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Axion Pharmaceuticals is a South San Francisco, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Lysogene is a biopharmaceutical gene therapy company with lead programs in neurodegenerative lysosomal storage disorders that affect children. Founded in 2009 by Karen Aiach, whose own child was affected by a neurodegenerative disease, Lysogene is rooted in a deep and compassionate understanding of the impact these diseases have on patients and families. Karen`s personal experience fuels her determination to deliver real solutions that will improve patient outcomes and enhance quality of life for both patients and caregivers. Over the past 10 years, Lysogene has acquired deep experience in developing gene therapies—from early discovery through the clinical, registration and review phases. Our strong science and execution track record has been validated by our partnership with Sarepta Therapeutics and the support of top-tier investors, with nearly $100m raised since inception.
Morphotek is a Exton, PA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Celldex Therapeutics is a Needham Heights, MA-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.