Welcome

Dear Shareholders, 

We are very proud to have revamped our website, which is an important tool in our constant effort to improve our communication with you. 

I founded Lysogene in 2009 with the passion to find a cure for children suffering from MPSIIIA. 10 years later, I am impressed with what we have achieved thanks to your support. Lysogene is now a leading gene therapy platform company focused on rare neurological disorders with high unmet medical need targeting central nervous system diseases, capitalizing on a differentiated in-house knowhow of direct-to-CNS administration of AAV vectors. It has accumulated a deep experience in developing gene therapies, covering the full development range from early discovery to registrational clinical phases. Its strong science and execution track record has been validated by the support of Tier-1 investors with close to $100m raised since inception and by our partnership with Sarepta.

2019 and the start to 2020 have been years of strong execution for the company. We have made tremendous progress reaching key milestones with our two most advanced drug candidates. LYS-SAF302 for MPS IIIA has entered pivotal phase 2/3 trial in the second half of 2018, and 19 patients out of a total of 20 have been treated in just more than one year. Our second program, LYS-GM101 for GM1 gangliosidosis, is about to enter the clinicwith dosing of the first patient expected in 2020. We are proud to have been able to execute despite the Covid-19 pandemic with no impact expected on our clinical timelines, while taking all necessary measures to ensure the safety of our employees and continuity of clinical trial. 

We have further expanded our pipeline, signing a research collaboration with Yeda, the commercial arm of the Weizmann Institute of Science, one of the world’s leading multidisciplinary research institutes, to develop a novel AAV gene therapy approach for neuronopathic Gaucher disease, Parkinson disease, and other diseases associated with mutations of the GBA1 gene. This partnership is a key addition to the partnership we formed with Conectus and the IGBMC in Strasbourg to develop AAV-based gene therapies aimed at treating Fragile X syndrome, the most common hereditary cause of learning disabilities and autism spectrum disorders, and the discovery collaboration aiming at the development of novel AAV capsids with IRBM, a global partner research organization with proven experience and track record in integrated neuroscience drug discovery.  

Our partnership with Sarepta, a world leader in innovative treatments for genetic diseases, is an invaluable asset for bringing the MPSIIIA program to success. We are confident that Sarepta is the ideal partner to market LYS-SAF302 in the United States and other countries outside of Europe. This clear validation of our work was once again confirmed in March 2020 by their subscription to a new capital increase along with top-tier US investor OrbiMed. 

Finally, we have significantly strengthened our Board of Directors, welcoming Carole Deffeza seasoned executive in life science executive search and corporate governance, and Mathieu Simonwho has more than 30 years of experience in the pharmaceutical and biotech industry.  

These changes show that Lysogene has entered a new phase of its development as we become a leading player in gene therapy, approaching commercialization for our lead asset. 

Thank you for your support and trust, we are at a turning point for creating significant value for our shareholders. 

Yours sincerely, 

Karen Pignet-Aiach 

Founder and CEO 

Lysogene COVID-19 update

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