool for a handful of elite researchers—it will catalyse a wave of innovation across genomics, medicine and synthetic biology.
The Big Picture: AI is Supercharging Genomics
AI is now playing an active role in hypothesis generation, drug repurposing and discovery—a trend made clear by Google’s newly announced AI co-scientist.
What does this mean for investors? It means that the next decade will see a massive transformation in healthcare, biotech and life sciences.
DNA sequencing and synthesis costs are declining ever faster. The cost of reading biology through multiomics tools like DNA sequencing has plummeted by 10 billion-fold in its 35-year history. DNA synthesis costs have dropped 100,000-fold and could continue to fall 10 million-fold by 2030.
Since 2001, the computer time required to analyse a human genome has plummeted from 180 days to 10 minutes. Today we can analyse 1.4billion genomes for the cost of analysing a single genome in 2001.
Furthermore, the value of AI-driven drug development should compound thanks to lower costs, an acceleration in time-to-market, and a longer timeline of patent-protected revenue. Over a 30-year period, the cumulative cashflow for the average AI-designed drug could reach $4 billion, compared to less than $1 billion for traditional drug models. Even before a traditional drug breaks even, an AI-developed drug could generate $2.5 billion in cashflow.
