We are at the forefront of developing compounds against genetic modifier targets
a new generation of
Scenic’s CD47/QPCTL immune-oncology program builds on seminal work by the Netherlands Cancer Institute (NKI) and Leiden University Medical Centre (LUMC). An enzyme present in cancer cells, QPCTL was first demonstrated as a promising target for immuno-oncology by using the Company’s proprietary high-resolution genetics platform called Cell-Seq. The results of this discovery were published in the journal Nature Medicine.
QPCTL was found to be a druggable modifier of the CD47 innate immune checkpoint, which is one of the major mechanisms by which cancer cells evade detection by the immune system. As a result of this activity, CD47 is also known as the ‘don’t eat me signal’.
After being the first to discover and validate QPCTL as a promising target in immuno-oncology, NKI and LUMCs scientists also showed that small molecule inhibitors of QPCTL can prevent the expression of functional CD47 on cancer cells, thereby causing the cancer cells to be attacked by macrophages and destroyed.
Scenic is now developing a series of proprietary chemical inhibitors with potent inhibition against QPCTL and has filed a patent application related to this chemical series.
Learn more – read our white paper entitled: “QPCTL is a novel drug target to modify the CD47 immune checkpoint CD47”
Scenic is actively pursuing targets in several inherited metabolic disorders. One of these programs concerns Niemann Pick Type C (NP-C). Niemann-Pick is an inherited disease that results from a mutation in the NP-C1 gene. It is a rare lipid storage disorder that affects lipid metabolism, or the way fats, lipids, and cholesterol are stored in or removed from your body. NP-CI affects an estimated 1:150,000 people worldwide.
A second program concerns Barth Syndrome, an inherited mitochondrial disorder caused by mutations in the gene encoding Tafazzin. Barth Syndrome is characterized by defects in cardiolipin, a critical component of the inner mitochondrial membrane.