GHP June 2017

26 GHP / June 2017 , The company’s President and CEO, Dr. Hahn-Jun Lee, M.Sc., Ph.D. is an accomplished entrepreneur with experience in launching biotech companies. The treatment being developed by Polaryx has been validated in an animal model of Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL), and has the potential to treat other types of NCLs, such as the juvenile form of NCL. Polaryx has been interviewed by respected journals and is very pleased to be featured in Global Health & Pharma magazine. The company’s core technology was originally identified by a world-renowned 1706GH01 Small Molecule Based Non-Invasive Therapy for Late Infantile Neuronal Ceroid Lipofuscinosis PolaryxTherapeutics, Inc. is solelydedicated todevelopingdrug candidates for neuronal ceroid lipofuscinosis (NCL), also called, Battendisease, forwhich there is currentlynopatient-friendly treatment optionavailable. Polaryx is repurposing anexisting safelyusedmedication, so that the treatment is patient-friendly for prolongeduse.We interviewed thefirm’s President andCEO, Dr. Hahn-JunLee,M.Sc., Ph.D., Dr. KalipdaPahan, Ph.D., Professor at RushUniversityMedical Center inChicago, andDr. JayVijayan, Ph.D.,MBA, AssociateDirector, InnovationandTechnology TransferDivision, Office of ResearchAffairs, RushUniversityMedical Center inChicago to learn more about the background and core technology. scientist, Dr. Kalipada Pahan, Ph.D, a Professor of Neurological Sciences, Biochemistry, and Pharmacology and the Floyd A. Davis, M.D., Endowed Chair in Neurology at the Rush University Medical Center in Chicago. Dr. Pahan is the Editor-in-Chief of the Journal of Multiple Sclerosis, Senior Editor of the Journal of Neuroimmune Pharmacology, Editor of three other journals, and a chartered member of the Clinical Neuroimmunology and Brain Tumours study section at the NIH. Currently, he is engaged in translational biomedical research involving multiple sclerosis, Parkinson’s disease, Alzheimer’s disease, and Batten disease. Dr. Pahan explained what Neuronal ceroid lipofuscinsosis (NCL) is. “Neuronal Ceroid Lipofuscinsosis (NCL), also known as Batten disease, is a group of neurodegenerative, lysosomal storage disorders affecting 1-4 children/100,000 live births, that leads to premature death. The major characteristic of NCL is an excessive accumulation of auto fluorescent ceroid-lipofuscin in neuronal cells and in other cells. These accumulated lipofuscins show a fluorescent greenish-yellow colour under a UV microscope. NCL starts with seizures and/or vision failure followed by progressive motor dysfunction and cognitive decline leading to a premature death.” Dr. Hahn-Jun Lee then revealed more about the scientific background behind the company’s product. “Because NCL starts with the aforementioned dysfunctions, it is often misdiagnosed as one of the other neurological disorders. NCLs are inherited in an autosomal recessive pattern, and their frequencies vary based on genetic mutations and region. Over 400 different mutations are found in 14 different genes so far.” Dr. Pahan explained the gene products and core technology in detail. “Most of the proteins encoded by the mutated genes are lysosomal, including soluble