Imagine
Treating neurodegeneration by restoring lysosomal function at its origin
Lysosomal dysfunction is emerging as a causal, shared driver of neurodegeneration across genetic and age-related neurodegenerative diseases
Pioneers in the discovery of new drug targets to enable the development of transformative therapeutics that restore lysosomal function
Scenic Biotech is a neurometabolic company developing disease-modifying therapies that restore lysosomal function, a core driver of neurodegeneration. Its lead program, SC6177, a brain-penetrant small molecule, targets lysosomal dysfunction across genetic and age-related forms of neurodegeneration, with IND-enabling studies underway and first-in-human trials targeted for 2027.
Just as gene mutations can cause disease, we now know they can also protect us from disease. We use our unique platform to identify novel targets that re-balance disease pathways and open them up to alternative approaches for pharmacological intervention.
SC6177, a first-in-class PLA2G15 inhibitor restoring lysosomal function
Our lead program SC6177 is a brain penetrant small molecule inhibitor of PLA2G15 – a first-in-class lysosomal target with broad potential across neurodegenerative diseases. Our development strategy prioritizes rapid proof-of-concept in orphan indications, while laying the foundation for expansion into broader neurodegenerative diseases, including genetically defined and age-related patient populations. PLA2G15 was identified through our target discovery engine Cell-Seq™ and published in Nature (2025).
Cell-Seq™ — Scenic’s target discovery powerhouse
Scenic’s proprietary functional genomics engine Cell-Seq™ overcomes the long-standing challenge of identifying “hidden disease protection factors”, enabling the discovery of the novel drug targets and the development of first-in-class, disease-modifying therapies across multiple indications. Our approach is validated through partnerships with blue-chip pharma, including Genentech, Bristol Myers Squibb, Alnylam, and Ono Pharmaceutical, and underpins a growing pipeline in neurometabolic diseases.
