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Modifiers are powerful targets to develop
disease-modifying therapies with strong genetic links to disease

Building on our exceptional understanding of metabolic pathways, we focus our internal pipeline on neuro and metabolic diseases with high unmet medical need. In our partnerships, we are open to collaborate on any indication.

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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 rare genetic disorders.

In Scenic’s most advanced program, preclinical studies for a small-molecule inhibitor targeting an undisclosed, first-in-class drug target for Niemann Pick Type C (NPC) are ongoing. NPC is a lysosomal storage disorder resulting from mutations in the NPC1 gene. It affects an estimated 1:100,000 people worldwide and is marked by severe, progressive neurological deficits due to problems of the body to store and transport lipids and cholesterol correctly. We are currently investigating the inhibitor for other rare metabolic disorders.

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 Barth Syndrome, an inherited mitochondrial disorder causing cardiomyopathy. We also work on HSAN1, an inherited peripheral neuropathy, and MacTel2, an eye-disease affecting the macula.

Modifier therapy also holds great potential beyond rare 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.

Scenic’s small molecule inhibitor SC-2882 is designated for immuno-oncology. Its target QPCTL was first discovered through Scenic’s proprietary Cell-Seq platform (Logtenberg et al., Nature Medicine, 2019).

By targeting QPCTL, SC-2882 inhibits the CD47-SIRPα “don’t eat me” checkpoint on cancer cells, and simultaneously lowers immune suppression in the tumor microenvironment by modifying chemokines. This dual mechanism provides effective means for cancer cell killing by increasing the presence of anti-tumor macrophages while also priming these macrophages for phagocytosis.

SC-2882 is a potent oral inhibitor of QPCTL currently completing IND-enabling studies. The inhibitor shows preclinical efficacy in several hematological and solid tumor models, both as single agent as in combination. Additional indications include cardiovascular, fibrosis and other inflammatory diseases.

In addition to our platform and discovery collaborations, Scenic is interested in development partnerships on its pipeline. Please contact our Business Development representatives in the US or Europe or write us at

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Questions about our pipeline?

We are always interested to collaborate or partner in order to bring new treatments to patients by leveraging our platform and advance our pipeline and programs.

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