Neel Bhut: Can AI Help Us Make Gene Therapy More Efficient?
Neel Bhut, Medical Editor at Stealth Startup, shared a post on LinkedIn:
“Can AI help us make gene therapy more efficient?
I came across this story in The Indian Express and then looked up the original paper in Nature to understand what the researchers had actually done.
One challenge in gene therapy is size.
AAV (adeno-associated virus), a commonly used delivery vehicle, can carry only a limited amount of genetic material. This can be a challenge when the gene we want to deliver is large.
What if AI could make the protein smaller?
The study introduced Raygun, an AI framework that starts with an existing protein and rewrites its sequence.
It removes parts that may not be essential and redesigns the remaining sequence to make the protein smaller while preserving its structure and function.
What did they find?
Raygun could reduce protein length by 10–25%, sometimes by more than 50%.
Why does this matter?
Gene therapy is already being used or developed for conditions such as spinal muscular atrophy, hemophilia, and Duchenne muscular dystrophy.
If therapeutic proteins can be made smaller without losing their function, they could potentially help overcome AAV packaging limits and expand what can be delivered.
This is still basic research, not a clinical treatment.
But the direction is fascinating:
Could AI make therapies that are currently too large to deliver, small enough to fit?
Nature July 29, 2026 | Raygun | Rohit Singh | Duke University”
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