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We are a rare disease therapeutics company leading with science to make life-changing therapeutics available to patients with significant unmet needs. We involve key thought leaders, physicians, patients, care partners, and advocacy groups in all of our clinical and regulatory development strategies. With a keen understanding that drug development often requires creative solutions, we have the insight and expertise to forge new pathways to success that others have missed. By following the data without bias, our transparent narratives and common-sense perspective have successfully overcome complex development challenges to make much-needed therapies available to patients. Nimble and dauntless, we push boundaries beyond what is thought to be possible and advance new therapies that have the potential to bring meaningful improvement to patients lives.
Also known as Advantagene. Inc., we are a Massachusetts-based biotechnology company developing our proprietary immuno-oncology platforms for the treatment of solid tumors, including our Gene Mediated Cytotoxic (GMCI™) platform and our rQNestin34.5 platform. GMCI™ is an "off-the-shelf" immunotherapy designed to generate a personalized, robust, and precise systemic response from the patient's own immune system against his or her cancer.
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.
Driven by world class scientists and an entrepreneurial culture focused on rigorous science, innovative treatments, and data-driven decision making, we discover and develop medicines that have the potential to alter the treatment landscape and contribute value to patients and society. The company has developed a unique drug discovery approach, ImmunoPhage™️, that generates powerful and specific immune responses. Its most advanced program, SNS-301, is currently enrolling patients in Phase 1/2 clinical trials.
Palatin Technologies is a Cranbury, NJ-based company in the Healthcare, Pharmaceuticals, and Biotech sector.