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Palvella Therapeutics was founded with the single-minded goal of serving individuals suffering from serious rare genetic diseases with no approved treatments.
AveXis, now a Novartis Company, is a clinical-stage gene therapy company relentlessly focused on bringing gene therapy out of the lab and into the clinical setting for patients and families devastated by rare and life-threatening neurological genetic d...
Aptinyx Inc. (formerly Naurex Inc.) is a biopharmaceutical company focused on discovery and development of transformative therapies for challenging disorders of the brain and nervous system. Aptinyx has a proven platform for discovering compounds that enhance synaptic plasticity, or strengthen the network for neural cell communication. Molecules discovered by Aptinyx achieve this through a novel mechanism that modulates NMDA receptors, resulting in drugs that are both highly effective and well tolerated. This mechanism has applicability across a number of brain and nervous system disorders. Aptinyx is rapidly advancing promising drug candidates into clinical development for indications with high unmet medical need.
Paragon Bioservices is an industry-leading, private-equity backed contract development and manufacturing organization (CDMO) whose focus is the development and manufacturing of cutting-edge biopharmaceuticals. Paragon`s aim is to build strong client partnerships with the world`s best biotech and pharma companies, focusing on transformative technologies, including gene therapies (AAV), next-generation vaccines, oncology immunotherapies (oncolytic viruses and CAR-T cell therapies), therapeutic protein, and other complex biologics.
Assurex Health is a personalized medicine company that specializes in helping physicians determine the right medication for individual patients who have Behavioral Health disorders. The GeneSight analysis is based on pharmacogenomics, the study of the genetic factors that influence an individual`s response to drug treatments.