Our therapeutic programs are based on genetic modifier targets

QPCTL was first discovered and validated as a novel immuno-oncology target 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, appears well tolerated in preclinical models and shows single agent potential for treatment of both solid tumors and hematological malignancies.

Scenic is actively pursuing targets in several inherited disorders. The most advanced program concerns Niemann Pick Type C (NP-C). Niemann-Pick is an inherited disease that results from mutations in the NP-C1 gene. It is a lipid storage disorder that affects lipid metabolism, or the way different kind of lipids and cholesterol are stored in or removed from your body. NP-CI affects an estimated 1:120,000 people worldwide. Scenic discovered a new disease modifying target and is developing various NCE’s towards the clinic.

Other programs include HSAN1, an inherited peripheral neuropathy, and for the same target MacTel, an eye-disease affecting the macula.

We also work on X-ALD a disorder affecting the nervous system and adrenal glands, Galactosemia a disorder of carbohydrate metabolism, and Barth Syndrome, an inherited mitochondrial disorder characterized by defects in the biosynthesis of cardiolipin, a critical component of the inner mitochondrial membrane.