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Lysogene’s Founder & CEO: A Conversation With Karen Aiach

October 17, 2014

The Orphan Druganaut Blog is honored to have the opportunity to speak to Karen Aiach, founding president and CEO of Lysogene, a clinical stage biotechnology company specializing in intracerebral gene delivery for the treatment of neurological diseases. Lysogene’s first product is SAF-301, a gene therapy product that is for the treatment of a fatal Lysosomal Storage Disease (LSD), Mucopolysaccharidosis Type 3 or Sanfilippo Syndrome.

1)  Please share with the readers how Lysogene was formed and how you became involved?

KA.  My daughter, Ornella, was diagnosed with MPSIIIA in 2005, a few months after birth. MPSIIIA is a lethal rare lysosomal CNS disorder with an endless number of high unmet medical needs. It has a huge negative impact on patients and their families and there is no treatment. The development of treatment is complex, because biologically, the brain poses specific problems related to the difficulty of delivering therapeutic agents across the blood brain barrier. It progressively appeared that intracerebral gene therapy was likely to be the most realistic and robust option for these patients. This is when I made the decision to found the company Lysogene, with Olivier Danos as its scientific advisor. The aim was to bridge the gap between a huge unfairness on the one hand, and immense talents on the other, in order to bring a safe and effective treatment to children in desperate need. I leveraged on my original professional background (steering and piloting highly complex international projects at Arthur Andersen) to build the program, and raise funds. Attracted by the compelling mission, outstanding scientists, clinicians, regulatory experts, patient advocate groups and others rallied to the cause. We translated from bench to bedside in less than 5 years. Our phase I/II clinical trial, the first gene therapy trial in MPSIIIA, started in August 2011.

 2)  What is the mission of Lysogene?

KA.  Lysogene’s mission is to improve the normal course of incurable, life threatening CNS diseases. We do this by developing breakthrough, safe and effective, gene therapy. Lysogene has the support of leading life-science VCs and recently raised € 16.5 million. Our aim is to bring to the market the first gene therapy for Sanfilippo A. We are also expanding our pipeline with a second product for another rare CNS indication. Lysogene has a fascinating scientific case and our collaborators work relentlessly to develop new and often emerging skills, necessary to manage this extraordinarily challenging and cutting edge science.

  3)  Your first product in the Pipeline is gene therapy product SAF-301 for Sanfilippo Syndrome. What stage of development is SAF-301 in? What is “intracerebral gene delivery” and the importance of it?

KA.   In 2013, we successfully completed a phase I/II study with SAF-301. SAF-301 is an intracerebrally administered AAVrh10 encoding sulfamidase, the missing enzyme in Sanfilippo A. The aim now is to evaluate SAF-301 in a phase II/III pivotal clinical trial with the ultimate objective of applying to the regulatory authorities for a marketing authorisation in the EU and US, to start.

Intracerebral gene therapy enables delivery of the functional version of the defective gene directly into a young patient’s brain. The gene therapy is delivered in a single dose to the central nervous system providing constant levels of the therapeutic protein. Unlike other approaches focusing on managing symptoms or preventing complications, Lysogene’s technology addresses the root cause and, therefore, ultimately aims at curing the disease. Importantly, Lysogene’s single-dose approach is expected to represent a considerable benefit in the quality of life for patients and their families.

 4)  Are you working with patient advocacy organizations on the development of SAF-301?

KA.  Early interactions and collaborations with PAG have been central in our strategy. This is why, Samantha Parker’s mission as Chief Patient Access / Health Policies Officer, is a strategic position within our company. Samantha has 15 years’ experience working in rare disease research and public health collaborative networks. She has paid specific attention to the role of patient organizations and the provision of information and access to therapeutics. Working closely with PAGs and leveraging on our mutual expertise at every stage of the development has been an invaluable asset –their experience and advice has been essential in accurately capturing the natural history of MPSIIIA and the quality of life dimensions, to the protocol design, informed consent process and long-term follow up of the study –. We are proud of our outreach to patient groups and communities and aim at intensifying productive and pioneering interactions, including online communities and social networks, all across our portfolio of activities. Lysogene has just submitted a project to the European Commission to build an MPSIIIA consortium with substantial patient representation to ensure probity, openness and good science.

 5)  What do you see as the relationship between patient advocacy organizations, biotechnology companies, and international regulatory agencies for the future?         

 KA.  These relationships are key to the successful drug development process. It’s a fact. As this notion progresses, these relationships should intensify and multi-stakeholder approaches become the standard. I believe that it is for the best of patients and their families who have to be the ultimate beneficiaries of this battle.

 Thank you for your time and sharing with the Orphan Druganaut readers Lysogene’s  mission and the status of SAF-301.

Lysogene Logo courtesy of Lysogene.

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