Meet the French Woman who started a Biotech to Save her Daughter from a Rare Disease

27/10/2015 - 7 minutes

Karen Aiach had no background in science or the biotech industry, yet when her daughter Ornella was diagnosed with the super-rare Sanfilippo syndrome this inspired her to co-found and become the CEO of Lysogene, today a clinical-stage biotech based in Paris, France.

Now Ornella is participating in the ongoing clinical trials for a new gene therapy for Sanfilippo A, also known as mucopolysaccharidosis III (MPS-III). A disease for which currently there is only palliative care available. there are 4 types of MPS-III, and their combined incidence is only 1 of every 70,000 births. A genetic mutation in a particular enzyme causes toxic build-up of heparin sulfates, which cause permanent tissue damage and paralysis.

Akin to the famous John Crowley — another Biotech entrepreneur inspired by his children’s diagnosis with a rare disease — Karen’s fight is indeed personal. Her inspiring story has led to phase I/II clinical trials for a new gene therapy for Sanfilippo syndrome A, in which her daughter is one of the patients enrolled.

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