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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.
Harmony Biosciences, LLC, is a private biopharmaceutical company headquartered in Plymouth Meeting, PA. The company was established in October 2017 with a vision to develop and commercialize novel treatment options for people living with rare diseases. With a focus on the central nervous system, including disorders of sleep and wakefulness, Harmony Biosciences is currently working to advance the understanding of narcolepsy and provide information and resources to people who live with, and healthcare professionals who treat, this chronic, debilitating neurologic disorder.
Cellectar Biosciences is developing agents to detect, treat and monitor a broad spectrum of cancers. Using a novel phospholipid ether analog (PLE) platform technology as a targeted delivery and retention vehicle, Cellectar's compounds are designed to be selectively taken up and retained in cancer cells including cancer stem cells. With the ability to attach both imaging and therapeutic agents to its proprietary delivery platform, Cellectar has developed a portfolio of product candidates engineered to leverage the unique characteristics of cancer cells to "find, treat and follow" malignancies in a highly selective way. I-124-CLR1404 is a small-molecule, broad-spectrum, cancer-targeted PET imaging agent currently being evaluated in a Phase II glioblastoma imaging trial. Additionally, multiple investigator-sponsored Phase I/II clinical trials are ongoing across 11 solid tumor indications. I-131-CLR1404 is a small-molecule, broad-spectrum, cancer-targeted molecular radiotherapeutic that delivers cytotoxic radiation directly and selectively to cancer cells including cancer stem cells. A Phase Ib dose-escalation trial of I-131-CLR1404 in patients with advanced solid tumors was completed in the first quarter of 2014 and results have been submitted to the American Society of Clinical Oncology (ASCO) 2014 Annual Meeting. CLR1502 is a preclinical, cancer-targeted, non-radioactive optical imaging agent for intraoperative tumor margin illumination and non-invasive tumor imaging.
At the forefront of the medical and digital revolutions, Voluntis is a pioneer of digital therapeutics. We design prescription applications that embed medical intelligence in the smartphones of people suffering from chronic and rare diseases. By using our software, patients leverage their health data to simplify their medical care. Treatment is personalized, doctors and patients are connected to coordinate care and real-world outcomes are improved. Harnessing its proprietary technology, Voluntis has developed digital solutions for diabetes, respiratory diseases, cancer, anticoagulation and hemophilia. Whether in our French or US offices, we are always on the look out for talent that is passionate about digital technologies and changing patients` lives.
Amer Assoc of Electrodiagnostic Medicine is a Rochester, MN-based company in the Healthcare, Pharmaceuticals, and Biotech sector.