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Sapience Therapeutics is a preclinical-stage biotechnology company focused on advancing novel therapeutics for major unmet medical needs, particularly high mortality cancers. Our drug development program involves translating science into novel therapies, and our initial goal is to develop a first-in-class treatment for glioblastoma multiforme (GBM), which is the most severe and deadly form of brain cancer.
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.
Sensorion is a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders Its clinical-stage portfolio includes one Phase 2 product: SENS-401 (Arazasetron) progressing in a planned Phase 2 proof of concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity (CIO) and, with partner Cochlear Limited, a study of SENS-401 in patients scheduled for cochlear implantation. Sensorion has entered into a broad strategic collaboration with Institut Pasteur focused on the genetics of hearing. It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness including OTOF-GT, targeting deafness caused by a mutation of the gene encoding for otoferlin, and hearing loss related to mutation in GJB2 gene to potentially address important hearing loss segments in adults and children (GJB2-GT).
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.
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.