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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.
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.
La Jolla Bioengineering Institute is a San Diego, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Hanford Pharmaceuticals is a Syracuse, NY-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
ProtoKinetix, Inc. is a molecular biotechnology company that has developed and patented a family of hyper stable, potent glycopeptides (AAGP™) that dramatically enhance the therapeutic results and reduce the cost of stem cell medicine. Due to the anti-inflammatory effect of AAGP™ molecules, the Company is currently targeting the direct treatment of diseases that have a major inflammatory component.