Our CD47/QPCTL immuno-oncology program is our most advanced program and is in late stage preclinical development while our most advanced rare inherited diseases program focuses on NP-C1 (Nieman Pick disease, type C) is at the early preclinical stage.
CD47/QPCTL immuno-oncology program
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, QPTCL 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”
NP-C1 (Niemann Pick disease, type C) Rare inherited diseases program
Scenic Biotech’s scientists have identified several unique, undisclosed druggable modifiers of NP-C1.
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.
Scenic is also actively pursuing targets in other inherited metabolic disorders.