An inspiring story of a mother that took matters in her own hands

Karen Aiach

Karen Aiach is a mother of a child with Sanfilippo disease.  Karen and other parents discovered that there was a way to treat the disease with intracerebral gene therapy. They had been working as a parent association until then but things were not moving fast enough.  So Karen and the other parents founded a clinical stage pioneering biotechnology company – Lysogene.

Intercelebral gene therapy was first tried on animals and then very recently on humans.

The publication of the trial is not yet released but the initial results were promising.

This is yet another story of parents taking initiative but in this case they are going all the way to cure a rare disease by themselves.

The Lysogene team

The SANFILIPPO syndrome is a CNS illness or storage disorder that is extremely severe and debilitating, fatal in childhood and that currently has no known treatment. The clinical stage trials of an intracerebral gene therapy for a pediatric condition represent a world first for a private company. This comes only three years after validation of the proof of principle in animal trials.

Video of Karen giving a presentation