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BioMarin is a global pharmaceutical company focused on developing first-in-class and best-in-class therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. We remain steadfast to our original mission—to bring new treatments to market that will make a big impact on small patient populations. These patient populations are mostly children, suffering from diseases so rare, that the entire patient population can number as few as 1,000 people worldwide. These conditions are often inherited, difficult to diagnose, progressively debilitating, have few, if any, treatment options and are usually ignored. Time is critical to patients with rare diseases, and we strive to quickly develop important therapies for them. The efficiency and speed of our research, development, manufacturing, and commercial efforts is at the heart of our ability to urgently deliver therapies. Our track record of developing and commercializing new treatments has been significantly faster than the industry average and is a remarkable accomplishment that is ingrained in our culture. The company`s product portfolio comprises five marketed products and multiple clinical and pre-clinical product candidates.
Audentes is a biotechnology company committed to the development and commercialization of innovative new medicines for people with serious, rare muscle diseases. We are a passionate, energetic team driven by the goals of improving the lives of patients and building a successful business. We take pride in strong, global relationships with the patient, research, and medical communities.
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.
Color`s mission is to help people lead the healthiest lives that science and medicine can offer. We launched in April 2015 with a simple, affordable genetic test to help people understand their risk for hereditary cancer. In 2017, we added coverage for hereditary heart conditions.
Codiak is the leader in the emerging field of exosome therapeutics, working to harness the power of this cellular messenger system to create potentially life-changing medicines. A venture-backed startup founded in 2015, Codiak has rapidly built a proprietary technology platform for exosome engineering and manufacturing that allows for precise targeting of important molecular pathways, opening the door to the development of therapies for cancer and other diseases. Led by an experienced and dedicated team, Codiak has at its foundation a positive and collaborative culture that is deeply rooted in cutting edge science and promotes opportunity for growth. Recent discoveries have transformed our understanding of the role of exosomes in the body. We now know that these natural, cell-derived vesicles can act as a potent and safe delivery system for multiple therapeutic payloads. Recognizing the enormous potential of exosomes, Codiak is applying leading-edge translational science and rigorous drug development to build a pipeline of candidates with broad utility and the capacity to address currently “undruggable” targets. While our initial focus is on oncology/immuno-oncology, this approach also offers significant potential in hematology, neurology and gene therapy and other therapy areas. At Codiak, we are working to create a major shift in the development of tomorrow`s medicines through scientific ambition, remarkable ingenuity and deep passion for improving patients` lives. Located in the heart of Boston`s biotechnology hub in Cambridge, Massachusetts, we are advancing a bold vision by tapping into the unique talents of a varied pool of individuals who together are achieving truly exceptional work.