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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.
Chiron Corporation was an American multinational biotechnology firm founded in 1981, based in Emeryville, California, that was acquired by Novartis on April 20, 2006.
Cidara is developing therapeutics to improve the standard of care for patients facing serious fungal or viral infections. The Company`s portfolio is comprised of breakthrough approaches aimed at transforming existing treatment and prevention paradigms, first with its lead Phase 3 antifungal candidate, rezafungin, in addition to antiviral conjugates (AVCs) targeting influenza and other viral diseases from Cidara`s proprietary Cloudbreak antiviral platform. Cidara is headquartered in San Diego, California.
Bayer and CRISPR Therapeutics have entered into an agreement to create Casebia to discover, develop and commercialize new breakthrough therapeutics to cure blood disorders, blindness, and congenital heart disease. CRISPR Therapeutics will contribute its proprietary CRISPR-Cas9 gene-editing technology and intellectual property, while Bayer will make available its protein engineering expertise and relevant disease know-how. It is the first long-term strategic partnership of its kind to make a substantial investment in the development of target delivery systems in an effort to bring systemic in vivo CRISPR-Cas9 gene editing technology applications to patients.
FASgen is a Baltimore, MD-based company in the Healthcare, Pharmaceuticals, and Biotech sector.