Discover our pipeline of first-in-class therapies that revolutionize medicine
Building on our exceptional understanding of metabolic pathways, we focus our pipeline on neuro and metabolic diseases with high unmet medical need
We are building a pipeline of first‑in‑class, disease‑modifying small molecules derived from our function‑first genomics platform Cell-Seq. 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. Additional discovery programs for several neurometabolic diseases are being advanced in parallel.
In house pipeline
In house pipelineTarget | Validation | Clinical candidate | IND | P1/2 | PoC | ||||||||
Lysosomal modifier (PLA2G15) NCE, brain penetrant | |||||||||||||
Lysosomal modifier (PLA2G15) | NCE, brain penetrant | ||||||||||||
Galactokinase (GalK) NCE, allosteric covalent | |||||||||||||
Galactokinase (GalK) | NCE, allosteric covalent | ||||||||||||
Mitochondrial modifier (ABHD18) ASO development | |||||||||||||
Mitochondrial modifier (ABHD18) | ASO development | ||||||||||||
Multiple undisclosed targets Genetically validated | |||||||||||||
Multiple undisclosed targets | Genetically validated | ||||||||||||
Lead program – SC6177, a PLA2G15 inhibitor restoring lysosomal function
Scenic Biotech’s lead program, SC6177, is a first-in-class, brain-penetrant inhibitor designed to restore lysosomal function, a fundamental biological process increasingly recognized as a key driver of neurodegenerative disease. With protein aggregation strategies (amyloid, tau) delivering limited success, the field is increasingly converging on lysosomal health as a fundamental mechanism. Positioned early in this shift, Scenic combines a novel target with brain penetrant small molecule chemistry to lead in this next wave of innovation.
SC6177 targets PLA2G15, a novel lysosomal enzyme discovered through Scenic’s proprietary Cell-Seq™ platform and published in Nature in 2025. PLA2G15 regulates Bis(monoacylglycero)phosphate (BMP), a key protecting lipid in lysosomes. Inhibiting PLA2G15 through SC6177 represents a differentiated therapeutic approach with the potential to address a broad spectrum of neurodegenerative disorders characterized by lysosomal dysfunction, including CLN3 Batten Disease, Niemann-Pick Type C, Frontotemporal Dementia and genetic forms of Parkinson’s Disease.
The program is currently advancing through IND-enabling development, with first-in-human clinical trials targeted for 2027. Our development strategy is designed to deliver initial proof-of-concept in orphan indications, followed by expansion into broader neurodegenerative populations.