Lysogene continues to make extraordinary progress and has nurtured meaningful relationships with key stakeholders worldwide. Its unparalleled expertise covers the cutting edge and relevant areas of gene therapy and orphan drug development:
Basic research: We have a thorough understanding of viral gene transfer and the design of therapeutic vectors suitable for clinical development.
Pre-clinical development: Lysogene has the specific know-how and expertise required for gene therapy, pre-clinical efficacy, toxicology and bio-distribution studies in small and large animal models. We collaborate with distinguished academic groups dedicated to this field of development.
Clinical development: Lysogene is developing models and standards to find innovative solutions in setting up gene therapy trials for rare pediatric diseases. For example we are developing a model to fully appreciate parent reported outcomes in neurodegenerative diseases. Lysogene has a close working relationship with key opinion leaders, centers of expertise and research consortia in recruiting and managing sites for trials and for understanding the disease-specific patient treatment pathways and current standards of care. Lysogene engages with patient organizations and advocacy groups for its clinical development strategy.
Specialist regulatory affairs expertise: Pediatric rare disease gene therapy presents specific challenges from a regulatory perspective. Lysogene has achieved orphan drug designations in Europe and the US. We have set up the first pediatric intracerebral gene therapy trial in Europe. We believe in the value of early discussions with the regulatory authorities in preparing our pre-clinical and clinical trials with the overall aim of achieving marketing authorization.
Health technology assessment: Gene therapy presents specific challenges in the health technology field. Lysogene was selected for an EUnetHTA JA2 to ensure an efficient timely access to its lead gene therapy product for MPS IIIA.