Pipeline 1
Pipeline

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 pipeline
Target
Validation
Clinical candidate
IND
P1/2
PoC
Lysosomal modifier (PLA2G15)
NCE, brain penetrant
Lysosomal modifier (PLA2G15)
Batten Disease, NPC & FTD
Batten Disease, NPC & FTD
IND
Galactokinase (GalK)
NCE, allosteric covalent
Galactokinase (GalK)
Galactosemia
Galactosemia
Clinical candidate
Mitochondrial modifier (ABHD18)
ASO development
Mitochondrial modifier (ABHD18)
Cardiomyopathy
Cardiomyopathy
Clinical candidate
Multiple undisclosed targets
Genetically validated
Multiple undisclosed targets
Neurometabolic
Neurometabolic
Validation

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.

Learn more

Scenic’s target discovery and validation efforts around PLA2G15 have been published in Nature in 2025:
https://pubmed.ncbi.nlm.nih.gov/40335701/

An associated Research Briefing highlights the impact of Scenic’s findings and the potential of PLA2G15 inhibition for neurodegenerative disease:
https://pubmed.ncbi.nlm.nih.gov/40335801/

We also develop a small-molecule inhibitor against GALK for the treatment of Galactosemia, a disorder of carbohydrate metabolism resulting in neurological and psychiatric deficits. Preclinical studies are currently being initiated.

Other programs include developing treatments for X-ALD, a disorder affecting the nervous system and adrenal glands, and as well as for mitochondrial disease. We also discovered targets for HSAN1, an inherited peripheral neuropathy, and MacTel2, an eye-disease affecting the macula.

Our novel therapies also hold great potential beyond genetic diseases. We integrate human genetics and our knowledge of biological pathways to investigate the potential of our inhibitors for the applicability in common indications.

Programs 2@3x

We are always interested in collaborations that help advance our discoveries into innovative treatments

In addition to leveraging our Cell-Seq platform for discovery collaborations, Scenic is interested in development partnerships on its proprietary programs.

Contact us