Lysosomal Storage Diseases Infographic
Through comprehensive data analysis, Beacon has identified 175 assets for LSDs, accounting for 8% of our database. Most of these assets are still preclinical and use classical gene therapies. We have observed that adeno-associated viruses (AAV) are the most common delivery systems used, with AAV9 being the second most common serotype leveraged due to its unique targeting capabilities.
Our analysis also revealed that most genes targeted by LSD assets are lysosomal or responsible for breaking down large carbohydrates. Large pharmaceutical companies dominate the developer landscape for LSDs due to the considerable amount of money required to translate research into clinical trials. Join us as we explore the landscape of LSD research and development.
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