Our mission is to discover and develop innovative therapies to transform the lives of people with rare central nervous system disorders. Neurodegenerative diseases that affect children are our primary target.
Lysogene is a biopharmaceutical gene therapy company specializing in treatments for central nervous system (CNS) diseases with lead programs in neurodegenerative lysosomal storage disorders.
Founded in 2009 by Karen Aiach, whose own child was affected by a neurodegenerative disease, Lysogene is rooted in a deep and compassionate understanding of the impact these diseases have on patients and families. Karen’s personal experience fuels her determination to deliver real solutions that will improve patient outcomes and enhance quality of life for both patients and caregivers. Since its inception, the company has attracted and recruited a world-class team of scientists, researchers, and product developers who are all passionately united in the creation of novel treatments for CNS diseases.
Over the past 10 years, Lysogene has acquired deep experience in developing gene therapies—from early discovery through the clinical, registration and review phases. Our strong science and execution track record has been validated by our partnership with Sarepta Therapeutics and the support of top-tier investors, with nearly $100m raised since inception.
Lysogene’s robust pipeline of therapeutics capitalizes on our differentiated knowledge of direct-to-brain administration of AAV vectors, allowing us to precisely target the causes of disease. Leveraging the most advanced gene therapy technology and our pioneering research, Lysogene is uniquely positioned to bring safe and effective treatments to market.
Our investigational therapy for the treatment of MPSIIIA (LYS-SAF302) has received Orphan Drug designation in the US and the EU and Fast Track and Rare Pediatric Disease designations in the US. Our investigational treatment for GM1 gangliosidosis (LYS-GM101) was also granted Orphan Drug designation in the US and the EU and Rare Pediatric Disease designation in the US.
Lysogene’s global community of physicians, academics, patients, families and other key stakeholders is dedicated to the pursuit of groundbreaking research, clinical trials and product development. Together, we seek to transform lives by developing cutting-edge therapies for a broad range of genetic CNS diseases with high unmet need.