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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.
Vital Therapies, Inc. is a biotherapeutic company focused on developing a cell-based system for the treatment of acute liver failure. Our product candidate, the ELAD® System, is a human cell-based, bio-artificial liver support system that operates outside the body, or extracorporeally, and is designed with the proposed intent to allow the patient’s own liver to regenerate to a healthy state, or to stabilize the patient until liver transplant. The ELAD System incorporates our human liver-derived cells, or VTL C3A cells, contained in four hollow fiber cartridges, that are combined with single use customized disposable sets and an ancillary delivery system. Data from ELAD clinical studies has shown trends that may indicate a potential to increase survival rates in patients with acute liver failure. ELAD has received orphan designation in the United States and Europe for the treatment of acute liver failure. Prior to the initiation of our ongoing Phase III clinical trial program, over 145 subjects have received therapy with the ELAD System in seven clinical trials and through a compassionate use program, which we believe collectively suggests a promising therapeutic profile. In March 2013, we initiated VTI-208, a Phase III randomized, controlled clinical trial in 200 subjects with alcohol-induced liver decompensation. We reached the midpoint in enrollment of this trial in April 2014, and anticipate the release of preliminary data in the first half of 2015. In addition, we are conducting a second Phase III randomized, controlled clinical trial, VTI-210, in 150 subjects with severe acute alcoholic hepatitis, or AAH, which is a subset of AILD, and expect to initiate enrollment of subjects later in 2014. In the second quarter of 2014, we began enrollment of a Phase II clinical trial of the ELAD System in subjects with either fulminant hepatic failure, or FHF, or surgery-induced acute liver failure, or SILF. We anticipate the release of data from VTI-210 in 2016 and the Phase II component of VTI-212 in 2015 or 2016.
Unlocking the secrets of life itself is the driving force behind the Salk Institute. Our team of world-class, award-winning scientists pushes the boundaries of knowledge in areas such as neuroscience, cancer research, aging, immunobiology, plant biology, computational biology, and more. Founded by Jonas Salk, developer of the first safe and effective polio vaccine, the Institute is an independent, nonprofit research organization and architectural landmark: small by choice, intimate by nature, and fearless in the face of any challenge.
insitro is a data-driven drug discovery and development company that leverages machine learning and high-throughput biology to transform the way medicines are created to help patients. At insitro, we are rethinking the entire drug discovery process, from the perspective of machine learning, human genetics, and high-throughput, quantitative biology. Over the past five decades, we have seen the development of new medicines becoming increasingly more difficult and expensive, leaving many patients with significant unmet need. We`re embarking on a new approach to drug development – one that leverages machine learning and unique in vitro strategies for modeling disease state and designing new therapeutic interventions. We aim to eliminate key bottlenecks in traditional drug discovery, so we can help more people sooner and at a much lower cost to the patient and the healthcare industry. We believe that by harnessing the power of technology to interrogate and measure human biology, we can have a major impact on many diseases. We invest heavily in cutting edge bioengineering technologies to enable the construction of large-scale, high-quality data sets that are designed specifically to drive machine learning methods. Our first application is to use human genetics, functional genomics, and machine learning to build a new generation of in vitro human cell-derived disease models whose response to perturbation is designed to be predictive of human clinical outcomes.
We have developed a high-sensitivity next-generation sequencing-based system for the detection of cancer mutations in miniscule concentrations of circulating cell-free DNA (cfDNA) in blood. Our technology addresses the significant barriers associated with harnessing the information contained in cfDNA by combining our proprietary techniques in molecular biology and computational algorithm for error suppression. With our technology, nanogram quantities of highly-fragmented cfDNA can be amplified more than 1000-fold, and a panel of tens to hundreds of cancer-related genes can be screened with a sensitivity at the single-digit molecular level.