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NORD National Organization for Rare Disorders
QRX008
VLA-4 inhibition for treatment of Scleroderma
Systemic sclerosis (Scleroderma) is a multisystem, autoimmune disorder defined by progressive vascular, inflammatory, and fibrotic dysfunction. It is characterised by an early inflammatory phase, which is followed by progressive fibrosis of varying extent and distribution, resulting in severe dysfunction of involved organs. Interaction of vascular cell adhesion molecule 1 (VCAM-1) with its ligand, very late antigen 4 (VLA-4), is a key process in inflammatory disease. There is an established genetic and clinical link for VCAM-1 in Scleroderma, with VCAM-1 having a genome-wide association with Scleroderma, and Scleroderma patients having elevated VCAM-1 serum levels. VLA-4, an α1β4 integrin, plays a pivotal role in controlling immune cell migration into inflamed tissue as it directly participates in cell arrest under flow. The VCAM-1:VLA-4 interaction therefore makes an attractive target for therapeutic intervention in Scleroderma. A lead series of small molecule inhibitors has been developed to disrupt the VCAM-1:VLA-4 interaction. Proof of concept has been demonstrated with VLA-4 inhibition in the Tsk1/+ mouse model of Scleroderma. Further preclinical assessment is underway for the selection of a lead compound for entry into the clinic in 2023.
Denise Carter
Ms. Carter has over 30 years of experience in the drug delivery and specialty pharmaceutical industries. Prior to Quoin, Ms. Carter held various positions with Innocoll, Inc., including president Innocoll Pharmaceuticals and executive vice president of business development and corporate affairs of Innocoll Inc. She also served as vice president of business development of the drug delivery division of West Pharmaceuticals, and she has held executive positions at Eurand and Fuisz Technologies (Biovail). Ms. Carter earned her MBA from Wharton School of Business, University of Pennsylvania and a B.S. in Chemistry from the College of William and Mary.
Genetic and Rare Diseases (GARD) Information Center
QRX007
QRX007 is a bi-functional protein designed to be highly selective and potent inhibitor of the KLK5 and KLK7 kallikreins at nanomolar concentrations as a potential treatment for Netherton Syndrome.
Netherton syndrome (NS) is an autosomal recessive skin disorder with severe skin inflammation, scaling, constant allergy and specific hair defection. NS is caused by dysfunctional mutations in the LEKTI-encoding SPINK5 gene resulted in complete loss or reduction of LEKTI activity. As LEKTI is a key regulator of epidermal proteolytic activity, loss or reduction of LEKTI activity leads to hyperactivity of serine proteases, including KLK5, KLK7 and KLK14, and subsequent over-degradation of corneodesmosomes by these proteases. Therefore, skin sections of NS patients show thinning and detachment of the stratum corneum and partial or complete loss of the stratum granulosum. Further, KLK5 is postulated to activate phospholipase A2, possibly via the PAR-2 pathway, leading to release of inflammation-mediating prostaglandins. KLK7 is also postulated to stimulate inflammatory effects in NS by activation of pro-inflammatory cytokine interleukin-1β (IL-1β). It has been demonstrated that KLK5/KLK7 double knock-out mice are protected from developing NS, potentially validating KLK5/KLK7 dual inhibition as an important therapeutic goal.
Gordon Dunn
Mr. Dunn joined Quoin Pharmaceuticals in November of 2021. He brings over 30 years of finance experience and has served as CFO of both private and publicly traded companies. In addition, Mr. Dunn has deep experience in investment banking and private equity. Over the last five years he has served as Chief Financial Officer for several private companies, most recently with Qured, a UK-based healthcare provider, from 2020 to 2021. Mr. Dunn also served as CFO of Innocoll Inc. from 2012-2016. Prior to joining Innocoll, Mr. Dunn had 20 years’ experience in investment banking and private equity, including over ten years managing the private equity funds of NewSmith Capital, and nine years at Merrill Lynch where he served as co-head of the European Private Equity Group and Director of Equity Capital Markets. Mr. Dunn received a B.A. from Stanford University and a J.D. from New York University School of Law.
Molecular Diagnostic Testing for Netherton Syndrome
QRX004
Epidermolysis Bullosa
Epidermolysis Bullosa (EB) is a group of rare and genetic skin disorders in which the skin is so fragile, even minor trauma or friction can have devastating results, causing severe pain, blistering, scarring, infections, chronic wounds and immobility. Patients can experience agonizing, traumatic and time-consuming daily dressing changes. Severe forms of EB cause disfigurement, disability and early death. Recessive Dystrophic (RDEB) is a form of dystrophic EB. RDEB is a monogenic disease resulting in chronic skin blistering and wounding. Marked by devastating, progressive, painful blistering, RDEB is diagnosed at infancy and accompanied by a high mortality rate – 76% do not live beyond their 30s. The cost of bandaging alone can exceed $10,000 per month. The cause of RDEB is a mutation in the COL7A1 gene that encodes for COL7. There is no cure or approved treatment for EB
Also formulated with the Invisicare® technology, QRX004 is a topical lotion initially under development as a potential treatment for Recessive Dystrophic Epidermolysis Bullosa (RDEB).
QRX004 contains two active ingredients. The primary ingredient induces a read-through of nonsense mutations and leads to creation of robust and sustained type VII collagen. The result is improved wound closure, reduced blistering and stronger skin.
Dennis H. Langer, MD, JD
Dr. Langer has served as a director of Quoin Inc. since 2019 and as a director of Quoin Ltd. since October 28, 2021. After graduating from Georgetown School of Medicine and Harvard Law School, Dr. Langer spent over 35 years in the pharmaceutical industry at Eli Lilly, Abbott, Searle, GSK and as President at Dr. Reddy’s Pharmaceuticals. He has also served as Chief Executive Officer at Neose Technologies, Inc. Dr. Langer has served as director at a number of companies including Sirna Therapeutics (acquired by Merck), Ception Therapeutics (acquired by Cephalon), Transkaryotic Therapies (acquired by Shire), Pharmacopeia (acquired by Ligand), Cytogen (acquired by EUSA Pharma), Brooklyn ImmunoTherapeutics, Inc. and Delcath Systems. Dr. Langer is currently a Director at Myriad Genetics, Dicerna Pharmaceuticals and Pernix Therapeutics. He is a Clinical Psychiatry Professor at Georgetown and serves on the Dean’s Advisory Board of Harvard Law School. He received an M.D. from Georgetown University School of Medicine, a J.D. from Harvard Law School, and a B.A. in Biology from Columbia University.