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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.
BiPar Sciences Incorporated is a South San Francisco, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
MicuRx mission is to discover and develop safer and more convenient antibiotics to combat drug-resistant bacterial infections. Multi-drug resistant (MDR) infections represent a major global threat to the public health, routinely forcing the use of antibiotics with high incidence of adverse effects. To combat resistant infections, physicians may increase dosing of antibiotics, or resort to less safe agents, pushing the safety envelope. Thus, healthcare providers and regulatory agencies increasingly face a difficult choice of balancing the risk of infection against unacceptably high adverse event rates from an effective but relatively toxic antibiotic. For example, in July 2016, FDA imposed a new labeling warning on the toxicity of fluoroquinolones, further restricting the use of this essential class, exemplified by anti-pseudomonal agent ciprofloxacin. Anti-MRSA agent linezolid (Zyvox®) is often used beyond its approved 14-day therapy, resulting in several myelosuppression-associated adverse effects listed in the black box warning for this important drug. MicuRx is addressing the critical need for effective but safer therapeutics by designing novel new compounds with reduced toxicity and adverse events while maintaining excellent clinical efficacy.
AllStripes is a healthcare technology company dedicated to unlocking new treatments for people with rare diseases. AllStripes has developed a technology platform that generates regulatory-ready evidence to accelerate rare disease research and drug development, as well as a patient application that empowers patients and families to securely participate in treatment research online and benefit from their own medical data. AllStripes was founded by CEO Nancy Yu and technology developer Onno Faber, following his diagnosis and journey with the rare disease neurofibromatosis type 2. The company is backed by Lux Capital, JAZZ Venture Partners, Spark Capital, Medidata Solutions, McKesson Ventures, Maveron, and a number of angel investors.
Entrada Therapeutics is a biopharmaceutical company aiming to transform the lives of patients by establishing a new class of medicines, Endosomal Escape Vehicles (EEV™), to engage intracellular targets that have long been considered inaccessible and undruggable. The Company`s EEV therapeutics are designed to enable the efficient intracellular delivery of a wide range of therapeutics into a variety of organs and tissues with an improved therapeutic index. Through its proprietary, highly versatile and modular EEV Platform, Entrada is building a robust development portfolio of oligonucleotide-, antibody- and enzyme-based programs for the potential treatment of neuromuscular disease, immunology, oncology and diseases of the central nervous system.