#RARETalk: meet Karen Aiach )

We are glad to introduce Karen Aiach, our April #RARETalk guest. In 2009 she started her own company Lysogene to develop a gene therapy treatment for her daughter Ornella and all patients diagnosed with Mucopolysaccharidosis Type III (MPS IIIA.

1)  Welcome to the RE(ACT) Community Karen Aiach. You have a business background, you are an entrepreneur and the mother of a child with MPS IIIA, can you please tell our readers more about yourself?

Thank you, I am delighted to join the RE(ACT) Community. Yes, my background is very much in finance – I graduated from ESSEC business school and then worked for Arthur Andersen before starting my own consultancy firm. Shortly after Ornella was diagnosed with MPS IIIA, I decided to devote my time to her and to finding a cure for the disease. When I could not find a company interested in developing a drug, I started my own company - Lysogene in 2009 -  to develop a gene therapy treatment with the help of Olivier Danos, the former director of one of Europe’s top gene therapy labs, who is now CSO at RegenXBio.

2)  You are the founder and CEO of Lysogene. Can you please tell us more about your company goals, your approach to research project and your achievements?

Lysogene’s mission is to radically improve the health of patients suffering from incurable life threatening conditions by developing adeno-associated viral (AAV) vectors that have demonstrated their effectiveness in safely delivering genetic material to the central nervous system. We have two programs underway developing treatments for MPS IIIA and GM1-gangliosidosis (GM1). Our first Phase1/2 study for MPS III A has been completed and we are now undertaking the Phase 3 study with an improved, optimized product. 
The GMI program is also advancing and we expect to be in the clinic in the coming years.
I would add that, pediatric rare disease gene therapy presents specific challenges from a regulatory perspective and that Lysogene has already achieved orphan drug designations in Europe and the US for both programs. We strongly believe in the value of early discussions with regulatory authorities in preparing our pre-clinical and clinical trials with the overall aim of achieving marketing authorization.

3) We launched an international petition called #RAREvolution Petition asking more support for rare disease research.  How important do you think it is to advocate and improve resources for research on rare diseases? 

I think this is extremely important.  30 million  people in the US or in Europe wake up each day with a rare disease. Often ignored by Big Pharma because of the cost of developing treatments - it is important to raise awareness that,  aside from the moral obligation to provide a solution for patients suffering from these diseases, many of whom are children - treatments for rare diseases can actually have ramifications for other, more “common” diseases which affect the CNS. Rare diseases open the way for therapeutic innovation and this innovation is likely to expand to numerous other diseases.
Finally, I would stress that 50% of advanced therapy medicinal products (ATMPs) with Marketing Authorization are for a Rare Disease.

4) You participated at the RE(ACT) Congress in March 2014. How, according to your point of view, these types of conferences help rare disease stakeholders other than researchers in their work?

I think the RE(ACT) Congress has a vital role to play as an unique platform which brings together all the rare disease stakeholders in one place to review the different developments in the field from all viewpoints:  research, regulation, patient associations, and so on. 

5) The online RE(ACT) Community encourages both researchers to publish scientific info and patients to share their personal health experiences. What do you think about health knowledge sharing to push research forward? Can digital media help this process? What is the point of you of the private sector?

I think this is a very interesting development and indeed some countries are already taking steps to get patient information online. We are in an era where patients are much better informed on the different treatments available and where they are more and more willing to shop around for treatments in different countries.  
I would therefore strongly support this development which also encourages researchers to share information more readily.
From the business point of view, I would add that Lysogene has always put patients at the center of our business. Furthermore, at Lysogene we strongly believe in sharing our  experience to help further research into rare diseases and improve public health globally. We also have many advisory and speaking roles at international or national policy and scientific meetings.

6) Can you please share with us three things you have learned from your personal and professional experience that can help rare disease researchers and patients?

I have learnt not to be afraid to ask difficult questions – this was a big advantage when I first started out with a non-scientific background and I still find it very useful when exploring new territory.
I have also learnt never to take no as an answer. I was told that there was no hope of finding a cure for Ornella within the next 20 years and Lysogene is now aiming to take our treatment to market by 2020; 12 years after getting that diagnosis.
I have learnt to share that experience with others; I try as much as possible to respond to speaker invitations to share the Lysogene and also my own personal story with different audiences. I had the privilege of giving the keynote speech at last year’s Precision Medicine Conference – Rogue Therapeutics - at Harvard Medical School and next May I will have the honour of presiding ESSEC’s annual Gala.