Karen Aiach, 43, is the founder and CEO of Lysogene, a biotech company developing gene therapy treatments for rare central nervous system diseases.
Remarkably, the French native had no background in science or the pharmaceutical industry, let alone the advanced fields of gene therapy, before starting the business. Aiach was an audit specialist for Arthur Andersen who went on to found her own boutique consultancy in 2001. Then, in 2005 her baby daughter, Ornella, was diagnosed with Sanfilippo Syndrome A, a rare neurodegenerative disease that has no cure and can reduce life expectancy to less than 20 years.
That prompted Aiach to leave her career behind and dive into the world of biotech, connecting with scientists, funding research, and eventually starting her own company in 2009. This past May, Lysogene raised 16.5 million euros in its Series A, and is currently raising another larger round from North American investors (the company is based in a suburb of Paris). Aiach spoke recently about her experience as an entrepreneur and mother in a conversation that has been condensed and edited.
Brian Solomon: Take me back 10 years to when your daughter was diagnosed.
Karen Aiach: My husband and I were called to the hospital in Paris after a few months of anxiety. Our pediatrician had noticed our daughter, Ornella, had a few special features, including wide eyebrows and liver enlargement. These were very specific signs of alert for this kind of disease. She went through clinical examinations and blood testing, genotyping. After five, six weeks of this, we were told she was affected with a rare condition called Sanfilippo Syndrome A.
Solomon: What was the prognosis?
Aiach: We were told our six-month-old child would be mentally impaired, then physically handicapped, then die in her second decade. We went rapidly on the web and looked for information. We downloaded scientific articles related to the disease. Our first surprise was to realize that we were able to understand the contents of scientific publications and navigate into these papers and find the authors. We contacted them and started educating ourselves on science behind this syndrome and the symptoms.
Solomon: Did you eventually need help?
Aiach: I hired a neurobiologist who had been involved for years in gene therapy for neurodegenerative disorders. She was knowledgeable in how the science worked and the labs worked, had connections with biotech companies. Then in February 2006 we visited biotech companies like
Solomon: Where did you begin?
Aiach: We began funding a research program out of a nonprofit I founded, raising funding from patient groups and putting our own savings into the program. We had to think about building vectors – the technology that transports the gene.
Solomon: When did it become a for-profit company?
Aiach: We were doing efficacy studies on mice, and a team had published preliminary data in Italy at the same time with very compelling data using the same vectors. It looked like more than a philanthropy program—I had a drug development program in my hands. So I started Lysogene in 2009.
Solomon: Was it more difficult to run a for-profit than a not-for-profit?
Aiach: No, it was the opposite. I’m a corporate person – and even though the company was tiny, it was easier. I’m more at ease in that type of environment than the not-for-profit world where the governance and rules are different.
Solomon: You didn’t have the scientific background, though. Did people in the industry take you seriously?
Aiach: The biggest challenge for me at the beginning was that I was not a scientist or a clinician or gene therapist. I was a mother taking responsibility to build a program and manage it. No one else would have done it so I wasn’t infringing on anyone else’s territory, but even so my being a mother of a patient was strange for some people.
There was skepticism and lack of support because they believed I wouldn’t make it. The investigator we worked with on our Phase 1 study was always quite difficult with me because he only considered me the bank. He couldn’t see why on Earth I would care about the clinical design. It was hard for him to listen to my voice. There were also patient groups who wouldn’t support this program because it was managed by non-scientist. They considered me arrogant. Some said, “If that idea was so good,
Solomon: How did you respond?
Aiach: Just because something has not been done doesn’t mean it is a bad idea. But I was afraid too. Not being originally from the biotech field, there were rules and practices I didn’t know about. I might not have been armed enough to face the challenges I was ignorant of.
Solomon: Were there times when you weren’t sure you would make it?
Aiach: In 2013 we were approaching a time when we didn’t have any more money. We had our first patients dosed -- including my daughter -- but we couldn’t leave them like that. We needed money to follow up. It was an emergency situation. So we went to friends, family members, showed that we had already done so much with clinical data and regulatory feedback. In a period of three weeks we raised 400,000 euros.
Solomon: You’ve run your own company before, but this must have been different.
Aiach: Since my daughter was involved, it was a question of life or death for me. I cannot pretend today that she will live longer than other patients with Sanfilippo Syndrome A, but she is living better than I could have expected. We didn’t know really what would come out of the trial treatment for her or the three other patients. But without doing anything, the consequences of the disease were dire—Ornella lost speech skills around age of 3 and become extremely hyperactive – she would run day and night everywhere in the house and break everything, putting herself and everyone in danger. We needed someone to take care of her 24 hours a day. That was our everyday life, without doing anything that’s what we’d have to face for years.
That’s a very strong motivation. I had to make this trial happen. We translated from animal to human trials within 5 years which is not unprecedented but is outstanding. Our teams could see me working night and day to have the program in the right direction. My daughter has been treated and benefited, and we’ll see how she progresses or regresses. But the pioneering spirit to transform this investigation treatment into something accessible for other patients has become a real passion for me. That’s something special and different from another company where you only want to make profit.
Solomon: Your daughter is now 10 years old. How is she doing?
Aiach: She is doing better. She remains a child with Sanfilippo Syndrome A, and was only dosed with treatment at the age of 6, which is pretty advanced in the disease. Since then she’s been much better for last three years from a behavioral standpoint -- not hyperactive, sleeps well. That’s changed her life, and the lives of her family around her. Now I have a smiling kid at home, which is the best outcome I could have dreamed of.
