Meet the mom curing her daughter's incurable disease

When Karen Aiach’s infant daughter Ornella was diagnosed with a fatal genetic disorder, she upended her own life to find a cure. Five years after doctors told her Ornella likely wouldn’t live past adolescence, Karen won approval to put the treatment she developed into clinical trials. Her first subject? Ornella.

This is practically unheard of. Karen isn’t a doctor, and had never worked in medicine. The disease Ornella has–called Sanfillipo syndrome–is so rare that almost no one was trying to cure it. But by raising the money and coordinating researchers from across the globe, she was able to develop a revolutionary treatment. And she did it using gene therapy.

“Some people would say, ‘What is she going to do? If no one has done it before, it’s because it cannot be done.” – Karen Aiach

Right now, there are only three gene therapies on the market in the entire world. First developed in 1988, it’s one of the newer and most cutting edge therapy techniques we have. It involves using a virus to replace faulty genetic code with good genetic code.

Lysogene, the company Karen started, has changed Ornella’s and Karen’s lives. And if it passes clinical trial stage, it could change the lives of Sanfilippo sufferers across the globe–and pave the way for a bold new era of medicine.

Related
Pacemaker powered by light eliminates need for batteries and lets the heart to function more naturally
Scientists designed a pacemaker that transforms light into bioelectricity, or heart cell-generated electrical signals.
Generative AI tech is dreaming up new antibodies
A new tool for designing antibodies relies on the same kind of tech underpinning DALL-E and other image-generating AIs.
Pill to prevent Lyme disease kills ticks before they can infect you
A pill to prevent Lyme disease quickly killed ticks that bit treated volunteers, suggesting it could slow the spread of tick-borne diseases.
Drugs made in space “cooked real good,” says startup
Varda Space Industries has shared the results of its first mission to manufacture “space drugs” in Earth’s orbit.
Soaring insulin costs? Cows could help.
A genetically engineered cow that produce milk containing with human insulin could help cut the cost of the life-saving diabetes med.
Up Next
Subscribe to Freethink for more great stories