Pipeline - Scenic Biotech

Pipeline

Discover our pipeline of first-in-class therapies that revolutionize medicine

Our pipeline

Building on our exceptional understanding of metabolic pathways, we focus our pipeline on neuro and metabolic diseases with high unmet medical need.

We have an exceptional understanding of metabolic pathways and solid expertise in designing brain penetrating compounds, which we leverage to develop novel therapies for several (neuro-) metabolic diseases. Our lead program is a first-in-class small molecule inhibitor for PLA2G15, a novel lysosomal modifier for neurodegenerative diseases, such as Batten Disease, Niemann Pick Type C (NPC) and FTLD.

In house pipeline

Lysosomal modifier (PLA2G15)

NCE, brain penetrant

Lysosomal modifier (PLA2G15)

Batten Disease, NPC & FTD

NCE, brain penetrant

Batten Disease, NPC & FTD

IND

Galactokinase (GalK)

NCE, allosteric covalent

Galactokinase (GalK)

Galactosemia

NCE, allosteric covalent

Galactosemia

Clinical candidate

Mitochondrial modifier (ABHD18)

ASO development

Mitochondrial modifier (ABHD18)

Cardiomyopathy

ASO development

Cardiomyopathy

Clinical candidate

Multiple undisclosed targets

Genetically validated

Multiple undisclosed targets

Neurometabolic

Genetically validated

Neurometabolic

Clinical candidate

Learn more

Lead program – PLA2G15 inhibitor modifying lysosomal disease

In Scenic’s most advanced program, IND-enabling studies for a small-molecule inhibitor targeting PLA2G15 are ongoing. PLA2G15 is a first-in-class drug target with broad potential for lysosomal storage disorders and neurodegenerative disease. We are currently investigating the inhibitor for rare metabolic disorders including for example Batten Disease, NPC, and FTLD.

PLA2G15 was discovered through Cell-Seq. 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/

Additional discovery and development programs

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.

Modifier therapy also holds 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.

Collaborations

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.