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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.
MouldWorks, LLC is a Beulah, MI-based company in the Healthcare, Pharmaceuticals, & Biotech sector.
MBL is a Woburn, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
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.
Practical Applications is a Boston, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.