Hear how Lysogene discussed and validated their approach into clinical trials#raredisease

orphan drug, Lysogene, rare disease, karen aiachAt the World Orphan Drug Congress 2011 in Geneva, Karen Aiach, Founding President & CEO of Lysogene made a presentation about how their ambitious intra-cerebral gene therapy product, SAF-301, entering into the clinic for the treatment of SANFILIPPO type A syndrome.

Founded only in 2009, Lysogene is proof that it is the scale of innovation and not the scale of the organisation that brings about promising research into rare diseases. As a relatively young company entering the clinical stage for the first time, the World Orphan Drug Congress was the perfect platform for Lysogene to engage with the industry at large, present on their research and harness external experience and expertise to validate their clinical studies going forward.

During her presentation, Karen Aiach outines:

  • The complex, stringent and evolving regulatory frameworks
  • Scientific and clinical challenges to clinical protocol design
  • The operational challenges to clinical development
  • Future perspective of orphan business model challenges


Hope you enjoyed that insight into orphan drug development from an industry perspective, but here is exclusive insight from the view of a patient organisation

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