Our therapeutic programs are based on genetic modifier targets
leading to a new generation of
innovative medicines that
rebalance health for patients
with devastating conditions
including inherited diseases
Scenic’s QPCTL immuno-oncology program builds on seminal work by the Netherlands Cancer Institute (NKI) and Leiden University Medical Centre (LUMC). The enzyme named QPCTL was first demonstrated as a promising target for immuno-oncological use by applying the Company’s proprietary high-resolution genetics platform called Cell-Seq. The results of this discovery were published in the journal Nature Medicine.
Druggable modifier, “dual approach”
After being the first to discover and validate QPCTL in vivo as a promising target in immuno-oncology, NKI and LUMC scientists also showed that small molecule inhibitors of QPCTL can modify the activity of functional CD47 on cancer cells. CD47 is an innate immune checkpoint, and 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’. QPCTL was found to be a druggable modifier of CD47. More recent findings show that QPCTL also modifies another important I-O axis, namely that of chemokine-signaling, and therefore has the potential to impact the tumor micro environment.
Scenic is now developing a series of proprietary chemical molecules with potent inhibition against QPCTL and has filed patent applications related to various chemical series.
Learn more – read our white paper entitled: “QPCTL is a novel drug target to modify immune checkpoint CD47”
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, 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.