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Stoke Therapeutics is pioneering a new way to treat the underlying causes of severe #geneticdiseases by precisely upregulating protein expression. We develop antisense oligonucleotide medicines that increase gene expression to treat genetic epilepsies and other severe monogenic diseases, including genetic conditions affecting the central nervous system, eye, liver and kidney. At Stoke, we believe our proprietary technology platform, Targeted Augmentation of Nuclear Gene Output, or #TANGO, will allow us to deliver disease-modifying therapies in a highly precise, durable and controlled manner. We have identified approximately 2,900 genetic diseases which we believe may be amenable to TANGO. At Stoke, we are united around a common goal: to improve the lives of people living with severe genetic diseases. Stoke is located in Bedford, MA with offices in Cambridge, MA.
Oxford Nanopore Technologies aims to disrupt the paradigm of biological analysis. Our technology and commercial model has already opened up DNA analysis to researchers who previously had no direct access to sequencing technologies, freeing them up to perform analyses in their own labs, in real time or in the field. Over time, the technology will continue to improve, new form factors of the technology will be introduced and workflows will be further simplified by new preparation techniques or analysis workflows. This technology pathway is designed to enable the analysis of any living thing, by any person, in any environment. Oxford Nanopore has developed the worlds first and only nanopore DNA sequencer, the MinION. The MinION is a portable, real time, long-read, low cost device that has been designed to bring easy biological analyses to anyone, whether in scientific research, education or a range of real world applications such as disease/pathogen surveillance, environmental monitoring, food chain surveillance, self-quantification or even microgravity biology. Commercially available since 2015, the MinION is in use by a thriving community of scientists in >50 countries, where it is enabling myriad applications within the traditional laboratory environment and in the field. Nanopore sensing technology is fully scalable. The GridION X5 is a desktop device that includes compute module and the ability to run up to five MinION Flow Cells. The the high-throughput/sample number PromethION is currently being released in the PromethION Early Access Programme (PEAP). Oxford Nanopore is focused on making DNA based analyses easy enough for any user and so we are working to simplify the sample preparation and data analysis processes. For sample preparation this includes a 5-10 minute sample prep kit, and VolTRAX (in development), a rapid, programmable, portable, disposable sample preparation device designed to prepare DNA for addition to a nanopore sequencing device.
Aruvant`s mission is to bring hope to patients living with rare diseases by developing life changing and potentially curative gene therapies, with a near-term focus on sickle cell disease. Aruvant is a part of the Roivant family of companies. We have a talented team with extensive experience in the development, manufacturing and commercialization of gene therapy products. We have an active research program with a lead product candidate in development for individuals suffering from sickle cell disease (SCD). Our lead product candidate, ARU-1801, an investigational lentiviral gene therapy, is currently in a clinical trial as a potential one-time curative treatment for SCD. Preliminary clinical data from the ongoing Phase 1/2 study demonstrated durable efficacy in reducing the negative impacts of SCD. We are preparing for the launch of our pivotal clinical trial in SCD. We continue to look for new people to add to our talented team.
Corventis is a San Jose, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno‐associated virus (AAV) gene therapy. REGENXBIO`s NAV® Technology Platform, a proprietary AAV gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO`s mission is to transform the lives of patients suffering from severe diseases with significant unmet medical need by developing and commercializing in vivo gene therapy products based on REGENXBIO`s NAV Technology Platform. REGENXBIO seeks to accomplish this mission through a combination of internal development efforts and third‐party NAV Technology Platform licensees.