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NeoPharm, Inc. is a Waukegan, IL-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Orchard Therapeutics is a leading global fully integrated commercial-stage company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies. Orchard’s portfolio of autologous ex vivo gene therapy programs has demonstrated sustained clinical benefit in over 150 patients across five disease areas. These programs include Strimvelis®, the first autologous ex vivo gene therapy approved by the EMA in 2016, 3 programs in advanced registrational studies in MLD (metachromatic leukodystrophy), WAS (Wiskott Aldrich syndrome) and ADA-SCID (adenosine deaminase severe combined immunodeficiency), 2 other clinical programs in X-CGD (X-linked chronic granulomatous disease) and beta-thalassemia, as well as an extensive preclinical pipeline. The company is partnered with world-leading institutions in gene therapy, including University College London, Great Ormond Street Hospital, the University of Manchester and Central Manchester University Hospitals, the University of California Los Angeles and Boston Children’s Hospital, and Telethon Institute of Gene Therapy/Ospedale San Raffaele. Orchard is a publicly traded company (NASDAQ: ORTX) with offices in the UK and the US, including London, San Francisco and Boston.
FMD K&L is a global contract research organization supporting data management, biostatistics, statistical programming, clinical operations, regulatory affairs, safety, pharmacovigilance, toxicology, medical affairs, medical writing, quality, risk, and compliance services to the pharmaceutical, biotechnology, and medical device industries worldwide. We continuously strive to raise the standard of excellence through accuracy and efficiency to achieve the highest quality output for our partners. K&L was established in the US in 1995, and has grown rapidly worldwide, with key offices and delivery centers in the US, UK, China, India, Armenia, Japan and throughout the Asia-Pacific region.
REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno‐associated virus (AAV) gene therapy. REGENXBIO`s NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO`s mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO`s NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third‐party NAV Technology Platform licensees.
Knopp Neurosciences is a Pittsburgh, PA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.